LENTIVIRAL VECTOR TECHNOLOGY FOR INNER EAR GENE THERAPY
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United States of America Patent
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Sep 12, 2024
app pub date -
Dec 3, 2021
filing date -
Dec 3, 2020
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Abstract
The present invention relates to the field of gene therapy for the treatment of sensorineural hearing loss. In particular, the invention discloses a composition comprising a 3rd generation lentiviral vector pseudotyped with a viral envelope glycoprotein capable of binding to a receptor expressed in a cell of the inner ear for use in the treatment or prevention of sensorineural hearing loss, wherein said composition has a viral titer of at least 107 TU/mL and is administered to the inner ear of a subject suffering from or in danger to undergo sensorineural hearing loss. The viral envelope glycoprotein is capable of binding to a receptor selected from the group consisting of the LDL-receptor and LDL-R family members, the SLC1 A5-receptor, the Pit1/2-receptor and the PIRYV-G-receptor. Preferably, the viral glycoprotein is MARAV-G, COCV-G, VSV-G, VSV-G ts or PIRYV-G, most preferably, MARAV-G. The lentiviral vector further comprises a cargo sequence comprising, e.g. a protein-coding gene, a miRNA, an shRNA, a lncRNA or an sgRNA, wherein expression of said cargo sequence in the cell of the inner ear is capable of reducing, eliminating or preventing at least a symptom of sensorineural hearing loss in the subject. The invention further discloses a method for treating sensorineural hearing loss in a subject in need thereof.
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