• Bertarelli Professor of Translational Medical Science
    Professor of Neurobiology
    Department of Neurobiology
    Harvard Medical School

    Lecture Title: "Gene Therapy for Deafness"

    READING LIST:

    1. György B, Nist-Lund C, Pan B, Asai Y, Karavitaki KD, Kleinstiver BP, Garcia SP, Zaborowski MP, Solanes P, Spataro S, Schneider BL, Joung JK, Géléoc GSG, Holt JR, Corey DP. Allele-specific gene editing prevents deafness in a model of dominant progressive hearing loss. Nat Med. 2019 Jul;25(7):1123-1130
    2. Ren Y, Landegger LD, Stankovic KM. Gene Therapy for Human Sensorineural Hearing Loss. Front Cell Neurosci. 2019 Jul 16;13:323.
    3. Omichi R, Shibata SB, Morton CC, Smith RJH. Gene therapy for hearing loss. Hum Mol Genet. 2019 Jun 22. pii: ddz129. doi: 10.1093/hmg/ddz129. [Epub ahead of print]
    4. Akil O, Lustig L. AAV-Mediated Gene Delivery to the Inner Ear. Methods Mol Biol. 2019;1950:271-282
    5. Al-Moyed H, Cepeda AP, Jung S, Moser T, Kügler S, Reisinger E. A dual-AAV approach restores fast exocytosis and partially rescues auditory function in deaf otoferlin knock-out mice. EMBO Mol Med. 2019 Jan;11(1). pii: e9396.
    6. György B, Meijer EJ, Ivanchenko MV, Tenneson K, Emond F, Hanlon KS, Indzhykulian AA, Volak A, Karavitaki KD, Tamvakologos PI, Vezina M, Berezovskii VK, Born RT, O'Brien M, Lafond JF, Arsenijevic Y, Kenna MA, Maguire CA, Corey DP. Gene Transfer with AAV9-PHP.B Rescues Hearing in a Mouse Model of Usher Syndrome 3A and Transduces Hair Cells in a Non-human Primate. Mol Ther Methods Clin Dev. 2018 Nov 20;13:1-13.
    7. Tao Y, Huang M, Shu Y, Ruprecht A, Wang H, Tang Y, Vandenberghe LH, Wang Q, Gao G, Kong WJ, Chen ZY. Delivery of Adeno-Associated Virus Vectors in Adult Mammalian Inner-Ear Cell Subtypes Without Auditory Dysfunction. Hum Gene Ther. 2018 Apr;29(4):492-506
    8. György B, Sage C, Indzhykulian AA, Scheffer DI, Brisson AR, Tan S, Wu X, Volak A, Mu D, Tamvakologos PI, Li Y, Fitzpatrick Z, Ericsson M, Breakefield XO, Corey DP, Maguire CA. Rescue of Hearing by Gene Delivery to Inner-Ear Hair Cells Using Exosome-Associated AAV. Mol Ther. 2017 Feb 1;25(2):379-391
    9. Askew C, Rochat C, Pan B, Asai Y, Ahmed H, Child E, Schneider BL, Aebischer P, Holt JR. Tmc gene therapy restores auditory function in deaf mice. Sci Transl Med. 2015 Jul 8;7(295):295ra108.
    10. Akil O, Seal RP, Burke K, Wang C, Alemi A, During M, Edwards RH, Lustig LR. Restoration of hearing in the VGLUT3 knockout mouse using virally mediated gene therapy. Neuron. 2012 Jul 26;75(2):283-93.