CRISPR/CAS-related methods and compositions for treating cystic fibrosis
US11028394B2 · kind B2 · utility
Assignee
Inventors
Key dates
| Filing date | Oct 7, 2016 |
| Grant date | Jun 8, 2021 |
| Priority date | — |
| Expiry date | Nov 10, 2038 |
Classification
- Technology area (CPC C)Chemistry; Metallurgy
- CPC primaryC12N2320/34
- WIPO fieldBiotechnology
- WIPO sectorChemistry
Abstract
Disclosed herein are genome editing systems and compositions that target a cystic fibrosis transmembrane conductance regulator (CFTR) gene and a sodium channel epithelial 1 alpha (SCNN1A) gene, comprising a Cas9 molecule, and a gRNA molecule comprising a targeting domain that is complementary with a target sequence of a CFTR gene or a SCNN1A gene, and cells comprising such genome editing systems and compositions. Also provided are methods for using the genome editing systems, compositions, and cells for genome engineering (e.g., altering a CFTR gene and/or a SCNN1A gene), and for preventing or treating Cystic Fibrosis (CF) and CF-like disease.
Source: USPTO / EPO open patent data. Objective bibliographic and citation counts.