首页> 外文OA文献 >Design of a Phase 1/2 Open-Label, Dose-Escalation Study of the Safety and Efficacy of Gene Therapy in Adults With Classic Congenital Adrenal Hyperplasia (CAH) Due to 21-hydroxylase Deficiency Through Administration of an Adeno-Associated Virus (AAV) Serotype 5-Based Recombinant Vector Encoding the Human CYP21A2 Gene

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Design of a Phase 1/2 Open-Label, Dose-Escalation Study of the Safety and Efficacy of Gene Therapy in Adults With Classic Congenital Adrenal Hyperplasia (CAH) Due to 21-hydroxylase Deficiency Through Administration of an Adeno-Associated Virus (AAV) Serotype 5-Based Recombinant Vector Encoding the Human CYP21A2 Gene

机译：设计1/2阶段开放标签，剂量升级研究，对成年人患者患者的成人安全性和疗效的效果，由于21-羟化酶缺乏给予腺相关病毒（AAV）编码人CYP21A2基因的血清型5基重组载体

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Deborah P Merke; Richard Joseph Auchus; Kyriakie Sarafoglou; Mitchell E Geffner; Mimi S Kim; Rafael D Escandon; Kamal N Bharucha; Adam J Shaywitz; Rachel Eclov; Clayton W Beard; Sophie Le Fur; Pierre F Bougneres;
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1. Congenital Adrenal Hyperplasia (CAH) due to 21-Hydroxylase Deficiency: A Comprehensive Focus on 233 Pathogenic Variants of CYP21A2 Gene [J] . Concolino Paola, Costella Alessandra Molecular diagnosis & therapy . 2018,第3期

机译：由于21-羟化酶缺乏，先天性肾上腺增生（CAH）：全面关注CYP21A2基因的233份致病变异
2. Late onset 21-hydroxylase deficiency is a genetic variant of the classic form of congenital adrenal hyperplasia (CAH) [J] . G P Chrousos, G B Cutler, D Mann, Pediatric Research . 1981,第12期

机译：迟发性21-羟化酶缺乏症是先天性肾上腺皮质增生（CAH）经典形式的遗传变异
3. CLINICAL AND MOLECULAR STUDIES OF 21 BRAZILIAN FAMILIES WITH CLASSIC CONGENITAL ADRENAL HYPERPLASIA |[lpar]|CAH|[rpar]| DUE TO 21-HYDROXYLASE DEFICIENCY [J] . G Guerra, M P Mello, S B Farah, Pediatric Research . 1995,第4期

机译：| 21例巴西先天性肾上腺皮质增生的临床和分子研究| [lpar | CAH | rpar ||由于21羟化酶缺乏症
4. The development of adeno-associated virus serotype 3 vector-based potential gene therapy for human liver cancer [D] . Ling, Chen 2011

机译：基于腺相关病毒血清型3载体的潜在基因治疗人类肝癌的研究进展
5. Molecular genetic testing of congenital adrenal hyperplasia due to 21-hydroxylase deficiency should include CAH-X chimeras [O] . Qizong Lao, Deborah P. Merke 2021

机译：由于21羟化酶缺乏引起的先天性肾上腺增生的分子遗传学试验应包括CAH-X Chimeras
6. Identification of non-amplifying CYP21 genes when using PCR-based diagnosis of 21-hydroxylase deficiency in congenital adrenal hyperplasia (CAH) affected pedigrees [O] . D. Day 1996

机译：使用基于PCR基于PCR的21-羟化酶缺陷（CAH）的诊断鉴定非扩增CYP21基因受影响的百分比

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