首页>外文期刊>医学、药学、卫生>临床医学>Molecular Therapy - Methods & Clinical Development

Molecular Therapy - Methods & Clinical Development

中文名称：分子疗法-方法与临床开发

ISSN：
出版周期：-

发文量：803

期刊论文

热门论文

年度选择

更多>>

全选（0）

1.Targeting Root Cause by Systemic scAAV9-hIDS Gene Delivery: Functional Correction and Reversal of Severe MPS II in Mice

机译：通过Sysic Scaav9-HIDS基因递送定位根本原因：小鼠中严重MPS II的功能校正和逆转
- 作者：Haiyan Fu;Kim Zaraspe;Naoko Murakami;Aaron S. Meadows;Ricardo J. Pineda;Douglas M. McCarty;Joseph Muenzer
- 期刊名称：《Molecular Therapy - Methods & Clinical Development》 | 2018年第3期
- MPS IIneuropathic LSDsystemic rAAV9 gene delivery;
2.An Isolated Limb Infusion Method Allows for Broad Distribution of rAAVrh74.MCK.GALGT2 to Leg Skeletal Muscles in the Rhesus Macaque

机译：孤立的肢体输注方法允许Raavrh74.mck.galgt2的广泛分布到恒河猕猴的腿部骨骼肌
- 作者：Rui Xu;Ying Jia;Deborah A. Zygmunt;Megan L. Cramer;Kelly E. Crowe;Guohong Shao;Agatha E. Maki;Haley N. Guggenheim;Benjamin C. Hood;Danielle A. Griffin;Ellyn Peterson;Brad Bolon;John P. Cheatham;Sharon L. Cheatham;Kevin M. Flanigan;Louise R. Rodino-Klapac;Louis G. Chicoine;Paul T. Martin
- 期刊名称：《Molecular Therapy - Methods & Clinical Development》 | 2018年第3期
- limb perfusiongene therapyGalgt2muscular dystrophy;
3.A Recombinant Baculovirus Efficiently Generates Recombinant Adeno-Associated Virus Vectors in Cultured Insect Cells and Larvae

机译：重组杆状病毒有效地产生培养的昆虫细胞和幼虫中的重组腺相关病毒载体
- 作者：Yang Wu;Liangyu Jiang;Hao Geng;Tian Yang;Zengpeng Han;Xiaobing He;Kunzhang Lin;Fuqiang Xu
- 期刊名称：《Molecular Therapy - Methods & Clinical Development》 | 2018年第3期
- recombinant baculovirusrAAV productioninsect cells and larvaegene therapy;
4.A Library-Based Screening Strategy for the Identification of DARPins as Ligands for Receptor-Targeted AAV and Lentiviral Vectors

机译：基于库的筛选策略，用于鉴定受体靶向AAV和慢病毒载体的配体
- 作者：Jessica Hartmann;Robert C. Münch;Ruth-Therese Freiling;Irene C. Schneider;Birgit Dreier;Washington Samukange;Joachim Koch;Markus A. Seeger;Andreas Plückthun;Christian J. Buchholz
- 期刊名称：《Molecular Therapy - Methods & Clinical Development》 | 2018年第3期
- DARPinreceptor-targeted viral vectorsLVAAVribosome displayGluA4CD105NKp46;
5.Ultrasound-Mediated Gene Therapy in Swine Livers Using Single-Element, Multi-lensed, High-Intensity Ultrasound Transducers

机译：超声介导的猪肝脏基因治疗单元素，多透镜，高强度超声换能器
- 作者：Misty L. Noble-Vranish;Shuxian Song;Kyle P. Morrison;Dominic M. Tran;Ryan R. Sun;Keith R. Loeb;George W. Keilman;Carol H. Miao
- 期刊名称：《Molecular Therapy - Methods & Clinical Development》 | 2018年第3期
- ultrasoundgene deliveryanimal studiesliverluciferaseultrasound-mediated gene transfernonviral gene transferfocused ultrasound;
6.Integrated Human Evaluation of the Lysophosphatidic Acid Pathway as a Novel Therapeutic Target in Atherosclerosis

机译：在动脉粥样硬化中的一种新的治疗靶标的溶血磷脂酸途径的整合人体评价
- 作者：Silvia Aldi;Ljubica Perisic Matic;Gregory Hamm;Dani?lle van Keulen;Dennie Tempel;Kim Holmstr?m;Agnieszka Szwajda;Boye Schnack Nielsen;Valur Emilsson;Rima Ait-Belkacem;Mariette Lengquist;Gabrielle Paulsson-Berne;Per Eriksson;Jan H.N. Lindeman;Alain J. Gool;Jonathan Stauber;Ulf Hedin;Eva Hurt-Camejo
- 期刊名称：《Molecular Therapy - Methods & Clinical Development》 | 2018年第3期
- atherosclerosistherapybiobank profiling;
7.Bioengineered Viral Platform for Intramuscular Passive Vaccine Delivery to Human Skeletal Muscle

机译：生物工程病毒平台，用于肌内被动疫苗给人骨骼肌
- 作者：Nicole K. Paulk;Katja Pekrun;Gregory W. Charville;Katie Maguire-Nguyen;Michael N. Wosczyna;Jianpeng Xu;Yue Zhang;Leszek Lisowski;Bryan Yoo;Jose G. Vilches-Moure;Gordon K. Lee;Joseph B. Shrager;Thomas A. Rando;Mark A. Kay
- 期刊名称：《Molecular Therapy - Methods & Clinical Development》 | 2018年第3期
- AAVpassive vaccinedirected evolutionskeletal musclehumanscreenfunctional transduction;
8.Targeting Heparan Sulfate Proteoglycans as a Novel Therapeutic Strategy for Mucopolysaccharidoses

机译：靶向硫酸乙酰肝素蛋白多糖作为粘性多糖类的新疗法策略
- 作者：Valeria De Pasquale;Patrizia Sarogni;Valeria Pistorio;Giuliana Cerulo;Simona Paladino;Luigi Michele Pavone
- 期刊名称：《Molecular Therapy - Methods & Clinical Development》 | 2018年第3期
- cell signalingfibroblast growth factorglycosaminoglycanheparan sulfatelysosomal storage diseasesmucopolysaccharidosis;
9.Toxicology Study of Intra-Cisterna Magna Adeno-Associated Virus 9 Expressing Iduronate-2-Sulfatase in Rhesus Macaques

机译：在岩石猕猴中表达近磺酸-2-硫酸酶的Cisterna Magna腺癌腺癌腺相关病毒9的毒理学研究
- 作者：Juliette Hordeaux;Christian Hinderer;Tamara Goode;Elizabeth L. Buza;Peter Bell;Roberto Calcedo;Laura K. Richman;James M. Wilson
- 期刊名称：《Molecular Therapy - Methods & Clinical Development》 | 2018年第3期
- AAV9intrathecalMPS II;
10.Inferring Novel Tumor Suppressor Genes with a Protein-Protein Interaction Network and Network Diffusion Algorithms

机译：用蛋白质 - 蛋白质相互作用网络和网络扩散算法推断新型肿瘤抑制基因
- 作者：Lei Chen;Yu-Hang Zhang;Zhenghua Zhang;Tao Huang;Yu-Dong Cai
- 期刊名称：《Molecular Therapy - Methods & Clinical Development》 | 2018年第3期
- tumor suppressor geneLaplacian heat diffusionrandom walk with restartprotein-protein interaction networkpermutation test;
11.Toxicology Study of Intra-Cisterna Magna Adeno-Associated Virus 9 Expressing Human Alpha-L-Iduronidase in Rhesus Macaques

机译：在岩石猕猴中表达人α-L-incuronidase的Cisterna Magna腺瘤腺癌腺癌酶的毒理学研究
- 作者：Juliette Hordeaux;Christian Hinderer;Tamara Goode;Nathan Katz;Elizabeth L. Buza;Peter Bell;Roberto Calcedo;Laura K. Richman;James M. Wilson
- 期刊名称：《Molecular Therapy - Methods & Clinical Development》 | 2018年第3期
- AAV9intrathecalMPS I;
12.Prevalence of Pre-existing Antibodies to CRISPR-Associated Nuclease Cas9 in the USA Population

机译：预先存在于美国人群中富含CRISPR相关的核酸酶CAS的预先存在的抗体
- 作者：Vijaya L. Simhadri;Joseph McGill;Shane McMahon;Junxia Wang;Haiyan Jiang;Zuben E. Sauna
- 期刊名称：《Molecular Therapy - Methods & Clinical Development》 | 2018年第3期
- CRISPR/Cas9immunogenicityanti-drug antibodiespre-existing antibodiesgene editingdrug development;
13.Validation of an Immunoassay for Anti-thymidine Phosphorylase Antibodies in Patients with MNGIE Treated with Enzyme Replacement Therapy

机译：用酶替代疗法治疗MIGE的抗胸苷磷酸化酶抗体免疫测定验证
- 作者：Michelle Levene;Dario Pacitti;Charlotte Gasson;Jamie Hall;Marcia Sellos-Moura;Bridget E. Bax
- 期刊名称：《Molecular Therapy - Methods & Clinical Development》 | 2018年第3期
- assay validationbridging immunoassayenzyme replacement therapyMNGIEthymidine phosphorylase;
14.Multiple Integrated Non-clinical Studies Predict the Safety of Lentivirus-Mediated Gene Therapy for β-Thalassemia

机译：多综合的非临床研究预测β-地中海贫血的慢病毒介导的基因治疗的安全性
- 作者：Maria Rosa Lidonnici;Ylenia Paleari;Francesca Tiboni;Giacomo Mandelli;Claudia Rossi;Michela Vezzoli;Annamaria Aprile;Carsten Werner Lederer;Alessandro Ambrosi;Franck Chanut;Francesca Sanvito;Andrea Calabria;Valentina Poletti;Fulvio Mavilio;Eugenio Montini;Luigi Naldini;Patrizia Cristofori;Giuliana Ferrari
- 期刊名称：《Molecular Therapy - Methods & Clinical Development》 | 2018年第3期
- thalassemiagene therapyhematopoiesishematopoietic stem cellpreclinical modelsafetylentiviral vectorbiodistributiongenotoxicityintegration site;
15.Destabilizing Domains Enable Long-Term and Inert Regulation of GDNF Expression in the Brain

机译：不稳定的域使大脑中GDNF表达的长期和惰性调节能够
- 作者：Luis Quintino;Angrit Namislo;Marcus Davidsson;Ludivine S. Breger;Patrick Kavanagh;Martino Avallone;Erika Elgstrand-Wettergren;Christina Isaksson;Cecilia Lundberg
- 期刊名称：《Molecular Therapy - Methods & Clinical Development》 | 2018年第3期
- GDNFdestabilizing domainsin?vivogene therapyParkinson’s disease;
16.Dose-Dependent Effects of FKRP Gene-Replacement Therapy on Functional Rescue and Longevity in Dystrophic Mice

机译：FKRP基因替代疗法对营养不良小鼠功能救援和长寿的剂量依赖性作用
- 作者：Charles Harvey Vannoy;Victoria Leroy;Qi Long Lu
- 期刊名称：《Molecular Therapy - Methods & Clinical Development》 | 2018年第3期
- muscular dystrophy-dystroglycanopathyfukutin-related proteingene therapy;
17.P-FN12, an H4R-Based Epitope Vaccine Screened by Phage Display, Regulates the Th1/Th2 Balance in Rat Allergic Rhinitis

机译：P-FN12，噬菌体展示的基于H4R的表位疫苗，调节大鼠过敏性鼻炎中的Th1 / Th2平衡
- 作者：Yuqian Wang;Jichao Sha;Heng Wang;Lifeng An;Tie Liu;Lin Li
- 期刊名称：《Molecular Therapy - Methods & Clinical Development》 | 2018年第3期
- allergic rhinitishistamine receptor 4Th1/Th2 responsesphage display peptide libraryepitope;
18.Challenges in Advanced Therapy Medicinal Product Development: A Survey among Companies in Europe

机译：先进治疗药品开发的挑战：欧洲公司之间的调查
- 作者：Renske M.T. ten Ham;Jarno Hoekman;Anke M. H?vels;Andre W. Broekmans;Hubert G.M. Leufkens;Olaf H. Klungel
- 期刊名称：《Molecular Therapy - Methods & Clinical Development》 | 2018年第3期
- ATMPgene therapycell-based therapychallengesdevelopmentEuroperegulationmanufacturing;
19.Adeno-Associated Virus Neutralizing Antibodies in Large Animals and Their Impact on Brain Intraparenchymal Gene Transfer

机译：腺相关病毒中和大型动物中的抗体及其对脑脑内联的基因转移的影响
- 作者：Dan Wang;Li Zhong;Mengxin Li;Jia Li;Karen Tran;Lingzhi Ren;Ran He;Jun Xie;Richard P. Moser;Cara Fraser;Tim Kuchel;Miguel Sena-Esteves;Terence R. Flotte;Neil Aronin;Guangping Gao
- 期刊名称：《Molecular Therapy - Methods & Clinical Development》 | 2018年第3期
- adeno-associated virusCNS gene transfergene therapylarge animalsneutralizing antibody;
20.Development of a Novel Recombinant Adeno-Associated Virus Production System Using Human Bocavirus 1 Helper Genes

机译：使用人类Bocavirus 1辅助基因的新型重组腺相关病毒生产系统的开发
- 作者：Zekun Wang;Fang Cheng;John F. Engelhardt;Ziying Yan;Jianming Qiu
- 期刊名称：《Molecular Therapy - Methods & Clinical Development》 | 2018年第3期
- rAAVHBoV1helper geneDNA replicationgene expression;
21.MicroRNA-96 Promotes Schistosomiasis Hepatic Fibrosis in Mice by Suppressing Smad7

机译：MicroRNA-96通过抑制Smad7促进小鼠中的血吸虫病肝纤维化
- 作者：Xufeng Luo;Dongmei Zhang;Jun Xie;Qin Su;Xing He;Ruipu Bai;Guangping Gao;Weiqing Pan
- 期刊名称：《Molecular Therapy - Methods & Clinical Development》 | 2018年第3期
- schistosomiasismicroRNATGF-β/Smad7hepatic stellate cell;
22.Getting the Most: Enhancing Efficacy by Promoting Erythropoiesis and Thrombopoiesis after Gene Therapy in Mice with Hurler Syndrome

机译：充分利用最多：通过促进小鼠在休闲综合征的小鼠基因治疗后促进红细胞生成和血栓形成性，提高疗效
- 作者：Jing-fen Han;Salim S. El-Amouri;Mei Dai;Phuong Cao;Dao Pan
- 期刊名称：《Molecular Therapy - Methods & Clinical Development》 | 2018年第3期
- preclinical efficacyhematopoietic stems cellsgene therapystress erythropoiesis and thrombopoiesiserythroid and megakaryocytic lineageslysosomal storage diseaseminimum effective transgene dosagestargeted expressionmucopolysaccharidoses;
23.Micro-utrophin Improves Cardiac and Skeletal Muscle Function of Severely Affected D2/mdx Mice

机译：Micro-Utrophin改善了严重影响的D2 / MDX小鼠的心脏和骨骼肌功能
- 作者：Tahnee L. Kennedy;Simon Guiraud;Ben Edwards;Sarah Squire;Lee Moir;Arran Babbs;Guy Odom;Diane Golebiowski;Joel Schneider;Jeffrey S. Chamberlain;Kay E. Davies
- 期刊名称：《Molecular Therapy - Methods & Clinical Development》 | 2018年第3期
- DuchenneutrophinAAVD2mdxcardiomyopathycardiac cine-MRI;
24.Lentiviral Vector Purification Using Genetically Encoded Biotin Mimic in Packaging Cell

机译：使用遗传编码的生物素模拟包装细胞中的慢病毒载体纯化
- 作者：Leila Mekkaoui;Farhaan Parekh;Ekaterini Kotsopoulou;David Darling;Glenda Dickson;Gordon W. Cheung;Lucas Chan;Kirsty MacLellan-Gibson;Giada Mattiuzzo;Farzin Farzaneh;Yasuhiro. Takeuchi;Martin Pule
- 期刊名称：《Molecular Therapy - Methods & Clinical Development》 | 2018年第3期
- lentiviral vectorssynthetic peptidebiotin mimicstreptavidinpurificationaffinity chromatographycompetitive elution;
25.Alpha 1 Antitrypsin Gene Therapy Extends the Lifespan of Lupus-Prone Mice

机译：Alpha 1 Anturikrypsin基因疗法延伸狼疮易一鼠的寿命
- 作者：Ahmed Samir Elshikha;Ye Yuan;Yuanqing Lu;Mong-Jen Chen;Georges Abboud;Mohammad Ahsanul Akbar;Henrike Plate;Hedwig Wolney;Tanja Hoffmann;Eleni Tagari;Leilani Zeumer;Laurence Morel;Sihong Song
- 期刊名称：《Molecular Therapy - Methods & Clinical Development》 | 2018年第3期
- alpha 1 antitrypsinAATsystemic lupus erythematosusSLErAAVdendritic cellsDCsautoimmunityautoantibodieslife span;
26.Pre-clinical Development of a Lentiviral Vector Expressing the Anti-sickling βAS3 Globin for Gene Therapy for Sickle Cell Disease

机译：表达抗病βAS3球蛋白的慢病毒载体的临床临床发展，用于镰状细胞病的基因治疗
- 作者：Valentina Poletti;Fabrizia Urbinati;Sabine Charrier;Guillaume Corre;Roger P. Hollis;Beatriz Campo Fernandez;Samia Martin;Michael Rothe;Axel Schambach;Donald B. Kohn;Fulvio Mavilio
- 期刊名称：《Molecular Therapy - Methods & Clinical Development》 | 2018年第3期
- gene therapysickle cell diseaselentiviral vectorintegrationgenotoxicityhematopoietic stem-progenitor cellsstem cell clonalityxenograftNSG mice;
27.Barcoding of Macaque Hematopoietic Stem and Progenitor Cells: A Robust Platform to Assess Vector Genotoxicity

机译：猕猴茎和祖细胞的条形码：评估载体遗传毒性的强大平台
- 作者：Idalia M. Yabe;Lauren L. Truitt;Diego A. Espinoza;Chuanfeng Wu;Samson Koelle;Sandhya Panch;Marcus A.F. Corat;Thomas Winkler;Kyung-Rok Yu;So Gun Hong;Aylin Bonifacino;Allen Krouse;Mark Metzger;Robert E. Donahue;Cynthia E. Dunbar
- 期刊名称：《Molecular Therapy - Methods & Clinical Development》 | 2018年第3期
- lentivirusgenotoxicitygene therapynon-human primate;
28.Highly Efficient Ultracentrifugation-free Chromatographic Purification of Recombinant AAV Serotype 9

机译：重组AAV血清型9的高效超速离心色谱纯化9
- 作者：Taro Tomono;Yukihiko Hirai;Hironori Okada;Yoshitaka Miyagawa;Kumi Adachi;Shuhei Sakamoto;Yasuhiro Kawano;Hideto Chono;Junichi Mineno;Akiko Ishii;Takashi Shimada;Masafumi Onodera;Akira Tamaoka;Takashi Okada
- 期刊名称：《Molecular Therapy - Methods & Clinical Development》 | 2018年第3期
- adeno-associated virusrAAV9quaternary ammonium anion exchangersultracentrifugation-free protocol;
29.IPRF Reflection Paper on Biodistribution

机译：关于生物分布的IPRF反思论文
- 作者：Mike Havert;Ying Huang;Martin Nemec;Tiina Palom?ki;Patrick Celis;Andreas Marti;Dino Petrin;Mercedes Serabian;Masakazu Hirata
- 期刊名称：《Molecular Therapy - Methods & Clinical Development》 | 2018年第3期
30.AAV8 Gene Therapy for Crigler-Najjar Syndrome in Macaques Elicited Transgene T Cell Responses That Are Resident to the Liver

机译：AAV8基因治疗克里克尔-NAJJAR综合征在猕猴中引发了驻留在肝脏的转基因T细胞应答
- 作者：Jenny A. Greig;Roberto Calcedo;Leticia Kuri-Cervantes;Jayme M.L. Nordin;Jessica Albrecht;Erin Bote;Tamara Goode;Edward A. Chroscinski;Peter Bell;Laura K. Richman;Michael R. Betts;James M. Wilson
- 期刊名称：《Molecular Therapy - Methods & Clinical Development》 | 2018年第3期
- gene therapyAAVT cellimmune responseUGT1A1Crigler-Najjartransgene expressionliver;
31.Efficient Induction of T Cells against Conserved HIV-1 Regions by Mosaic Vaccines Delivered as Self-Amplifying mRNA

机译：用作自我放大mRNA的马赛克疫苗有效地诱导T细胞对保守的HIV-1区域
- 作者：Nathifa Moyo;Annette B. Vogel;S?ren Buus;Stephanie Erbar;Edmund G. Wee;Ugur Sahin;Tomá? Hanke
- 期刊名称：《Molecular Therapy - Methods & Clinical Development》 | 2019年第3期
- RNA vaccineHIV vaccineconserved regionstHIVconsvXsimian adenovirusMVAT cell vaccineheterologous prime-boostin vivo killingT cells;
32.Codon-Optimization of Wild-Type Adeno-Associated Virus Capsid Sequences Enhances DNA Family Shuffling while Conserving Functionality

机译：密码子优化野生型腺相关病毒衣壳序列增强了DNA系列在节省功能时洗牌
- 作者：Marti Cabanes-Creus;Samantha L. Ginn;Anais K. Amaya;Sophia H.Y. Liao;Adrian Westhaus;Claus V. Hallwirth;Patrick Wilmott;Jason Ward;Kimberley L. Dilworth;Giorgia Santilli;Arkadiusz Rybicki;Hiroyuki Nakai;Adrian J. Thrasher;Adrian C. Filip;Ian E. Alexander;Leszek Lisowski
- 期刊名称：《Molecular Therapy - Methods & Clinical Development》 | 2019年第3期
- AAVlibrarydirected evolutioncodon optimizationDNA shufflingcapsid;
33.Shifting Retroviral Vector Integrations Away from Transcriptional Start Sites via DNA-Binding Protein Domain Insertion into Integrase

机译：将逆转录病毒载体的整合远离转录起始位点通过DNA结合蛋白结构域插入整体酶
- 作者：Jung-soo Nam;Ji-eun Lee;Kwang-hee Lee;Yeji Yang;Soo-hyun Kim;Gyu-un Bae;Hohsuk Noh;Kwang-il Lim
- 期刊名称：《Molecular Therapy - Methods & Clinical Development》 | 2019年第3期
- gammaretroviral vectorsDNA-binding protein domainintegraseprimer-binding sitetranscriptional start sites;
34.Preclinical Development of an AAV8-hUGT1A1 Vector for the Treatment of Crigler-Najjar Syndrome

机译：AAV8-HUGT1A1向量治疗CRIGLER-NAJJAR综合征的临床前发展
- 作者：Fanny Collaud;Giulia Bortolussi;Laurence Guianvarc’h;Sem J. Aronson;Thierry Bordet;Philippe Veron;Severine Charles;Patrice Vidal;Marcelo Simon Sola;Stephanie Rundwasser;Delphine G. Dufour;Florence Lacoste;Cyril Luc;Laetitia v. Wittenberghe;Samia Martin;Christine Le Bec;Piter J. Bosma;Andres F. Muro;Giuseppe Ronzitti;Matthias Hebben
- 期刊名称：《Molecular Therapy - Methods & Clinical Development》 | 2019年第3期
- AAV vectorCrigler-Najjar syndromeUGT1A1liver gene transferlong-term safety;
35.Protein-Engineered Coagulation Factors for Hemophilia Gene Therapy

机译：血友病基因治疗的蛋白质工程凝血因子
- 作者：Benjamin J. Samelson-Jones;Valder R. Arruda
- 期刊名称：《Molecular Therapy - Methods & Clinical Development》 | 2019年第3期
- Hemophilia Ahemophilia Bgene therapybioengineeringfactor VIIIfactor IXfactor IX PaduaB-domain delete factor VIIIimmunogenicityprotein engineering;
36.Transcriptional Targeting and MicroRNA Regulation of Lentiviral Vectors

机译：慢病毒载体的转录靶向和微小RNA调节
- 作者：Simone Merlin;Antonia Follenzi
- 期刊名称：《Molecular Therapy - Methods & Clinical Development》 | 2019年第3期
37.Novel Chimeric Gene Therapy Vectors Based on Adeno-Associated Virus and Four Different Mammalian Bocaviruses

机译：基于腺相关病毒的新型嵌合基因治疗载体和四种不同的哺乳动物的双瓦伐病
- 作者：Julia Fakhiri;Marc A. Schneider;Jens Puschhof;Megan Stanifer;Verena Schildgen;Stefan Holderbach;Yannik Voss;Jihad El Andari;Oliver Schildgen;Steeve Boulant;Michael Meister;Hans Clevers;Ziying Yan;Jianming Qiu;Dirk Grimm
- 期刊名称：《Molecular Therapy - Methods & Clinical Development》 | 2019年第3期
- adeno-associated virusAAVbocavirusBoVcapsid engineering;
38.Engineering Nanoparticles for Targeted Delivery of Nucleic Acid Therapeutics in Tumor

机译：工程纳米粒子用于靶向肿瘤核酸治疗剂的靶向递送
- 作者：Yao Xiao;Kun Shi;Ying Qu;Bingyang Chu;Zhiyong Qian
- 期刊名称：《Molecular Therapy - Methods & Clinical Development》 | 2019年第3期
- gene deliverynanoparticlestumor therapyliposomepolymersinorganics stimuli responseco-deliverycellular uptakenuclear entry;
39.Surface-Engineered Lentiviral Vectors for Selective Gene Transfer into Subtypes of Lymphocytes

机译：表面工程的慢病毒载体，用于选择性基因转移到淋巴细胞亚型中
- 作者：Annika M. Frank;Christian J. Buchholz
- 期刊名称：《Molecular Therapy - Methods & Clinical Development》 | 2019年第3期
- lymphocytegene therapycancer immunotherapyentry targetinglentiviral vectorin?vivo gene transferCAR T?cell;
40.TALEN-Mediated Gene Editing of HBG in Human Hematopoietic Stem Cells Leads to Therapeutic Fetal Hemoglobin Induction

机译：Talen介导的人造血干细胞HBG的基因编辑导致治疗胎儿血红蛋白诱导
- 作者：Christopher T. Lux;Sowmya Pattabhi;Mason Berger;Cynthia Nourigat;David A. Flowers;Olivier Negre;Olivier Humbert;Julia G. Yang;Calvin Lee;Kyle Jacoby;Irwin Bernstein;Hans-Peter Kiem;Andrew Scharenberg;David J. Rawlings
- 期刊名称：《Molecular Therapy - Methods & Clinical Development》 | 2019年第3期
- TALENhemoglobinopathyHBGhemoglobinHPFHSCDsickle cell diseasethalassemiagene editingHSC;
41.A Self-Deleting AAV-CRISPR System for In?Vivo Genome Editing

机译：一种自我删除的AAV-CRISPR系统，用于in？体内基因组编辑
- 作者：Ang Li;Ciaran M. Lee;Ayrea E. Hurley;Kelsey E. Jarrett;Marco De Giorgi;Weiqi Lu;Karol S. Balderrama;Alexandria M. Doerfler;Harshavardhan Deshmukh;Anirban Ray;Gang Bao;William R. Lagor
- 期刊名称：《Molecular Therapy - Methods & Clinical Development》 | 2019年第3期
- CRISPR/Cas9adeno-associated virusAAVin vivo deliveryself-deletingsomatic genome editingliverAAV-CRISPRgene therapy;
42.AAV Gene Transfer with Tandem Promoter Design Prevents Anti-transgene Immunity and Provides Persistent Efficacy in Neonate Pompe Mice

机译：AAV基因转移具有串联启动子设计可防止抗转基因免疫，并在新生儿Pompe小鼠中提供持续的疗效
- 作者：Pasqualina Colella;Pauline Sellier;Helena Costa Verdera;Francesco Puzzo;Laetitia van Wittenberghe;Nicolas Guerchet;Nathalie Daniele;Bernard Gjata;Solenne Marmier;Severine Charles;Marcelo Simon Sola;Isabella Ragone;Christian Leborgne;Fanny Collaud;Federico Mingozzi
- 期刊名称：《Molecular Therapy - Methods & Clinical Development》 | 2019年第3期
- tandem promoterstransgene immunitytransgene persistenceAAVgene therapy;
43.Preproinsulin Designer Antigens Excluded from Endoplasmic Reticulum Suppressed Diabetes Development in NOD Mice by DNA Vaccination

机译：从内质网中排除的前胰岛素设计者抗原抑制了NOD小鼠的糖尿病发育通过DNA疫苗接种
- 作者：Katja Stifter;Cornelia Schuster;Jana Krieger;Andreas Spyrantis;Bernhard Otto Boehm;Reinhold Schirmbeck
- 期刊名称：《Molecular Therapy - Methods & Clinical Development》 | 2019年第3期
- type 1 diabetesmouse modelsDNA vaccinesendoplasmic reticulumpreproinsulin/proinsulin antigens;
44.Intravenous Injection of an AAV-PHP.B Vector Encoding Human Acid α-Glucosidase Rescues Both Muscle and CNS Defects in Murine Pompe Disease

机译：编码人酸α-葡糖苷酶的AAV-PHP.B载体的静脉注射注射群体损害鼠群疾病的肌肉和CNS缺陷
- 作者：Jeong-A Lim;Haiqing Yi;Fengqin Gao;Nina Raben;Priya S. Kishnani;Baodong Sun
- 期刊名称：《Molecular Therapy - Methods & Clinical Development》 | 2019年第3期
- Pompe diseaseacid alpha-glucosidase deficiencygene therapyAAV-PHP.B vectorglycogen storageCNSneurological deficits;
45.Development of a Novel Anti-CD19 Chimeric Antigen Receptor: A Paradigm for an Affordable CAR T Cell Production at Academic Institutions

机译：一种新型抗CD19嵌合抗原受体的发展：学术机构实惠的汽车T细胞生产的范式
- 作者：Maria Castella;Anna Boronat;Raquel Martín-Ibá?ez;Vanina Rodríguez;Guillermo Su?é;Miguel Caballero;Berta Marzal;Lorena Pérez-Amill;Beatriz Martín-Antonio;Julio Casta?o;Clara Bueno;Olga Balagué;Europa Azucena González-Navarro;Carles Serra-Pages;Pablo Engel;Ramon Vilella;Daniel Benitez-Ribas;Valentín Ortiz-Maldonado;Joan Cid;Jaime Tabera
- 期刊名称：《Molecular Therapy - Methods & Clinical Development》 | 2019年第3期
- chimeric antigen receptorCD19leukemialymphomaimmunotherapy4-1BBT cellpreclinical studies;
46.Engineering and Design of Chimeric Antigen Receptors

机译：嵌合抗原受体的工程与设计
- 作者：Sonia Guedan;Hugo Calderon;Avery D. Posey;Marcela V. Maus
- 期刊名称：《Molecular Therapy - Methods & Clinical Development》 | 2019年第3期
- CAR T cellsimmunotherapychimeric antigen receptorscancer;
47.Efficacy of a Bicistronic Vector for Correction of Sandhoff Disease in a Mouse Model

机译：双函数载体对小鼠模型Sandhoff疾病校正的疗效
- 作者：Evan Woodley;Karlaina J.L. Osmon;Patrick Thompson;Christopher Richmond;Zhilin Chen;Steven J. Gray;Jagdeep S. Walia
- 期刊名称：《Molecular Therapy - Methods & Clinical Development》 | 2019年第3期
- Sandhoff diseaseTay-Sachs diseaseAAV9gene therapyhexosaminidase;
48.Engineering Globin Gene Expression

机译：工程球蛋白基因表达
- 作者：Rachael Davis;Aishwarya Gurumurthy;Mir A. Hossain;Eliot M. Gunn;J?rg Bungert
- 期刊名称：《Molecular Therapy - Methods & Clinical Development》 | 2019年第3期
- hemoglobinglobinlocus control regiongene therapygene editingzinc fingerTALENCRISPR/Cas9hematopoiesis;
49.CD46 Null Packaging Cell Line Improves Measles Lentiviral Vector Production and Gene Delivery to Hematopoietic Stem and Progenitor Cells

机译：CD46空包装细胞系改善了麻疹慢病毒载体生产和基因递送至造血干和祖细胞
- 作者：Stosh Ozog;Craig X. Chen;Elizabeth Simpson;Olivia Garijo;Nina D. Timberlake;Petra Minder;Els Verhoeyen;Bruce E. Torbett
- 期刊名称：《Molecular Therapy - Methods & Clinical Development》 | 2019年第3期
- lentiviral vectorenhanced gene deliverystem cellsmeasles lentiviral vectorsyncytiaCD46CD46 knockoutmeasles glycoproteinpseudotypingvector production;
50.Rapid AAV-Neutralizing Antibody Determination with a Cell-Binding Assay

机译：用细胞结合测定的快速AAV中和抗体测定法测定
- 作者：Ping Guo;Junping Zhang;Matthew Chrzanowski;Jianhe Huang;Helen Chew;Jenni A. Firrman;Nianli Sang;Yong Diao;Weidong Xiao
- 期刊名称：《Molecular Therapy - Methods & Clinical Development》 | 2019年第3期
- AAVneutralizing antibody assaygene therapyvector;
51.Kinetics and MR-Based Monitoring of AAV9 Vector Delivery into Cerebrospinal Fluid of Nonhuman Primates

机译：动力学和基于MR-MR的AAV9载体输送到非人印象脑脊液中的脑脊液
- 作者：Kousaku Ohno;Lluis Samaranch;Piotr Hadaczek;John R. Bringas;Philip C. Allen;Vivek Sudhakar;Diane E. Stockinger;Christopher Snieckus;Michael V. Campagna;Waldy San Sebastian;Jerusha Naidoo;Haifeng Chen;John Forsayeth;Ernesto A. Salegio;Granger G.C. Hwa;Krystof S. Bankiewicz
- 期刊名称：《Molecular Therapy - Methods & Clinical Development》 | 2019年第3期
- gene therapyAAV vectornon-human primatesintrathecal infusioncisterna magnalumbarintracerebroventricularpharmacokineticsmagnetic resonance imagingcerebrospinal fluid;
52.Achieving High-Yield Production of Functional AAV5 Gene Delivery Vectors via Fedbatch in an Insect Cell-One Baculovirus System

机译：通过Fedbatch在昆虫细胞 - 一体杆状病毒系统中实现高产函数Aav5基因递送载体的高产生产
- 作者：Pranav R.H. Joshi;Laura Cervera;Ibrahim Ahmed;Oleksandr Kondratov;Sergei Zolotukhin;Joseph Schrag;Parminder S. Chahal;Amine A. Kamen
- 期刊名称：《Molecular Therapy - Methods & Clinical Development》 | 2019年第3期
- AAVgene therapy vector,s insect cell baculovirus technologyOneBacFedbatchprocess intensificationhigh yield production;
53.Quantifying Antigen-Specific T Cell Responses When Using Antigen-Agnostic Immunotherapies

机译：使用抗原 - 无症免疫疗法时量化抗原特异性T细胞反应
- 作者：Jacob P. van Vloten;Lisa A. Santry;Thomas M. McAusland;Khalil Karimi;Grant McFadden;James J. Petrik;Sarah K. Wootton;Byram W. Bridle
- 期刊名称：《Molecular Therapy - Methods & Clinical Development》 | 2019年第3期
54.CRISPR Activation Enhances In?Vitro Potency of AAV Vectors Driven by Tissue-Specific Promoters

机译：CRISPR激活增强了由组织特异性启动子驱动的AAV载体的体外效力
- 作者：Devin S. McDougald;Thu T. Duong;Katherine C. Palozola;Anson Marsh;Tyler E. Papp;Jason A. Mills;Shangzhen Zhou;Jean Bennett
- 期刊名称：《Molecular Therapy - Methods & Clinical Development》 | 2019年第3期
- AAVCRISPRCas9dCas9retinaphotoreceptor;
55.Anti-cancer Effects of a Chemically Modified miR-143 on Bladder Cancer by Either Systemic or Intravesical Treatment

机译：通过系统性或膀胱内治疗对化学改性miR-143对膀胱癌的抗癌作用
- 作者：Yuki Yoshikawa;Kohei Taniguchi;Takuya Tsujino;Kazuki Heishima;Teruo Inamoto;Tomoaki Takai;Koichiro Minami;Haruhito Azuma;Kanjiro Miyata;Kotaro Hayashi;Kazunori Kataoka;Yukihiro Akao
- 期刊名称：《Molecular Therapy - Methods & Clinical Development》 | 2019年第3期
- miR-143K-RASSOS1bladder cancerPI3K/AktMEK/Erk signalingintravesical infusion;
56.Adeno-Associated Viral Vector 2 and 9 Transduction Is Enhanced in Streptozotocin-Induced Diabetic Mouse Retina

机译：在链脲佐菌素诱导的糖尿病小鼠视网膜中增强了腺相关病毒载体2和9转导
- 作者：Si Hyung Lee;Jin Young Yang;Sanjar Madrakhimov;Ha Yan Park;Keerang Park;Tae Kwann Park
- 期刊名称：《Molecular Therapy - Methods & Clinical Development》 | 2019年第3期
- adeno-associated virusdiabetes mellitusdiabetic retinopathygene therapy;
57.miR-195 Has a Potential to Treat Ischemic and Hemorrhagic Stroke through Neurovascular Protection and Neurogenesis

机译：MiR-195有可能通过神经血管保护和神经发生来治疗缺血性和出血性卒中
- 作者：Hsin-Yun Cheng;Yung-Song Wang;Po-Yuan Hsu;Chien-Yuan Chen;Yi-Chu Liao;Suh-Hang H. Juo
- 期刊名称：《Molecular Therapy - Methods & Clinical Development》 | 2019年第3期
- microRNAstrokeneuroprotectionneural stem cellSema3A;
58.High-Efficiency Lentiviral Transduction of Human CD34+ Cells in High-Density Culture with Poloxamer and Prostaglandin E2

机译：高密度培养中人CD34 +细胞的高效慢病毒转导，用泊洛昔体和前列腺素E2
- 作者：Naoya Uchida;Tina Nassehi;Claire M. Drysdale;Jackson Gamer;Morgan Yapundich;Selami Demirci;Juan J. Haro-Mora;Alexis Leonard;Matthew M. Hsieh;John F. Tisdale
- 期刊名称：《Molecular Therapy - Methods & Clinical Development》 | 2019年第3期
- lentiviral vectorhematopoietic stem cellstransduction efficiency;
59.PGE2 and Poloxamer Synperonic F108 Enhance Transduction of Human HSPCs with a β-Globin Lentiviral Vector

机译：PGE2和泊洛沙姆Synperonic F108通过β-珠蛋白慢病毒载体增强人Hspcs的转导
- 作者：Katelyn E. Masiuk;Ruixue Zhang;Kyle Osborne;Roger P. Hollis;Beatriz Campo-Fernandez;Donald B. Kohn
- 期刊名称：《Molecular Therapy - Methods & Clinical Development》 | 2019年第3期
- HSCslentivirusgene therapysickle cell disease;
60.An AAV-SGCG Dose-Response Study in a γ-Sarcoglycanopathy Mouse Model in the Context of Mechanical Stress

机译：机械应力背景下γ-Sarcoglycanopathy小鼠模型中的AAV-SGCG剂量反应研究
- 作者：David Israeli;Jérémie Cosette;Guillaume Corre;Fatima Amor;Jér?me Poupiot;Daniel Stockholm;Marie Montus;Bernard Gjata;Isabelle Richard
- 期刊名称：《Molecular Therapy - Methods & Clinical Development》 | 2019年第3期
- LGMD2Cγ-sarcoglycanopathylimb girdle muscular dystrophygene therapytranslational medicineAAVmiRNA biomarkermechanical stress;
61.Trans-ocular Electric Current In?Vivo Enhances AAV-Mediated Retinal Gene Transduction after Intravitreal Vector Administration

机译：在玻璃体载体给药后，体内的逆眼电流αvivo增强AAV介导的视网膜基因转导
- 作者：Hongman Song;Ronald A. Bush;Yong Zeng;Haohua Qian;Zhijian Wu;Paul A. Sieving
- 期刊名称：《Molecular Therapy - Methods & Clinical Development》 | 2019年第3期
62.Targeted Gene Delivery into the Mammalian Inner Ear Using Synthetic Serotypes of Adeno-Associated Virus Vectors

机译：使用腺相关病毒载体的合成血清型靶向基因进入哺乳动物内耳
- 作者：Min-A Kim;Nari Ryu;Hye-Min Kim;Ye-Ri Kim;Byeonghyeon Lee;Tae-Jun Kwon;Jinwoong Bok;Un-Kyung Kim
- 期刊名称：《Molecular Therapy - Methods & Clinical Development》 | 2019年第3期
- gene therapygenetic hearing lossinner earadeno-associated virusserotype;
63.Human Galectin-1 Improves Sarcolemma Stability and Muscle Vascularization in the mdx Mouse Model of Duchenne Muscular Dystrophy

机译：人的Galectin-1改善了Duchenne肌营养不良的MDX小鼠模型中的肌剧稳定性和肌肉血管化
- 作者：Ryan D. Wuebbles;Vivian Cruz;Pam Van Ry;Pamela Barraza-Flores;Paul D. Brewer;Peter Jones;Dean J. Burkin
- 期刊名称：《Molecular Therapy - Methods & Clinical Development》 | 2019年第3期
- Duchenne muscular dystrophyGalectin-1protein therapeuticbiologicvascularization;
64.EU Regulatory Pathways for ATMPs: Standard, Accelerated and Adaptive Pathways to Marketing Authorisation

机译：欧盟常压的监管途径：营销授权的标准，加速和自适应途径
- 作者：Giulia Detela;Anthony Lodge
- 期刊名称：《Molecular Therapy - Methods & Clinical Development》 | 2019年第3期
- Marketing Authorisationadvanced therapy medicinal productcell therapygene therapyregulationconditional approvalaccelerated assessmentPRIMERMAT designationSakigake designation;
65.AAV5-miHTT Gene Therapy Demonstrates Sustained Huntingtin Lowering and Functional Improvement in Huntington Disease Mouse Models

机译：AAV5-MIHTT基因治疗证明了亨廷顿疾病小鼠模型的持续亨廷顿降低和功能性改进
- 作者：Elisabeth A. Spronck;Cynthia C. Brouwers;Astrid Vallès;Martin de Haan;Harald Petry;Sander J. van Deventer;Pavlina Konstantinova;Melvin M. Evers
- 期刊名称：《Molecular Therapy - Methods & Clinical Development》 | 2019年第3期
- Huntington diseasegene silencingHD mouse modelgene therapytherapeutic microRNAsadeno-associated viral vector serotype 5;
66.Highly Efficient and Selective CAR-Gene Transfer Using CD4- and CD8-Targeted Lentiviral Vectors

机译：使用CD4-和CD8靶向慢病毒载体的高效和选择性的汽车基因转移
- 作者：Arezoo Jamali;Laura Kapitza;Thomas Schaser;Ian C.D. Johnston;Christian J. Buchholz;Jessica Hartmann
- 期刊名称：《Molecular Therapy - Methods & Clinical Development》 | 2019年第3期
- transduction enhancerVectofusin-1receptor-targeted viral vectorsLVΔLNGFRprotein transfer;
67.Advanced Technology for Gene Delivery with Homing Peptides to Spinal Cord through Systemic Circulation in Mice

机译：通过小鼠的全身循环与母乳肽与脊髓一起递送的先进技术
- 作者：Tomoya Terashima;Nobuhiro Ogawa;Toshiyuki Sato;Miwako Katagi;Yuki Nakae;Junko Okano;Hiroshi Maegawa;Hideto Kojima
- 期刊名称：《Molecular Therapy - Methods & Clinical Development》 | 2019年第3期
- homing peptidesgene deliveryspinal cordsystemic circulationtargeting;
68.In?Vitro Macrophage Assay Predicts the In?Vivo Anti-inflammatory Potential of Exosomes from Human Mesenchymal Stromal Cells

机译：在体外巨噬细胞测定中预测来自人间充质基质细胞外来的体内抗炎潜力
- 作者：Natalia Pacienza;Ryang Hwa Lee;Eun-Hye Bae;Dong-ki Kim;Qisong Liu;Darwin J. Prockop;Gustavo Yannarelli
- 期刊名称：《Molecular Therapy - Methods & Clinical Development》 | 2019年第3期
- in-process assaybioactivityextracellular vesicles;
69.Improved Loading of Plasma-Derived Extracellular Vesicles to Encapsulate Antitumor miRNAs

机译：改善血浆衍生细胞外囊泡的载荷以包封抗肿瘤miRNA
- 作者：Margherita Alba Carlotta Pomatto;Benedetta Bussolati;Sergio D’Antico;Sara Ghiotto;Ciro Tetta;Maria Felice Brizzi;Giovanni Camussi
- 期刊名称：《Molecular Therapy - Methods & Clinical Development》 | 2019年第3期
- exosomeelectroporationvesicle engineeringdrug deliverymiRNA loadingmiRNA enrichmentmiR-31miR-451ahepatocarcinomacancer therapy;
70.Bridging from Intramuscular to Limb Perfusion Delivery of rAAV: Optimization in a Non-human Primate Study

机译：从肌肉内弥合到RAAV的肺灌注递送：在非人类灵长类动物研究中优化
- 作者：Alisha M. Gruntman;Gwladys Gernoux;Qiushi Tang;Guo-Jie Ye;Dave R. Knop;Gensheng Wang;Janet Benson;Kristen E. Coleman;Allison M. Keeler;Christian Mueller;Louis G. Chicoine;Jeffrey D. Chulay;Terence R. Flotte
- 期刊名称：《Molecular Therapy - Methods & Clinical Development》 | 2019年第3期
71.Strategies for the Induction of Immune Tolerance to Enzyme Replacement Therapy in Mucopolysaccharidosis Type I

机译：粘性多种子系统型酶替代疗法诱导免疫耐受症的策略
- 作者：Arunabha Ghosh;Aiyin Liao;Claire O’Leary;Jean Mercer;Karen Tylee;Anu Goenka;Rebecca Holley;Simon A. Jones;Brian W. Bigger
- 期刊名称：《Molecular Therapy - Methods & Clinical Development》 | 2019年第3期
- lysosomal storage diseasehaematopoietic stem cell transplantationHurlerimmune tolerancemucopolysaccharidosisenzyme replacement therapyantiCD4antiCD8methotrexate;
72.Induction of Expandable Tissue-Specific Progenitor Cells from Human Pancreatic Tissue through Transient Expression of Defined Factors

机译：通过瞬时表达局限性因子诱导人胰腺组织的可扩张组织特异性祖细胞
- 作者：Hirofumi Noguchi;Chika Miyagi-Shiohira;Yoshiki Nakashima;Takao Kinjo;Naoya Kobayashi;Issei Saitoh;Masami Watanabe;A. M. James Shapiro;Tatsuya Kin
- 期刊名称：《Molecular Therapy - Methods & Clinical Development》 | 2019年第3期
- induced tissue-specific progenitor cellsiTPinduced tissue-specific stem cellsiTSinduced pluripotent stem cellsiPSCsreprogramming factorspancreas;
73.In?Vitro Expansion of Anti-viral T Cells from Cord Blood by Accelerated Co-cultured Dendritic Cells

机译：通过加速共培养的树突细胞从脐带血的抗病毒T细胞的体外膨胀
- 作者：Klaudia Kuranda;Sophie Caillat-Zucman;Sylvaine You;Roberto Mallone
- 期刊名称：《Molecular Therapy - Methods & Clinical Development》 | 2019年第3期
- cord bloodexpansionvirus-specific T cellsdendritic cellscytokine cocktailstem cell transplantation;
74.AAV2/8 Anti-angiogenic Gene Therapy Using Single-Chain Antibodies Inhibits Murine Choroidal Neovascularization

机译：使用单链抗体的AAV2 / 8抗血管生成基因治疗抑制了鼠脉络膜新生血管
- 作者：Chris P. Hughes;Neil M.J. O’Flynn;Maureen Gatherer;Michelle E. McClements;Jennifer A. Scott;Robert E. MacLaren;Srinivas Goverdhan;Martin J. Glennie;Andrew J. Lotery
- 期刊名称：《Molecular Therapy - Methods & Clinical Development》 | 2019年第3期
- AAVvectorgene therapyAMDCNV mouse modelanti-VEGF scFv antibodiessingle-chainangiogenesis;
75.Hippocampal GAD67 Transduction Using rAAV8 Regulates Epileptogenesis in EL Mice

机译：使用RAAV8调节EL小鼠的海马GAD67转导
- 作者：Kuniko Shimazaki;Takashi Kobari;Keiji Oguro;Hidenori Yokota;Yuko Kasahara;Yoshiya Murashima;Eiju Watanabe;Kensuke Kawai;Takashi Okada
- 期刊名称：《Molecular Therapy - Methods & Clinical Development》 | 2019年第3期
- gene therapyEL mouseepilepsyGAD67CA3AAV8;
76.AAV9-TAZ Gene Replacement Ameliorates Cardiac TMT Proteomic Profiles in a Mouse Model of Barth Syndrome

机译：Aav9-TAZ基因更换改善了Barth综合征小鼠模型中的心脏TMT蛋白质组学曲线
- 作者：Silveli Suzuki-Hatano;Madhurima Saha;Meghan S. Soustek;Peter B. Kang;Barry J. Byrne;W. Todd Cade;Christina A. Pacak
- 期刊名称：《Molecular Therapy - Methods & Clinical Development》 | 2019年第3期
- Barth syndromeBTHSTMT proteomicsmitochondrial diseasecardiac gene therapyAAV9TMEM65tafazzinCx43gap junction localization;
77.Gene Transfer with AAV9-PHP.B Rescues Hearing in a Mouse Model of Usher Syndrome 3A and Transduces Hair Cells in a Non-human Primate

机译：与AAV9-PHP.B救出的基因转移在亚瑟综合征3A的小鼠模型中救出听力，并在非人灵长类动物中转换毛细胞
- 作者：Bence Gy?rgy;Elise J. Meijer;Maryna V. Ivanchenko;Kelly Tenneson;Frederick Emond;Killian S. Hanlon;Artur A. Indzhykulian;Adrienn Volak;K. Domenica Karavitaki;Panos I. Tamvakologos;Mark Vezina;Vladimir K. Berezovskii;Richard T. Born;Maureen O’Brien;Jean-Fran?ois Lafond;Yvan Arsenijevic;Margaret A. Kenna;Casey A. Maguire;David P. Corey
- 期刊名称：《Molecular Therapy - Methods & Clinical Development》 | 2019年第3期
- inner earAAVnon-human primatecochleahair cellsadeno-associated virus vectorhereditary deafnessgene delivery;
78.Large-Scale Generation and Characterization of Homogeneous Populations of Migratory Cortical Interneurons from Human Pluripotent Stem Cells

机译：人类多能干细胞迁移皮质型迁移性皮质间均群的大规模生成及表征
- 作者：Peiyan Ni;Haneul Noh;Zhicheng Shao;Qian Zhu;Youxin Guan;Joshua J. Park;Fatima Arif;James M. Park;Chiderah Abani;Cameron Beaudreault;Joy S. Park;Elizabeth Berry;Alexander Moghadam;Patric Stanton;John N. Hutchinson;Bill Andrews;Clare Faux;John Parnevelas;Leonard M. Eisenberg;Kyungjoon Park
- 期刊名称：《Molecular Therapy - Methods & Clinical Development》 | 2019年第3期
- cortical interneuronsGABAhuman pluripotent stem cellsdifferentiationtransplantationmaturationmigration;
79.Utilization of HEPES for Enhancing Protein Transfection into Mammalian Cells

机译：HEPES用于增强蛋白质转染哺乳动物细胞的利用率
- 作者：Shun-Hua Chen;Angel Chao;Chia-Lung Tsai;Shih-Che Sue;Chiao-Yun Lin;Yi-Zong Lee;Yi-Lin Hung;An-Shine Chao;Ann-Joy Cheng;Hsin-Shih Wang;Tzu-Hao Wang
- 期刊名称：《Molecular Therapy - Methods & Clinical Development》 | 2019年第3期
- HEPESprotein transfectionintracellularly targeting;
80.MTBVAC-Based TB-HIV Vaccine Is Safe, Elicits HIV-T Cell Responses, and Protects against Mycobacterium tuberculosis in Mice

机译：基于MTBVAC的TB-HIV疫苗是安全的，ELICITS HIV-T细胞反应，并保护小鼠结核分枝杆菌
- 作者：Esther Broset;Narcís Saubi;Núria Guitart;Nacho Aguilo;Santiago Uranga;Athina Kilpel?inen;Yoshiki Eto;Tomá? Hanke;Jesús Gonzalo-Asensio;Carlos Martín;Joan Joseph-Munné
- 期刊名称：《Molecular Therapy - Methods & Clinical Development》 | 2019年第3期
- HIVtuberculosisvaccineMTBVACBCGmycobacteriumHIVAp2auxoSCIDBALB/c;
81.Bezafibrate Enhances AAV Vector-Mediated Genome Editing in Glycogen Storage Disease Type Ia

机译：Bezafbibrate增强AAV载体介导的糖原储存疾病型IA中的基因组编辑
- 作者：Hye-Ri Kang;Lauren Waskowicz;Andrea M. Seifts;Dustin J. Landau;Sarah P. Young;Dwight D. Koeberl
- 期刊名称：《Molecular Therapy - Methods & Clinical Development》 | 2019年第3期
- glycogen storage disease type Iaadeno-associated virusbezafibrategene therapygenome editingzinc-finger nucleasepan-agonist of peroxisome proliferator-activated receptorsautophagy;
82.Novel Diagnostic Tool for p47 -Deficient Chronic Granulomatous Disease Patient and Carrier Detection

机译：P47 - 缩义慢性肉芽肿病患者和载体检测的新型诊断工具
- 作者：Dominik Wrona;Ulrich Siler;Janine Reichenbach
- 期刊名称：《Molecular Therapy - Methods & Clinical Development》 | 2019年第3期
- chronic granulomatous diseaseCGDNCF1NCF1BNCF1Cpseudogenegenetic diagnostics;
83.Generate TALE/TALEN as Easily and Rapidly as Generating CRISPR

机译：像产生CRISPR一样轻松迅速地生成故事/塔伦
- 作者：Shuyan Zhang;Huiting Chen;Jinke Wang
- 期刊名称：《Molecular Therapy - Methods & Clinical Development》 | 2019年第3期
- TALEconstructionrapidone day;
84.Preclinical Testing in Translational Animal Models of Prader-Willi Syndrome: Overview and Gap Analysis

机译：Prader-Willi综合征的翻译动物模型中的临床前测试：概述和差距分析
- 作者：K. Vanessa Carias;Rachel Wevrick
- 期刊名称：《Molecular Therapy - Methods & Clinical Development》 | 2019年第3期
- Prader-Willi syndromepreclinical studiesmouse models of diseaseoxytocindiazoxidegrowth hormonemelanocortincannabinoid;
85.Advancements and Obstacles of CRISPR-Cas9 Technology in Translational Research

机译：CRISPR-CAS9技术在翻译研究中的进步和障碍
- 作者：Liting You;Ruizhan Tong;Mengqian Li;Yuncong Liu;Jianxin Xue;You Lu
- 期刊名称：《Molecular Therapy - Methods & Clinical Development》 | 2019年第3期
- CRISPR-Cas9gene editingoff-targetgene therapytumor immunotherapy;
86.Pre-clinical Safety and Efficacy of Lentiviral Vector-Mediated Ex?Vivo Stem Cell Gene Therapy for the Treatment of Mucopolysaccharidosis IIIA

机译：慢病毒载体介导的前的临床安全性和疗效介导的EX？体内干细胞基因治疗治疗粘性多种素病IIIA
- 作者：Stuart M. Ellison;Aiyin Liao;Shaun Wood;Jessica Taylor;Amir Saam Youshani;Sam Rowlston;Helen Parker;Myriam Armant;Alessandra Biffi;Lucas Chan;Farzin Farzaneh;Rob Wynn;Simon A. Jones;Paul Heal;H. Bobby Gaspar;Brian W. Bigger
- 期刊名称：《Molecular Therapy - Methods & Clinical Development》 | 2019年第3期
87.The Impact of Pre-existing Immunity on the Non-clinical Pharmacodynamics of AAV5-Based Gene Therapy

机译：预先存在的免疫对基于AAV5基因治疗的非临床药效学的影响
- 作者：Brian R. Long;Krystal Sandza;Jennifer Holcomb;Lucy Crockett;Gregory M. Hayes;Jeremy Arens;Carlos Fonck;Laurie S. Tsuruda;Becky Schweighardt;Charles A. O’Neill;Stephen Zoog;Christian Vettermann
- 期刊名称：《Molecular Therapy - Methods & Clinical Development》 | 2019年第3期
- gene therapyAAVcynomolgus monkeypharmacodynamicsimmunogenicityenrollment criteriatotal antibodytransduction inhibitionhemophiliaFVIII;
88.Production of CRISPR/Cas9-Mediated Self-Cleaving Helper-Dependent Adenoviruses

机译：生产CRISPR / CAS9介导的自我切割辅助患者依赖性腺病毒
- 作者：Donna J. Palmer;Dustin L. Turner;Philip Ng
- 期刊名称：《Molecular Therapy - Methods & Clinical Development》 | 2019年第3期
- adenovirushelper-dependentCRISPRCas9gene editingindels;
89.Endothelin-1 Augments Therapeutic Potency of Human Mesenchymal Stem Cells via CDH2 and VEGF Signaling

机译：内皮素-1通过CDH2和VEGF信号传导增强人间充质干细胞的治疗效力
- 作者：Eun Ju Lee;Injoo Hwang;Gi-Hwan Kim;Dodam Moon;Su Yeon Kang;In-Chang Hwang;Seo-Yeon Lee;P.J. Marie;Hyo-Soo Kim
- 期刊名称：《Molecular Therapy - Methods & Clinical Development》 | 2019年第3期
- endothelin-1human umbilical cord blood mesenchymal stem celltherapeutic potency;
90.In-Depth Characterization of a Mifepristone-Regulated Expression System for AAV5-Mediated Gene Therapy in the Liver

机译：深入表征肝脏AAV5介导的基因治疗的米非司酮调节表达系统
- 作者：Jolanda M. Liefhebber;Raygene Martier;Tom Van der Zon;Sonay Keskin;Angelina Huseinovic;Jacek Lubelski;Bas Blits;Harald Petry;Pavlina Konstantinova
- 期刊名称：《Molecular Therapy - Methods & Clinical Development》 | 2019年第3期
- regulated gene expressionmifepristone-inducible GeneSwitch systemAAV5 gene therapy;
91.A Digestion-free Method for Quantification of Residual Host Cell DNA in rAAV Gene Therapy Products

机译：用于定量RAAV基因治疗产品中残留宿主细胞DNA的消化方法
- 作者：Yu Wang;Rebecca Cooper;Albert Kiladjian;Svetlana Bergelson;Marina Feschenko
- 期刊名称：《Molecular Therapy - Methods & Clinical Development》 | 2019年第3期
- rAAVresidual host cell DNAhAluqPCR;
92.Scalable Production of AAV Vectors in Orbitally Shaken HEK293 Cells

机译：可扩展生产AAV载体在轨道上摇摆HEK293细胞
- 作者：Daniel Blessing;Gabriel Vachey;Catherine Pythoud;Maria Rey;Vivianne Padrun;Florian M. Wurm;Bernard L. Schneider;Nicole Déglon
- 期刊名称：《Molecular Therapy - Methods & Clinical Development》 | 2019年第3期
- adeno-associated virusAAV8AAV9suspension HEK293 cellstransient transfectionorbitally shaken bioreactorsimmune affinity chromatographyCNS transduction;
93.Dissecting the Pharmacodynamics and Pharmacokinetics of MSCs to Overcome Limitations in Their Clinical Translation

机译：解剖MSCs的药效学和药代动力学克服临床翻译中的限制
- 作者：Michela Salvadori;Nicola Cesari;Alba Murgia;Paola Puccini;Benedetta Riccardi;Massimo Dominici
- 期刊名称：《Molecular Therapy - Methods & Clinical Development》 | 2019年第3期
- MSCsmesenchymal cellsmesenchymal stromal cellspharmacodynamicspharmacokineticsPK-PD modelclinical translationbiodistributionstem cell therapylung regeneration;
94.Expression of Human ACE2 in Lactobacillus and Beneficial Effects in Diabetic Retinopathy in Mice

机译：人ACE2在小鼠中乳酸杆菌和糖尿病视网膜病变的有益效果的表达
- 作者：Amrisha Verma;Kang Xu;Tao Du;Ping Zhu;Zhibing Liang;Shengquan Liao;Juantao Zhang;Mohan K. Raizada;Maria B. Grant;Qiuhong Li
- 期刊名称：《Molecular Therapy - Methods & Clinical Development》 | 2019年第3期
- Renin angiotensin systemACE2diabetesdiabetic retinopathyprobioticsLactobacillusdrug delivery;
95.Rapamycin Rescues Age-Related Changes in Muscle-Derived Stem/Progenitor Cells from Progeroid Mice

机译：雷帕霉素从葡萄蛋白小鼠中拯救与肌肉衍生的茎/祖细胞中的年龄相关的变化
- 作者：Yohei Kawakami;William S. Hambright;Koji Takayama;Xiaodong Mu;Aiping Lu;James H. Cummins;Tomoyuki Matsumoto;Takashi Yurube;Ryosuke Kuroda;Masahiro Kurosaka;Freddie H. Fu;Paul D. Robbins;Laura J. Niedernhofer;Johnny Huard
- 期刊名称：《Molecular Therapy - Methods & Clinical Development》 | 2019年第3期
- vagingmuscle regenerationmTORC1rapamycinMDSPCmultipotent differentiation;
96.GluA4-Targeted AAV Vectors Deliver Genes Selectively to Interneurons while Relying on the AAV Receptor for Entry

机译：Glua4-Targeted AAV载体在依赖于AAV受体进行进入的同时选择性地递送基因
- 作者：Jessica Hartmann;Frederic B. Thalheimer;Felix H?pfner;Thomas Kerzel;Konstantin Khodosevich;Diego García-González;Hannah Monyer;Ilka Diester;Hildegard Büning;Jan E. Carette;Pascal Fries;Christian J. Buchholz
- 期刊名称：《Molecular Therapy - Methods & Clinical Development》 | 2019年第3期
- GluA4AAVinterneuronPVparvalbumintargetingAAVR;
97.Transcutaneous Ultrasound-Mediated Nonviral Gene Delivery to the Liver in a Porcine Model

机译：经皮超声介导的非血流基因递送到猪模型中的肝脏
- 作者：Dominic M. Tran;Feng Zhang;Kyle P. Morrison;Keith R. Loeb;James Harrang;Masaki Kajimoto;Francisco Chavez;Li Wu;Carol H. Miao
- 期刊名称：《Molecular Therapy - Methods & Clinical Development》 | 2019年第3期
- ultrasoundtranscutaneousmicrobubblenonviralgene deliverygene transferliverUMGDgene therapytranshepatic venous;
98.Optimizing Modified mRNA In?Vitro Synthesis Protocol for Heart Gene Therapy

机译：用于心脏基因疗法的体外合成方案中的修饰mRNA
- 作者：Yoav Hadas;Nishat Sultana;Elias Youssef;Mohammad Tofael Kabir Sharkar;Keerat Kaur;Elena Chepurko;Lior Zangi
- 期刊名称：《Molecular Therapy - Methods & Clinical Development》 | 2019年第3期
- in vitro mRNA synthesismodified mRNAgene therapy;
99.Enhancement of Adeno-Associated Virus-Mediated Gene Therapy Using Hydroxychloroquine in Murine and Human Tissues

机译：使用羟氯喹在鼠和人组织中增强腺相关病毒介导的基因治疗
- 作者：Laurel C. Chandler;Alun R. Barnard;Sarah L. Caddy;Maria I. Patrício;Michelle E. McClements;Howell Fu;Cristina Rada;Robert E. MacLaren;Kanmin Xue
- 期刊名称：《Molecular Therapy - Methods & Clinical Development》 | 2019年第3期
- adeno-associated virusAAVgene therapyhydroxychloroquinechloroquineinnate immunityToll-like receptor 9TLR9cGASAPOBEC;
100.Neuronal Activation Stimulates Cytomegalovirus Promoter-Driven Transgene Expression

机译：神经元激活刺激越细胞病毒启动子驱动的转基因表达
- 作者：Susanne B?ck;Amanda Dossat;Ilmari Parkkinen;Pyry Koivula;Mikko Airavaara;Christopher T. Richie;Yun-Hsiang Chen;Yun Wang;Brandon K. Harvey
- 期刊名称：《Molecular Therapy - Methods & Clinical Development》 | 2019年第3期

客服邮箱：kefu@zhangqiaokeyan.com

客服微信
服务号