CRISPR‐based therapeutic and modelling approaches in Huntington's disease: Progress, challenges and future directions
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