Genetic correction of human induced pluripotent stem cells from patients with spinal muscular atrophy.

Corti, Stefania

Genetic correction of human induced pluripotent stem cells from patients with spinal muscular atrophy. [electronic resource] - Science translational medicine Dec 2012 - 165ra162 p. digital

Publication Type: Journal Article; Research Support, Non-U.S. Gov't

1946-6242

10.1126/scitranslmed.3004108 doi


Cell Transplantation
Gene Expression
Genetic Therapy
Genetic Vectors
Humans
Motor Neurons--metabolism
Muscular Atrophy, Spinal--genetics
Pluripotent Stem Cells--cytology
RNA Splicing
Survival of Motor Neuron 1 Protein--genetics