Title:
COMPOSITIONS AND METHODS FOR PROMOTING LIVER REGENERATION BY GENE EDITING IN METABOLIC LIVER DISEASE
Document Type and Number:
WIPO Patent Application WO/2023/196908
Kind Code:
A3
Abstract:
Embodiments of the instant disclosure relate to novel gene editing vectors, compositions, and methods for editing a G6PC gene to treat glucose storage diseases (e.g., GSD Ia). In certain embodiments, vectors described herein comprise one or more CRISPR/Cas9 components to allow for integration of a G6PC transgene into a target gene locus in a subject in need thereof, thereby allowing for stable expression of a therapeutic protein (e.g., glucose-6-phosphatase) and reversal and/or treatment of disease symptoms in the subject.
Inventors:
KOEBERL DWIGHT (US)
KANG HYE RI (US)
ARNSON BENJAMIN (US)
CULLEN BRYAN (US)
KANG HYE RI (US)
ARNSON BENJAMIN (US)
CULLEN BRYAN (US)
Application Number:
PCT/US2023/065442
Publication Date:
November 16, 2023
Filing Date:
April 06, 2023
Export Citation:
Assignee:
UNIV DUKE (US)
International Classes:
C12N15/55; C12N15/11
Foreign References:
| US20100292085A1 | 2010-11-18 | |||
| US20190382798A1 | 2019-12-19 |
Attorney, Agent or Firm:
MCMULLEN, Michelle L. et al. (US)
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