Human Gene Therapy | Mary Ann Liebert, Inc., publishers

Human Gene Therapy | Mary Ann Liebert, Inc., publishers

Human Gene Therapy

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Read and share articles from this Special Section on Neuromuscular Disease Gene Therapy through September 30, 2020.

Access the full issue here.

Laying the Foundation for Neuromuscular Disease Gene Therapy Dongsheng Duan  Read Now

Adeno-Associated Virus-Induced Dorsal Root Ganglion Pathology Juliette Hordeaux, Elizabeth L. Buza, Cecilia Dyer, Tamara Goode, Thomas W. Mitchell, Laura Richman, Nathan Denton, Christian Hinderer, Nathan Katz, Ralf Schmid, Rod Miller, Gourav R. Choudhury, Makoto Horiuchi, Kalyani Nambiar, Hanying Yan, Mingyao Li, and James M. Wilson  Read Now

Stress-Induced Mouse Model of the Cardiac Manifestations of Friedreich's Ataxia Corrected by AAV-mediated Gene Therapy Christiana O. Salami, Katie Jackson, Clarisse Jose, Laith Alyass, Georges-Ibrahim Cisse, Bishnu P. De, Katie M. Stiles, Maria J. Chiuchiolo, Dolan Sondhi, Ronald G. Crystal, and Stephen M. Kaminsky  Read Now

Intralingual Administration of AAVrh10-miRSOD1 Improves Respiratory But Not Swallowing Function in a Superoxide Dismutase-1 Mouse Model of Amyotrophic Lateral Sclerosis Lori A. Lind, Ellyn M. Andel, Angela L. McCall, Justin S. Dhindsa, Katherine A. Johnson, Olivia E. Stricklin, Christian Mueller, Mai K. ElMallah, Teresa E. Lever, and Nicole L. Nichols  Read Now

Defining Transcription Regulatory Elements in the Human Frataxin Gene: Implications for Gene Therapy Jixue Li, Yanjie Li, Jun Wang, Trevor J. Gonzalez, Aravind Asokan, Jill S. Napierala, and Marek Napierala  Read Now