NewsMOLECULAR THERAPY : THE JOURNAL OF THE AMERICAN SOCIETY OF GENE THERAPYSaturday, March 7, 2026 · March 7, 2026
AAV gene therapy for GBA1-related diseases.
WHY IT MATTERS
Recent peer-reviewed research on Gaucher disease that may be relevant for patients and caregivers.
Mutations in GBA1, the gene encoding glucocerebrosidase (GCase), are the most common risk factor for Parkinson's disease (PD). GBA-PD patients are a genetic subpopulation of PD carrying heterozygous mutations in GBA1. Additionally, bi-allelic mutations in GBA1 cause Gaucher disease (GD), a lysosomal...
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