What Can We Learn From Clinical Trials of Exon Skipping for DMD?

by Qi-long Lu, Sebahattin Cirak, Terence Partridge
Published 2014-01-01

Dose-Dependent Effects of FKRP Gene-Replacement Therapy on Functional Rescue and Longevity in Dystrophic Mice

by Charles Harvey Vannoy, Victoria Leroy, Qi Long Lu
Published 2018-12-01

Distinct expression of functionally glycosylated alpha-dystroglycan in muscle and non-muscle tissues of FKRP mutant mice.

by Anthony Blaeser, Hiroyuki Awano, Pei Lu, Qi-Long Lu
Published 2018-01-01

Progressive Dystrophic Pathology in Diaphragm and Impairment of Cardiac Function in FKRP P448L Mutant Mice.

by Anthony Blaeser, Hiroyuki Awano, Bo Wu, Qi-Long Lu
Published 2016-01-01

Ribitol restores functionally glycosylated α-dystroglycan and improves muscle function in dystrophic FKRP-mutant mice

by Marcela P. Cataldi, Peijuan Lu, Anthony Blaeser, Qi Long Lu
Published 2018-08-01

Efficacy of Gene Therapy Is Dependent on Disease Progression in Dystrophic Mice with Mutations in the FKRP Gene

by Charles Harvey Vannoy, Will Xiao, Peijuan Lu, Xiao Xiao, Qi Long Lu
Published 2017-06-01

ISPD Overexpression Enhances Ribitol-Induced Glycosylation of α-Dystroglycan in Dystrophic FKRP Mutant Mice

by Marcela P. Cataldi, Anthony Blaeser, Peijuan Lu, Victoria Leroy, Qi Long Lu
Published 2020-06-01

Targeted skipping of human dystrophin exons in transgenic mouse model systemically for antisense drug development.

by Bo Wu, Ehsan Benrashid, Peijuan Lu, Caryn Cloer, Allen Zillmer, Mona Shaban, Qi Long Lu
Published 2011-01-01

Skeletal, cardiac, and respiratory muscle function and histopathology in the P448Lneo− mouse model of FKRP-deficient muscular dystrophy

by Qing Yu, Melissa Morales, Ning Li, Alexander G. Fritz, Ren Ruobing, Anthony Blaeser, Ershia Francois, Qi-Long Lu, Kanneboyina Nagaraju, Christopher F. Spurney
Published 2018-04-01

Efficient engraftment of pluripotent stem cell-derived myogenic progenitors in a novel immunodeficient mouse model of limb girdle muscular dystrophy 2I

by Karim Azzag, Carolina Ortiz-Cordero, Nelio A. J. Oliveira, Alessandro Magli, Sridhar Selvaraj, Sudheer Tungtur, Weston Upchurch, Paul A. Iaizzo, Qi Long Lu, Rita C. R. Perlingeiro
Published 2020-04-01

A universal gene correction approach for FKRP-associated dystroglycanopathies to enable autologous cell therapy

by Neha R. Dhoke, Hyunkee Kim, Sridhar Selvaraj, Karim Azzag, Haowen Zhou, Nelio A.J. Oliveira, Sudheer Tungtur, Carolina Ortiz-Cordero, James Kiley, Qi Long Lu, Anne G. Bang, Rita C.R. Perlingeiro
Published 2021-07-01