Revertant Phenomenon in DMD and LGMD2I and Its Therapeutic Implications: A Review of Study Under Mentorship of Terrence Partridge

por Lu, Qi Long
Publicado 2021

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

por Lu, Qi-long, Cirak, Sebahattin, Partridge, Terence
Publicado 2014

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

por Vannoy, Charles Harvey, Leroy, Victoria, Lu, Qi Long
Publicado 2018

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

por Blaeser, Anthony, Awano, Hiroyuki, Wu, Bo, Lu, Qi-Long
Publicado 2016

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

por Blaeser, Anthony, Awano, Hiroyuki, Lu, Pei, Lu, Qi-Long
Publicado 2018

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

por Cataldi, Marcela P., Lu, Peijuan, Blaeser, Anthony, Lu, Qi Long
Publicado 2018

Metabolomics Analysis of Skeletal Muscles from FKRP-Deficient Mice Indicates Improvement After Gene Replacement Therapy

por Vannoy, Charles Harvey, Leroy, Victoria, Broniowska, Katarzyna, Lu, Qi Long
Publicado 2019

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

por Vannoy, Charles Harvey, Xiao, Will, Lu, Peijuan, Xiao, Xiao, Lu, Qi Long
Publicado 2017

Ribitol alters multiple metabolic pathways of central carbon metabolism with enhanced glycolysis: A metabolomics and transcriptomics profiling of breast cancer

por Tucker, Jason Driver, Doddapaneni, Ravi, Lu, Pei Juan, Lu, Qi Long
Publicado 2022

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

por Cataldi, Marcela P., Blaeser, Anthony, Lu, Peijuan, Leroy, Victoria, Lu, Qi Long
Publicado 2019

Targeted Skipping of Human Dystrophin Exons in Transgenic Mouse Model Systemically for Antisense Drug Development

por Wu, Bo, Benrashid, Ehsan, Lu, Peijuan, Cloer, Caryn, Zillmer, Allen, Shaban, Mona, Lu, Qi Long
Publicado 2011

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

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

Ribitol dose-dependently enhances matriglycan expression and improves muscle function with prolonged life span in limb girdle muscular dystrophy 2I mouse model

por Wu, Bo, Drains, Morgan, Shah, Sapana N., Lu, Pei Juan, Leroy, Victoria, Killilee, Jessalyn, Rawls, Raegan, Tucker, Jason D., Blaeser, Anthony, Lu, Qi Long
Publicado 2022

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

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

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

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

Elusive sources of variability of dystrophin rescue by exon skipping

por Vila, Maria Candida, Klimek, Margaret Benny, Novak, James S., Rayavarapu, Sree, Uaesoontrachoon, Kitipong, Boehler, Jessica F., Fiorillo, Alyson A., Hogarth, Marshall W., Zhang, Aiping, Shaughnessy, Conner, Gordish-Dressman, Heather, Burki, Umar, Straub, Volker, Lu, Qi Long, Partridge, Terence A., Brown, Kristy J., Hathout, Yetrib, van den Anker, John, Hoffman, Eric P., Nagaraju, Kanneboyina
Publicado 2015