AN OPEN LABEL PHASE 1/2 CLINICAL TRIAL FOR AAV8-MEDIATED LIVER-DIRECTED GENE THERAPY IN ADULTS WITH GLYCOGEN STORAGE DISEASE TYPE IA

Weinstein David A.; Ahmad Ayesha; Poma Allen; Crombez Eric

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AN OPEN LABEL PHASE 1/2 CLINICAL TRIAL FOR AAV8-MEDIATED LIVER-DIRECTED GENE THERAPY IN ADULTS WITH GLYCOGEN STORAGE DISEASE TYPE IA

机译：AAV8介导的AAV8介导的肝脏针对基因治疗的开放标签相1/2临床试验，其糖原储存疾病IA型

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来源
《Molecular genetics and metabolism 》 |2019年第3期| 共1页
作者
Weinstein David A.; Ahmad Ayesha; Poma Allen; Crombez Eric;
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作者单位

Univ Connecticut Hartford CT 06112 USA;

Univ Michigan Ann Arbor MI 48109 USA;

Ultragenyx Gene Therapy Cambridge MA USA;

Ultragenyx Gene Therapy Cambridge MA USA;

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原文格式 PDF
正文语种 eng
中图分类内分泌腺疾病及代谢病 ;
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1. AN OPEN LABEL PHASE 1/2 CLINICAL TRIAL FOR AAV8-MEDIATED LIVER-DIRECTED GENE THERAPY IN ADULTS WITH GLYCOGEN STORAGE DISEASE TYPE IA [J] . Weinstein David A., Ahmad Ayesha, Poma Allen, Molecular genetics and metabolism . 2019 ,第3期

机译：AAV8介导的AAV8介导的肝脏针对基因治疗的开放标签相1/2临床试验，其糖原储存疾病IA型
2. AN OPEN LABEL PHASE 1/2 CLINICAL TRIAL FOR AAV8-MEDIATED LIVER-DIRECTED GENE THERAPY IN ADULTS WITH GLYCOGEN STORAGE DISEASE TYPE IA [J] . Weinstein David A., Ahmad Ayesha, Poma Allen, Molecular genetics and metabolism . 2019 ,第3期

机译：AAV8介导的AAV8介导的肝脏针对基因治疗的开放标签相1/2临床试验，其糖原储存疾病IA型
3. Positive cohort 1 results from the phase 1/2, AAV8-mediated liver-directed gene therapy trial in glycogen storage disease type Ia (GSDIa) [J] . Derks T. G., Martin-Hernandez E., Ahmad A., Human gene therapy . 2019 ,第11期

机译：阳性队列1结果由糖原储存疾病型IA（GSDIA）中的AAV8介导的肝导向性基因治疗试验
4. Randomised Clinical Trials in Radiotherapy of Benign Diseases: The Paradigm of Heterotopic Ossification Prophylaxis [C] . M. Heinrich Seegenschmiedt International congress of radiation research . 2000

机译：良性疾病放射治疗的随机临床试验：异位骨化预防域的范式
5. Transition to self-management: Perspectives and experiences of glycogen storage disease type I and type III patients and their parents. [D] . King, Kellie L. 2009

机译：过渡到自我管理：I型和III型糖原贮积病患者及其父母的观点和经验。
6. Liver-directed gene therapy for murine glycogen storage disease type Ib [O] . Joon Hyun Kwon, Young Mok Lee, Jun-Ho Cho, -1

机译：肝定向基因疗法治疗Ib型鼠糖原贮积病
7. Liver-directed gene therapy for murine glycogen storage disease type Ib [O] . Joon Hyun Kwon, Young Mok Lee, Jun-Ho Cho, 2017

机译：用于鼠醋储存疾病IB的肝导向基因治疗

AN OPEN LABEL PHASE 1/2 CLINICAL TRIAL FOR AAV8-MEDIATED LIVER-DIRECTED GENE THERAPY IN ADULTS WITH GLYCOGEN STORAGE DISEASE TYPE IA

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