Methods of treating lysosomal disorders
US11806367B2 · kind B2 · utility
0Cited by
0References
10Claims
0Family size
Assignee
Inventors
Key dates
| Filing date | Mar 15, 2018 |
| Grant date | Nov 7, 2023 |
| Priority date | — |
| Expiry date | Nov 12, 2040 |
Classification
- Technology area (CPC C)Chemistry; Metallurgy
- CPC primaryC12N2740/16043
- WIPO fieldPharmaceuticals
- WIPO sectorChemistry
Abstract
Provided herein are methods for treating a lysosomal transmembrane protein disease or disorder through ex vivo introduction of a nucleic acid molecule into hematopoietic stem and progenitor cells (HSPCs) followed by transplantation of the HSPCs into a subject in need of treatment. Also provided are vectors containing the nucleic acid molecule.
Source: USPTO / EPO open patent data. Objective bibliographic and citation counts.