PARALLEL GENESIS EXPERIENCE & OPTIMIZATION

平行创世体验

• 批准号: 6220996
• 负责人: NIGEL H GODDARD
• 金额: $ 0.13万
• 依托单位: MELLON PITTS CORPORATION (MPC CORP)
• 依托单位国家: 美国
• 财政年份: 1999
• 资助国家: 美国
• 起止时间: 1999-08-01 至 2000-07-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/6220996
• 关键词: Mammalia; biomedical resource; computers; nervous system

We are engaged in research which is aimed at developing biotherapeutic molecules for use in the treatment of a class of genetic diseases known as lysosomal storage diseases. We have received approval from the National Institute of Health's Recombinant DNA Advisory Committee to initiate clinical trials to treat Gaucher disease by gene therapy. This protocol will involve delivery of an enzyme (which is deficient in this disease) by retroviral transduction of bone marrow cells. Other biomolecular strategies employing recombinant DNA technology are being studied for development of treatment modalities for human genetic disease.

我们从事的研究旨在开发 用于治疗一类疾病的生物治疗分子 遗传病称为溶酶体贮积病。 我们有 获得美国国立卫生研究院重组批准 DNA咨询委员会启动治疗戈谢病的临床试验 通过基因治疗治疗疾病。 该协议将涉及交付 通过逆转录病毒转导产生酶（该疾病缺乏） 骨髓细胞。 其他生物分子策略采用 正在研究重组DNA技术以开发 人类遗传病的治疗方式。