Current avenues of gene therapy in Pompe disease

202315 citationsReviewgreen Open Access

Authors

Carmen Leon‐Astudillo · Epilepsy Foundation

Prasad D. Trivedi · Epilepsy Foundation

Ramon C. Sun · Epilepsy Foundation

Matthew S. Gentry · Epilepsy Foundation

David D. Fuller · Breathe California of Sacramento Emigrant Trails

Barry J. Byrne · University of Florida

Manuela Corti

Abstract

Gene therapy for the treatment of patients with Pompe disease is feasible and offers an opportunity to fully correct the principal pathology leading to cellular glycogen accumulation. Further work is needed to overcome the limitations related to vector production, immunologic reactions and redosing.

Topics & Keywords

Lysosomal Storage Disorders Research CRISPR and Genetic Engineering Virus-based gene therapy research

UN Sustainable Development Goals

Good health and well-being

Publication Details

Published in: Current Opinion in Neurology

Volume 36, Issue 5, pp. 464-473

DOI: 10.1097/wco.0000000000001187

Field-Weighted Citation Impact: 2.71