Improved efficacy of FKRP AAV gene therapy by combination with ribitol treatment for LGMD2I
Marcela P. Cataldi, Charles H. Vannoy, Anthony Blaeser, Jason D. Tucker, Victoria Leroy, Raegan Rawls, Jessalyn Killilee, Molly C. Holbrook, Qi Long Lu
Topics & Concepts
Genetic enhancementTransgeneCombination therapyMedicineGeneBiologyInternal medicineCancer researchPharmacologyBiochemistryMuscle Physiology and DisordersVirus-based gene therapy researchSirtuins and Resveratrol in Medicine