NewsMOLECULAR GENETICS AND METABOLISMThursday, March 5, 2026 · March 5, 2026
Novel promoters drive therapeutic transgene expression and evade transgene-specific immune responses in a mouse model of Pompe disease.
WHY IT MATTERS
Recent peer-reviewed research on Pompe disease that may be relevant for patients and caregivers.
Systemically administered gene therapy is under development for the treatment of Pompe disease, an inherited lysosomal storage disorder caused by acid alpha-glucosidase (GAA) deficiency. We evaluated recombinant AAV9 vectors expressing GAA driven by the minimal G6PC promoter (AAV9-G6PC.GAA) and by a...
Related conditions
Related news
Int J Circumpolar Health · -213 days ago
A population-based legacy study of myasthenia gravis in Iceland: insights from a small Arctic nation
Published in Int J Circumpolar Health. Hjaltason H et al.…
Ther Adv Rare Dis · -183 days ago
Could an outcome-based agreement be operationalized using real-world data from the Canadian Neuromuscular Disease Registry? Perspectives from an expert-led assessment in spinal muscular atrophy
Published in Ther Adv Rare Dis. Mitha A et al.…
Health Inf Sci Syst · -183 days ago
PhenoRareAI: Phenotype-based intelligent diagnosis for rare neuromuscular disorders of glycogen storage disease and spinal muscular atrophy
Published in Health Inf Sci Syst. Zhai W et al.…
J Allergy Clin Immunol Glob · -30 days ago
Emerging trends and research hot spots in inborn error of immunity: A bibliometric perspective
Published in J Allergy Clin Immunol Glob. Alblooshi H et al.…