Title: | Gene Correction of LGMD2A Patient-Specific iPSCs for the Development of Targeted Autologous Cell Therapy |
Journal : | Molecular therapy : the journal of the American Society of Gene Therapy |
Authors: | Selvaraj S, Author ; Dhoke NR ; Kiley J ; Mateos-Aierdi AJ ; Tungtur S ; Mondragon-Gonzalez R ; Killeen G ; Oliveira VKP ; Lopez de Munain A ; Perlingeiro RCR |
Material Type: | Article |
Publisher: | United States, 08/2019 |
Languages: | English |
Keywords : | Calpain-3-related limb-girdle muscular dystrophy R1 ; calpainopathy ; cell therapy ; limb girdle muscular dystrophy ; neuromuscular disease ; RESEARCH ; stem cells |
Keywords: | CAPN3;Calpain 3;autologous cell therapy;gene correction;genome editing;iPSCs;induced pluripotent stem cells;limb girdle muscular dystrophy type 2A;myogenic progenitors; |
Pubmed / DOI : | Pubmed : 31501033 / DOI : 10.1016/j.ymthe.2019.08.011 |
N° Profil MNM : | 2019092 |
Link for e-copy: | http://www.ncbi.nlm.nih.gov/pubmed/31501033 |
See also : |