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...
المؤلف الرئيسي: | Peddle, CF |
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مؤلفون آخرون: | MacLaren, RE |
التنسيق: | أطروحة |
اللغة: | English |
منشور في: |
2021
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الموضوعات: |
مواد مشابهة
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AAV-CRISPR/Cas9 Gene Editing Preserves Long-Term Vision in the P23H Rat Model of Autosomal Dominant Retinitis Pigmentosa
حسب: Saba Shahin, وآخرون
منشور في: (2022-04-01) -
Herencia de la retinosis pigmentaria en la provincia Camagüey Inheritance of retinitis pigmentosa in the province of Camagüey
حسب: Elisa Dyce Gordon, وآخرون
منشور في: (1999-06-01) -
Whole exome sequencing reveals novel EYS mutations in Chinese patients with autosomal recessive retinitis pigmentosa
حسب: Xiaoqiang Xiao, وآخرون
منشور في: (2019-01-01) -
Genetic dissection of non-syndromic retinitis pigmentosa
حسب: Aarti Bhardwaj, وآخرون
منشور في: (2022-01-01) -
Visual Acuity by Decade in 139 Males with RPGR-Associated Retinitis Pigmentosa
حسب: Samantha R. De Silva, DPhil, FRCOphth, وآخرون
منشور في: (2024-03-01)