Last Updated: 09/26/2026
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Found 15 publications
Optimal murine CD4+ T cell priming by mRNA-lipid nanoparticle vaccines requires endogenous antigen processing.
Journal: Nature communications
Published: August 27, 2025
AAV-microutrophin gene therapy confers durable cardioprotection against pharmacologic and exercise-induced injury in the mdx mouse.
Journal: Molecular therapy : the journal of the American Society of Gene Therapy
Published: January 14, 2025
Confronting complexity in the design and conduct of high-risk rare disease clinical trials.
Journal: Molecular therapy : the journal of the American Society of Gene Therapy
Published: March 31, 2023
Challenges at the Crossroads: Myopathy Trials in 2020 Hindsight.
Journal: Molecular therapy : the journal of the American Society of Gene Therapy
Published: January 21, 2021
Broader Implications of Progressive Liver Dysfunction and Lethal Sepsis in Two Boys following Systemic High-Dose AAV.
Journal: Molecular therapy : the journal of the American Society of Gene Therapy
Published: July 26, 2020
Non-immunogenic utrophin gene therapy for the treatment of muscular dystrophy animal models.
Journal: Nature medicine
Published: November 01, 2018
Mechanism of Deletion Removing All Dystrophin Exons in a Canine Model for DMD Implicates Concerted Evolution of X Chromosome Pseudogenes.
Journal: Molecular therapy. Methods & clinical development
Published: October 27, 2016
Suite of clinically relevant functional assays to address therapeutic efficacy and disease mechanism in the dystrophic mdx mouse.
Journal: Journal of applied physiology (Bethesda, Md. : 1985)
Published: September 02, 2016
Translational data from adeno-associated virus-mediated gene therapy of hemophilia B in dogs.
Journal: Human gene therapy. Clinical development
Published: February 13, 2015
Perspectives on best practices for gene therapy programs.
Journal: Human gene therapy
Published: February 06, 2015
Diaphragm remodeling and compensatory respiratory mechanics in a canine model of Duchenne muscular dystrophy.
Journal: Journal of applied physiology (Bethesda, Md. : 1985)
Published: January 11, 2014
Cardiac surgical delivery of the sarcoplasmic reticulum calcium ATPase rescues myocytes in ischemic heart failure.
Journal: The Annals of thoracic surgery
Published: December 11, 2012
Last Updated: 09/26/2026