Gene therapy shows promise for treating Niemann-Pick disease type C1

NIH researchers demonstrated that gene therapy may be the best method for correcting the single faulty gene that causes Niemann-Pick disease, type C1.

Gene therapy shows promise for treating Niemann-Pick disease type C1

Promising gene replacement therapy moves forward

Clinical applications of gene therapy for rare diseases: A review - Papaioannou - 2023 - International Journal of Experimental Pathology - Wiley Online Library

IJMS, Free Full-Text

Frontiers Current and Future Prospects for Gene Therapy for Rare Genetic Diseases Affecting the Brain and Spinal Cord

IJMS, Free Full-Text

mRNA Treatment Rescues Niemann–Pick Disease Type C1 in Patient Fibroblasts

Gene therapy shows promise for treating Niemann-Pick disease type C1

Niemann-Pick Disease, Type C: Diagnosis, Management and Disease-Targeted Therapies in Development - ScienceDirect