Genome editing methods and constructs
A genome and gene technology, applied in gene therapy, genetic engineering, biochemical equipment and methods, etc.
- Summary
- Abstract
- Description
- Claims
- Application Information
AI Technical Summary
Problems solved by technology
Method used
Image
Examples
Embodiment 1
[0337] Example 1: HITI in the retina
[0338] Gene therapy for dominant retinitis pigmentosa via HITI in photoreceptors
[0339] Historically, most studies have focused on the use of AAV to deliver the correct copy of a loss-of-function mutated gene under recessive conditions. However, in adRP due to RHO mutation (RP4), knockout of the RHO mutant allele rather than addition of the correct RHO copy is required for therapeutic effect [47, 48]. Different strategies have been used to knock down mutant RHO in an allele-independent or allele-specific manner. Allele-independent strategies include RHO silencing using short hairpin RNA coupled to AAV-mediated RHO expression [49–51] and RHO silencing using inactive transcription factors [21]. Allele-specific strategies include allele-specific silencing and allele-specific genome editing. Genome editing using CRISPR / Cas9 (Cas9) has emerged in recent years as a versatile and effective strategy for the treatment of dominant IRD [4, 48...
PUM
Login to View More Abstract
Description
Claims
Application Information
Login to View More