M. Eggers

Muscle‐directed gene therapy corrects Pompe disease and uncovers species‐specific GAA immunogenicity

F. Mavilio, M. Lawlor, Jianyong Huang, 2021, EMBO molecular medicine.

Lack of Toxicity in Nonhuman Primates Receiving Clinically Relevant Doses of an AAV9.U7snRNA Vector Designed to Induce DMD Exon 2 Skipping

K. Flanigan, L. Gushchina, M. Waldrop, 2021, Human gene therapy.

THE NEUROBIOLOGY OF PERIPHERALNERVE REGENERATION

D. D. Palande, M. Wiberg, N. Lago, 2009 .