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Gene Therapy for Congenital Adrenal Hyperplasia through Administration of an Adeno-Associated Virus (AAV) Serotype 5-Based Recombinant Vector Encoding the Human CYP21A2

Gene Therapy for Congenital Adrenal Hyperplasia through Administration of an Adeno-Associated Virus (AAV) Serotype 5-Based Recombinant Vector Encoding the Human CYP21A2
通过使用编码人 CYP21A2 的腺相关病毒 (AAV) 血清型 5 重组载体进行先天性肾上腺增生症的基因治疗
批准号：
10916860
负责人：
Deborah Merke
金额：
$0.0万
依托单位：
CLINICAL CENTER
依托单位国家：
美国
项目类别：
财政年份：
--
资助国家：
美国
项目状态：
未结题
起止时间：
至
来源：
https://reporter.nih.gov/project-details/10916860
关键词：
21-hydroxylase deficiencyAdenovirus VectorAdrenal GlandsAdultAgeAnimalsBiodistributionCYP21A2 geneCardiovascular DiseasesCessation of lifeClinical TrialsCongenital adrenal hyperplasiaDependovirusDiagnosisDoseHumanIncidenceInjectionsLeadLifeLongevityMendelian disorderMetabolic DiseasesMonitorMusParticipantPatientsPhasePhenotypeQuality of lifeRecombinantsResearch DesignRiskSafetySerotypingShockSteroid biosynthesisWeight Gainadeno-associated viral vectorclinical developmentdesigneffective therapyefficacy evaluationenzyme activityexpression vectorfirst-in-humangene therapyhuman studyintravenous injectionmortalitynonhuman primateopen labelpre-clinicalprogramssafety assessmentsexstandard of carevectorvector genome

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中文摘要
翻译
作为一种单基因疾病，先天性肾上腺皮质增生症由于21-羟化酶缺乏是适合遗传干预。 有前途的临床前项目已经导致了首次人体临床试验。这是一项1/2期、开放标签、剂量递增研究，旨在评估向多达25名被诊断患有由于21-羟化酶缺乏症引起的典型先天性肾上腺增生的成人参与者施用基于AAV 5的基因疗法的安全性、耐受性和功效。所有接受基因治疗的受试者将被密切监测一年，并至少再随访四年，以确保安全性和有效性。
英文摘要
As a monogenic disease, congenital adrenal hyperplasia due to 21-hydroxylase deficiency is amenable to genetic interventions. Promising pre-clinical programs have led to a first-in-human clinical trial. This is a Phase 1/2, open-label, dose-escalation study designed to evaluate the safety, tolerability, and efficacy of an AAV5 based gene therapy administered to up to 25 adult participants diagnosed with classic congenital adrenal hyperplasia due to 21-hydroxylase deficiency. All participants who receive gene therapy will be closely monitored for one year and followed for a minimum of an additional four years for safety and efficacy.
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  • 批准号：
    10916862
  • 项目类别：
  • 资助金额：
    $0.0万
  • 财政年份：
    --
  • 负责人：
    Deborah Merke
  • 依托单位：
    CLINICAL CENTER
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  • 项目类别：
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  • 财政年份：
    --
  • 负责人：
    Deborah Merke
  • 依托单位：
    CLINICAL CENTER
海外基金