A universal gene correction approach for FKRP-associated dystroglycanopathies to enable autologous cell therapy
Neha R. Dhoke, Hyunkee Kim, Sridhar Selvaraj, Karim Azzag, Haowen Zhou, Nélio Alessandro de Jesus Oliveira, Sudheer Tungtur, Carolina Ortiz‐Cordero, James P. Kiley, Qi Long Lu, Anne G. Bang, Rita C. R. Perlingeiro
Abstract
-NSG mouse model gives rise to myofiber and satellite cell engraftment and, importantly, restoration of α-DG functional glycosylation in vivo. These findings suggest the potential feasibility of using CRISPR-Cas9 technology in combination with patient-specific iPS cells for the future development of autologous cell transplantation for FKRP-associated MDs.
Topics & Concepts
BiologyGenetic enhancementTransplantationGlycosylationInduced pluripotent stem cellDystroglycanMuscular dystrophyGenome editingProgenitor cellCRISPRCancer researchGeneCell biologyComputational biologyGeneticsStem cellCellMedicineEmbryonic stem cellInternal medicineLamininCRISPR and Genetic EngineeringMuscle Physiology and DisordersVirus-based gene therapy research