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Genetic therapy for the nervous system

23 Mar 2011

Abstract excerpt

Genetic therapy is undergoing a renaissance with expansion of viral and synthetic vectors, use of oligonucleotides (RNA and DNA) and sequence-targeted regulatory molecules, as well as genetically modified cells, including induced pluripotent stem cells from the patients themselves. Several clinical trials for neurologic syndromes appear quite promising. This review covers genetic strategies to ameliorate...

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PMID: 21429918
DOI: 10.1093/hmg/ddr110

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