Thursday, January 24, 2013
This week in techniques
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Genetic correction of
spinal muscular atrophy (SMA) neurons with a nonviral, nonintegrating vector
mouse studies suggest genetic modification of patient-derived neurons with a
nonviral, nonintegrating vector could help treat SMA. In induced pluripotent
stem (iPS) cells derived from the fibroblasts of a patient with SMA, an
episomal vector was used to convert the gene encoding survival of motor neuron 2 centromeric
(SMN2) into a survival of motor neuron 1 telomeric
(SMN1)-like gene that
produced the full-length SMN protein. In a mouse model of SMA, spinal cord
engraftment of neurons derived from the vector-treated human iPS cells
decreased disease severity and increased lifespan and neuron engraftment
compared with engraftment of cells derived from untreated iPS cells. Next
steps could include testing the strategy in animal models of more advanced
Isis Pharmaceuticals Inc.
and Biogen Idec Inc. have ISIS-SMNRx, an antisense
oligonucleotide modulating the splicing of SMN2 pre-mRNA, in Phase
I/II testing to treat SMA.
Published online Jan. 24, 2013
Patent and licensing status
Corti, S. et al. Sci.
Transl. Med.; published online Dec. 19, 2012;
Contact: Giacomo P. Comi, University of Milan, Milan, Italy
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