Gene Therapy for Hemophilia A

A 型血友病的基因治疗

• 批准号: 6485364
• 负责人: GARRETT E BERGMAN
• 金额: $ 10万
• 依托单位: OCTAGEN CORPORATION
• 依托单位国家: 美国
• 财政年份: 2002
• 资助国家: 美国
• 起止时间: 2002-07-01 至 2003-12-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/6485364
• 关键词: blood cells; coagulation factor VIII; dogs; enzyme linked immunosorbent assay; gene delivery system; gene therapy; hemophilia As; recombinant DNA; technology /technique development; tissue /cell culture; transfection /expression vector; western blottings

DESCRIPTION (provided by applicant): Extensive efforts to develop a safe and effective method of gene therapy for hemophilia A have not met with success. In this project the applicant will test a promising ex vivo approach to delivering factor VIII using a recently identified population of cells called blood outgrowth endothelial cells or BOEC. The principal investigator has shown that engineered BOEC may safely be used to provide sustained, therapeutic Levels of fVIII in a mouse model of hemophilia A. The Specific Aim of this project is to demonstrate that engineered BOEC can be used to achieve therapeutic levels of canine coagulation factor VIII in the hemophilia A dog. In this study, BOEC will be obtained from two hemophilia A dogs, transfected with fVIII and infused into the same animals. Coagulation factor VIII levels will be assayed over time. If therapeutic levels are detected, cuticle bleed times will also be determined. If, as expected, affirmative results are obtained in this study, the applicant intends to pursue a full preclinical development plan for the BOEC technology and to file an IND for the hemophilia A indication. PROPOSED COMMERCIAL APPLICATION: BOEC represent a novel platform technology for gene therapy and other medical applications. Success in this and subsequent studies could lead to the ultimate approval of BOEC technology as a preferred therapy for hemophila A and other indications.

描述（由申请人提供）： 为开发安全且可靠的产品而付出的巨大努力 治疗A型血友病的有效基因治疗方法尚未取得成功。在 该项目申请人将测试一种有前途的离体方法来交付 因子 VIII 使用最近发现的称为血液的细胞群 生长的内皮细胞或 BOEC。主要研究人员表明 工程化的 BOEC 可安全地用于提供持续的、治疗水平的 血友病 A 小鼠模型中的 fVIII。该项目的具体目标是 证明工程化的 BOEC 可用于达到治疗水平 A 型血友病狗中的犬凝血因子 VIII。在这项研究中，BOEC 将从两只 A 型血友病狗身上获得，转染 fVIII 并输注 进入同一种动物。将检测凝血因子 VIII 水平 时间。如果检测到治疗水平，角质层出血时间也会 决定。如果正如预期的那样，本研究获得肯定的结果， 申请人打算寻求完整的临床前开发计划 BOEC 技术并提交 A 型血友病适应症的 IND 申请。 拟议的商业应用： BOEC 代表了基因治疗和其他医疗应用的新型平台技术。 本次研究及后续研究的成功可能会导致 BOEC 技术的最终批准 作为甲型血友病和其他适应症的首选疗法。