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...
Glavni autor: | Peddle, CF |
---|---|
Daljnji autori: | MacLaren, RE |
Format: | Disertacija |
Jezik: | English |
Izdano: |
2021
|
Teme: |
Slični predmeti
-
AAV-CRISPR/Cas9 Gene Editing Preserves Long-Term Vision in the P23H Rat Model of Autosomal Dominant Retinitis Pigmentosa
od: Saba Shahin, i dr.
Izdano: (2022-04-01) -
Herencia de la retinosis pigmentaria en la provincia Camagüey Inheritance of retinitis pigmentosa in the province of Camagüey
od: Elisa Dyce Gordon, i dr.
Izdano: (1999-06-01) -
Whole exome sequencing reveals novel EYS mutations in Chinese patients with autosomal recessive retinitis pigmentosa
od: Xiaoqiang Xiao, i dr.
Izdano: (2019-01-01) -
Genetic dissection of non-syndromic retinitis pigmentosa
od: Aarti Bhardwaj, i dr.
Izdano: (2022-01-01) -
Visual Acuity by Decade in 139 Males with RPGR-Associated Retinitis Pigmentosa
od: Samantha R. De Silva, DPhil, FRCOphth, i dr.
Izdano: (2024-03-01)