Homologous Recombination Mediates Functional Recovery of Dysferlin Deficiency following AAV5 Gene Transfer

mdx5cv Mice Manifest More Severe Muscle Dysfunction and Diaphragm Force Deficits than Do mdx Mice

(2011) Nicholas Beastrom et al.   AMERICAN JOURNAL OF PATHOLOGY

Early Treatment With Lisinopril and Spironolactone Preserves Cardiac and Skeletal Muscle in Duchenne Muscular Dystrophy Mice

(2011) Jill A. Rafael-Fortney et al.   CIRCULATION

Unmasking Potential Intracellular Roles For Dysferlin through Improved Immunolabeling Methods

(2011) Joseph A. Roche et al.   JOURNAL OF HISTOCHEMISTRY & CYTOCHEMISTRY

Sustained alpha-sarcoglycan gene expression after gene transfer in limb-girdle muscular dystrophy, type 2D

(2010) Jerry R. Mendell et al.   ANNALS OF NEUROLOGY

Efficient recovery of dysferlin deficiency by dual adeno-associated vector-mediated gene transfer

(2010) William Lostal et al.   HUMAN MOLECULAR GENETICS

Novel diagnostic features of dysferlinopathies

(2010) Xiomara Q. Rosales et al.   MUSCLE & NERVE

Dystrophin Immunity in Duchenne's Muscular Dystrophy

(2010) Jerry R. Mendell et al.   NEW ENGLAND JOURNAL OF MEDICINE

A Naturally Occurring Human Minidysferlin Protein Repairs Sarcolemmal Lesions in a Mouse Model of Dysferlinopathy

(2010) M. Krahn et al.   Science Translational Medicine

Limb-girdle muscular dystrophy type 2D gene therapy restores α-sarcoglycan and associated proteins

(2009) Jerry R. Mendell et al.   ANNALS OF NEUROLOGY

Evidence for the Failure of Adeno-associated Virus Serotype 5 to Package a Viral Genome ≥8.2 kb

(2009) Yi Lai et al.   MOLECULAR THERAPY

Characterization of Genome Integrity for Oversized Recombinant AAV Vector

(2009) Biao Dong et al.   MOLECULAR THERAPY

Persistent Expression of FLAG-tagged Micro dystrophin in Nonhuman Primates Following Intramuscular and Vascular Delivery

(2009) Louise R Rodino-Klapac et al.   MOLECULAR THERAPY

Effect of Genome Size on AAV Vector Packaging

(2009) Zhijian Wu et al.   MOLECULAR THERAPY

Overexpression of Galgt2 in skeletal muscle prevents injury resulting from eccentric contractions in both mdx and wild-type mice

(2008) Paul T. Martin et al.   AMERICAN JOURNAL OF PHYSIOLOGY-CELL PHYSIOLOGY

Serotype-dependent packaging of large genes in adeno-associated viral vectors results in effective gene delivery in mice

(2008) Mariacarmela Allocca et al.   JOURNAL OF CLINICAL INVESTIGATION

Late onset in dysferlinopathy widens the clinical spectrum

(2008) L. Klinge et al.   NEUROMUSCULAR DISORDERS

Complete fatty degeneration of lumbar erector spinae muscles caused by a primary dysferlinopathy

(2007) P. Seror et al.   MUSCLE & NERVE