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Title:
AAV-MEDIATED GENE TRANSFER FOR RETINOPATHY
Document Type and Number:
WIPO Patent Application WO/2021/252989
Kind Code:
A3
Abstract:
The present invention relates generally to gene therapy for treating ailments that can affect vision such as retinal degeneration, retinal dystrophy, macular degeneration, macular dystrophy, ischemic retinopathies, and glaucoma. Embodiments include systems and treatments that use AAV-mediated gene therapy or non AAV- mediated DNA, mRNA, or protein therapy to target all retinal cells. An AAV virion can be introduced (e.g., via intravitreal or subretinal injection) into an eye of an individual, or systemically, to express a heterologous gene product such as BMI1 protein (B lymphoma Mo-MLV insertion region 1 homolog).

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Inventors:
HEMA LAKSHMI RAMKUMAR (US)
Application Number:
PCT/US2021/037137
Publication Date:
February 03, 2022
Filing Date:
June 12, 2021
Export Citation:
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Assignee:
OCULOGENEX INC (US)
International Classes:
A61K48/00; C12N15/85; C12N15/86
Foreign References:
US20110123509A12011-05-26
US20180161405A12018-06-14
Other References:
BRUEWER ASHLEE R., MOWAT FREYA M., BARTOE JOSHUA T., BOYE SANFORD L., HAUSWIRTH WILLIAM W., PETERSEN-JONES SIMON M.: "Evaluation of Lateral Spread of Transgene Expression following Subretinal AAV–Mediated Gene Delivery in Dogs", PLOS ONE, vol. 8, no. 4, 3 April 2013 (2013-04-03), pages e60218, XP055903268, DOI: 10.1371/journal.pone.0060218
Attorney, Agent or Firm:
WEINSTEIN, Peter D. et al. (US)
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