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GeneVision focuses on developing a gene therapy for inherited retinal diseases using dual adeno-associated virus vectors. This approach aims to treat conditions like Usher syndrome type Ib by enabling the delivery of larger genes that traditional methods cannot accommodate.
The GeneVision proof-of-concept project spins from a major discovery made by the principle investigator (PI) within the RetGeneTx ERC project demonstrating that dual AAV vectors expand AAV DNA transfer capacity in the retina thus allowing delivery of genes whose size cannot be packaged in single AAVs.
Thus, dual AAV vectors allow gene therapy of those inherited blinding conditions, like Usher syndrome type Ib (USHIB), Stargardt disease or other forms of retinitis pigmentosa, due to mutations in…
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