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...

وصف كامل

التفاصيل البيبلوغرافية
المؤلف الرئيسي: Peddle, CF
مؤلفون آخرون: MacLaren, RE
التنسيق: أطروحة
اللغة:English
منشور في: 2021
الموضوعات: