GENE THERAPY VECTORS FOR TREATMENT OF DANON DISEASE
- 专利权人:
- Rocket Pharmaceuticals; Ltd.
- 发明人:
- Annahita KERAVALA,Simon MOORE,David RICKS
- 申请号：
- US16729002
- 公开号：
- US20200148745A1
- 申请日:
- 2019.12.27
- 申请国别(地区):
- US
- 年份:
- 2020
- 代理人:
- 摘要：
- The disclosure relates to optimized polynucleotide sequences for LAMP-2B, expression cassettes, vectors, and methods of use thereof in treating disease, e.g. Danon disease.
- 来源网站：
- 中国工程科技知识中心
