AAV2/8 LSPhGAA 在晚发性庞贝病中的安全性 I 期研究-猫眼课题宝

AAV2/8 LSPhGAA 在晚发性庞贝病中的安全性 I 期研究

批准号：

9309362

负责人：

Dwight D Koeberl

金额：

$37.1万

依托单位：

DUKE UNIVERSITY

依托单位国家：

美国

项目类别：

财政年份：

2017

资助国家：

美国

项目状态：

已结题

起止时间：

2017-09-05 至 2020-06-30

来源：

https://reporter.nih.gov/project-details/9309362

关键词：

Acids Address Adult Advisory Committees Antibodies Antibody Formation Antibody Response Biological Markers Clinical Clinical Investigator Clinical Protocols Clinical Research Clinical Trials Data Development Dose Glucan 1,4-alpha-Glucosidase Glycogen Glycogen storage disease type II Goals Health Benefit Hemophilia B Human Immune response Immunoblotting Infusion procedures Institutes Investigational Drugs Investigational New Drug Application Liver Measurement Mediating Medical Monitor Muscle Myocardium Myopathy Outcome Patients Phase I Clinical Trials Plasma Production Public Health Publications Pulmonary function tests Rare Diseases Recombinant DNA Recombinants Research Institute Research Personnel Safety Scientist Skeletal Muscle Smooth Muscle Specialist Striated Muscles Testing Toxic effect United States Food and Drug Administration Walking adeno-associated viral vector base clinical translation clinically significant curative treatments design dosage enzyme replacement therapy gene therapy gene therapy clinical trial glucosidase human subject improved infancy mortality novel therapeutics phase 1 study pre-clinical preclinical development receptor safety study uptake urinary vector

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中文摘要

翻译

项目摘要基因疗法的发展已经发展到可以预见庞培病的治愈的地步。 Pompe病(第二型糖原蓄积症；酸性麦芽糖酶缺乏症)是一种破坏性的肌病由横纹肌和平滑肌中的酸性α-葡萄糖苷酶(GAA)缺乏引起。尽管有可用的重组人(Rh)GAA的酶替代疗法(ERT)，许多患者预后不佳包括临床显著的抗-GAA抗体反应所致的死亡率。ERT的局限性有推动了庞贝病基因治疗的临床前发展。《有效》的临床翻译基因治疗将通过纠正GAA缺乏症和抑制Pompe病的治疗而大大推进治疗针对重组人GAA的免疫反应。

英文摘要

Project Summary The development of gene therapy has advanced to a point where a cure for Pompe disease can be foreseen. Pompe disease (glycogen storage disease type II; acid maltase deficiency) is a devastating myopathy resulting from acid alpha-glucosidase (GAA) deficiency in striated and smooth muscle. Despite the availability of enzyme replacement therapy (ERT) with recombinant human (rh) GAA, many patients have poor outcomes including mortality due to clinically significant anti-GAA antibody response. The limitations of ERT have prompted the preclinical development of gene therapy for Pompe disease. Clinical translation of efficacious gene therapy will greatly advance treatment for Pompe disease by correcting GAA deficiency and suppressing immune responses against rhGAA.

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