Current CRISPR therapeutic strategies for HD and FRDA are examined, including allele-specific editing, transcriptional suppression, repeat excision, epigenetic reactivation, and emerging precision editing approaches such as base editing and prime editing.
No approach yet combines adequate central nervous system distribution, mutant‐allele selectivity, durable neurological benefit, and established long‐term safety in HD research and development, but no approach yet combines adequate central nervous system distribution, mutant‐allele selectivity, durable neurological bene...
K. Zhakipbekov, Surayyo Eshkabilova, Z. Raximov et al.· Clinical and Translational D...· 0 citations
CRISPR base editors are developed that generate proteolysis-resistant HTT isoforms by disrupting the splice acceptor of HTT exon 13, an exon that encodes critical proteolytic cleavage sites implicated in N-terminal fragment production.
Shraddha Shirguppe, Michael Gapinske, Devyani Swami et al.· Nature Biomedical Engineerin...· 0 citations
These findings position somatic expansion as a promising therapeutic target and demonstrate the potential of RNAi-based cosilencing of MSH3 and HTT as a disease-modifying strategy for HD.
Jillian Belgrad, Ashley Summers, C. Landles et al.· Science Translational Medici...· 0 citations
Overall, CRISPR-based screening provides a useful framework for identifying candidate disease modifiers, but further validation across complementary human-relevant and in vivo models is required before therapeutic translation.
Feng Xue, Li-Li Bao, An-Da He et al.· Frontiers in Aging Neuroscie...· 0 citations
A knock-in mouse line at the Gt(ROSA)26Sor (Rosa26) locus carrying a single-copy, 11-kb human SOD1 genomic fragment, including all exons and introns, with the L126S mutation is generated, providing a versatile and clinically relevant platform for the preclinical evaluation of genome-editing therapies targeting heterozy...
T. Okunomiya, Tomoki Sakasai, Kayoko Tsukita et al.· Experimental animals· 0 citations
CRISPR has emerged as a next-generation gene-editing tool with the potential to target the molecular pathways associated with ageing and related disorders. It functions through RNA-guided Cas nucleases, directing DNA cleavage and utilizing the native DNA repair machinery for genetic manipulations. Advances in CRISPR te...
Sakshi Rathore, Akash Gupta, Kamal Shah et al.· Ageing Research Reviews· 0 citations
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