AAV10 for Delivery of Therapeutics to Treat ALS and Other CNS Disorders
- 详细技术说明
- AAV10 vector-based gene therapy for treating ALS with enhanced tropism for neuronal cells. Administration of rAAV encoding inhibitory RNA for superoxide dismutase 1 (SOD1) distributes widely throughou
- *Abstract
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AAV10 vector-based gene therapy for treating ALS with enhanced tropism for neuronal cells. Administration of rAAV encoding inhibitory RNA for superoxide dismutase 1 (SOD1) distributes widely throughout CNS with low toxicity. Long-term inhibition of mutant SOD1 improves lifespan in the animal models.
- *IP Issue Date
- None
- *IP Type
- Other Patent
- 国家
- Not Available
- 申请号码
- None
- 国家/地区
- 美国
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