IP Library › Patent Application 19387161
Patent Application
App. No. 19/387,161

GENE EDITING-BASED METHOD OF ATTENUATING THE BETA-AMYLOID PATHWAY

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Quick Facts
Patent No.
US None
App. No.
19/387,161
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.

Claims (15)

1 . 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.

2 . The method of claim 1 , wherein the truncation of the APP C-terminus occurs at an APP residue selected from the group consisting of 659, 670, 676, and 686.

3 . The method of claim 1 , wherein the gene-editing construct comprises a gRNA sequence selected from the group consisting of SEQ ID NOs:1-10.

4 . The method of claim 1 , wherein the construct and the nuclease are delivered in a composition comprising an adeno-associated viral vector and a nanocarrier delivery vehicle.

5 . The method of claim 4 , wherein the composition is delivered intravenously or intrathecally.

edit endogenous amyloid precursor protein (APP).

6 . 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.

7 . The method of claim 6 , wherein the truncation of the APP C-terminus occurs at an APP residue selected from the group consisting of 659, 670, 676, and 686.

8 . The method of claim 6 , wherein the gene-editing construct comprises a gRNA sequence selected from the group consisting of SEQ ID NO:1-10.

9 . The method of claim 6 , wherein the construct and the nuclease are delivered in a composition comprising an adeno-associated viral vector and a nanocarrier delivery vehicle.

10 . The method of claim 6 , wherein the composition is delivered intravenously or intrathecally.