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US 2026/0097136 A1Published application
Gene editing-based method of attenuating the beta-amyloid pathway
AssigneeWisconsin Alumni Research Foundation
InventorsSubhojit Roy, Jichao Sun
Life of this patent
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Abstract
Described herein are CRISPR/Cas9 constructs designed for the C-terminal truncation of human amyloid precursor protein (APP) as well as methods of making and using such a construct.
Claims10
A method of treating or pre-treating a patient having or at risk of forming amyloid plaques composed of amyloid beta (Aβ) peptides, wherein the method comprises the steps of
- (a) obtaining a gene-editing construct specific for the amyloid precursor protein (APP), wherein the construct facilitates truncation of the APP C-terminus when combined with a Cas9 nuclease, and
- (b) delivering the construct and a construct encoding the Cas9 nuclease to a patient in need of AD therapy, wherein the APP molecule is truncated and production of Aβ peptides is decreased in the patient's brain.
A method of editing endogenous amyloid precursor protein (APP), the method comprising the steps of
- (a) obtaining a gene-editing construct specific for APP, wherein the construct facilitates truncation of the APP C-terminus when combined with a Cas9 nuclease, and
- (b) delivering the construct and nuclease to a patient in need of AD therapy, wherein the APP molecule is truncated and production of Aβ peptides is decreased in the patient's brain.
Dependencies are inferred from the claim text.