A group of scientists from The University of Milan, Italy described another variation of stem cell therapy as a potential treatment for spinal muscular atrophy (SMA). The paper published in Science Translational Medicine suggests that genetically corrected pluripotent stem cells may provide a source of motor neurons for therapeutic transplantation for SMA.
The team generated induced pluripotent stem cells (iPSC) from SMA patients using nonviral, nonintegrating episomal vectors and used a targeted gene correction approach based on single-stranded oligonucleotides to convert the survival motor neuron 2 (SMN2) gene into an SMN1-like gene. Corrected iPSC lines contained no exogenous sequences. Motor neurons formed by differentiation of uncorrected SMA-iPSC reproduced disease-specific features. These features were ameliorated in motor neurons derived from genetically corrected SMA-iPSC. The different gene splicing profile in SMA-iPSC motor neurons was rescued after genetic correction. The transplantation of corrected motor neurons derived from SMA-iPSC into an SMA mouse model extended the life span of the animals and improved the disease phenotype.
The team generated induced pluripotent stem cells (iPSC) from SMA patients using nonviral, nonintegrating episomal vectors and used a targeted gene correction approach based on single-stranded oligonucleotides to convert the survival motor neuron 2 (SMN2) gene into an SMN1-like gene. Corrected iPSC lines contained no exogenous sequences. Motor neurons formed by differentiation of uncorrected SMA-iPSC reproduced disease-specific features. These features were ameliorated in motor neurons derived from genetically corrected SMA-iPSC. The different gene splicing profile in SMA-iPSC motor neurons was rescued after genetic correction. The transplantation of corrected motor neurons derived from SMA-iPSC into an SMA mouse model extended the life span of the animals and improved the disease phenotype.
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