Adeno-associated Virus (AAV) Dual Vector Strategies for Gene Therapy Encoding Large Transgenes.

Yale J Biol Med · 2017 · PMC5733846 · PMID 29259525

Europe PMC

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highly significantno p-value reported

Being able to provide some relief to the blinding aspect of the condition would be considered highly significant for these individuals that will become both deaf and blind in the absence of any treatment.

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