Joseph W. Foley

The ΔF508 CFTR defect: molecular mechanism of suppressor mutation V510D and the contribution of transmembrane helix unraveling

P. Manavalan, R. Scheule, G. Hurlbut, 2020, bioRxiv.

Iminosugar-based inhibitors of glucosylceramide synthase prolong survival but paradoxically increase brain glucosylceramide levels in Niemann-Pick C mice.

R. Scheule, K. Ashe, Christopher G. F. Cooper, 2012, Molecular genetics and metabolism.

Systemic Insulin-like growth factor-1 reverses hypoalgesia and improves mobility in a mouse model of diabetic peripheral neuropathy.

R. Scheule, Q. Chu, R. Moreland, 2008, Molecular therapy : the journal of the American Society of Gene Therapy.

Evaluation of systemic follistatin as an adjuvant to stimulate muscle repair and improve motor function in Pompe mice.

R. Scheule, P. Finn, Seng H. Cheng, 2010, Molecular therapy : the journal of the American Society of Gene Therapy.

Ability of adeno-associated virus serotype 8-mediated hepatic expression of acid alpha-glucosidase to correct the biochemical and motor function deficits of presymptomatic and symptomatic Pompe mice.

T. Taksir, R. Scheule, L. Shihabuddin, 2008, Human gene therapy.

Preexisting immunity and low expression in primates highlight translational challenges for liver-directed AAV8-mediated gene therapy.

R. Scheule, G. Hurlbut, E. Meyers, 2010, Molecular therapy : the journal of the American Society of Gene Therapy.

Combination brain and systemic injections of AAV provide maximal functional and survival benefits in the Niemann-Pick mouse

T. Taksir, L. Shihabuddin, Seng H. Cheng, 2007, Proceedings of the National Academy of Sciences.