Screening data from 19 patients with late‐onset Pompe disease for a phase I clinical trial of AAV8 vector‐mediated gene therapy

Authors: William B. Hannah, Laura E. Case, Edward C. Smith, Crista Walters, Deeksha Bali, Priya S. Kishnani, Dwight D. Koeberl

Source: JIMD Reports, Vol 64, Iss 5, Pp 393-400 (2023)

Subject Terms: adeno‐associated virus, gene therapy, Pompe disease, Diseases of the endocrine glands. Clinical endocrinology, RC648-665, Genetics, QH426-470

File Description: electronic resource

Relation: https://doaj.org/toc/2192-8312

Access URL: https://doaj.org/article/d8832d4d282b4cac8148d3645a6ca744