Advancing Gene-Targeted Therapies for Central Nervous System Disorders Proceedings of a Workshop (2019) / Chapter Skim
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Appendix A: References
Appendix A: References
Pages 59-64
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2008. Reversal of blindness in animal models of leber congenital amaurosis using optimized AAV2-mediated gene transfer.
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2015. Lentiviral gene therapy using cellular promoters cures Type 1 Gaucher disease in mice.
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2010. Antisense correction of SMN2 splicing in the CNS rescues necrosis in a type III SMA mouse model.
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C De Vivo, Muscle Study Group, and Pediatric Neuro uscularm Clinical Research Network for Spinal Muscular Atrophy.
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2014. Characterizing bacterial gene circuit dynamics with optically programmed gene expression signals.
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2013. Aberrant splicing of HTT generates the pathogenic exon 1 protein in Huntington disease.
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