JLK-247
/ Phoenix Nest
- LARVOL DELTA
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April 10, 2025
Gene Replacement Therapy for MPS IIIC with scAAV9/HGSNAT Vector
(ASGCT 2025)
- "The findings highlight that CNS-directed gene replacement therapy with JLK-247 is a promising strategy to treat MPSIIIC. Further studies will assess postmortem histological and biomarker changes in these mice. GLP toxicology studies will assess the safety of JLK-247 to advance this experimental gene therapy approach towards clinical application."
Alzheimer's Disease • CNS Disorders • Dementia • Developmental Disorders • Gene Therapies • Hunter Syndrome • Lysosomal Storage Diseases • Metabolic Disorders • Psychiatry • Rare Diseases
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