Preclinical Data Supporting CRISPR/Cas9 Gene Therapy for Tay-Sachs Disease
**CRISPR/Cas9 Gene Therapy for Tay-Sachs Disease: Preclinical Promise**
Preclinical studies demonstrate the potential of CRISPR/Cas9 gene therapy to effectively target and correct the genetic defect responsible for Tay-Sachs disease, offering hope for a potential cure.