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Cell therapy using retinal progenitor cells shows therapeutic effect in a chemically-induced rotenone mouse model of Leber hereditary optic neuropathy
- Fiona C Mansergh
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Allele-specific Col1a1 silencing reduces mutant collagen in fibroblasts from Brtl mouse, a model for classical osteogenesis imperfecta
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- , Roberta Gioia
- & Antonella Forlino
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In vitro antisense therapeutics for a deep intronic mutation causing Neurofibromatosis type 2
- Elisabeth Castellanos
- , Imma Rosas
- & Eduard Serra
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