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Michelle Mc Clements, Peter Charbel Issa, Veronique Blouin, Robert Mac Laren

Public reference bibliography and cataloged PDF materials authored or published by Michelle Mc Clements, Peter Charbel Issa, Veronique Blouin, Robert Mac Laren.

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A Fragmented Adeno-Associated Viral Dual Vector Strategy for Treatment of Diseases Caused by Mutations in Large Genes Leads to Expression of Hybrid Transcripts

Year: 2016 • Source: hal.science • Listed PDF

OBJECTIVE: Dual vector AAV systems are being utilised to enable gene therapy for disorders in which the disease gene is too large to fit into a single capsid. Fragmented adeno-associated viral (fAAV) vectors containing s...