Development of all-in-one CRISPR/Cas9 and CRISPRi AAV constructs to treat autosomal dominant retinitis pigmentosa
<p>Dominant mutations in RHO (rhodopsin) are the most common cause of autosomal dominant retinitis pigmentosa (RHO-adRP). RHO-adRP causes progressive loss of rod cells, followed by cone cells, leading to blindness. This disease is a candidate for CRISPR gene editing, as reduction of mutant rho...
| Autor principal: | |
|---|---|
| Outros Autores: | |
| Formato: | Tese |
| Idioma: | English |
| Publicado em: |
2021
|
| Assuntos: |