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US 2022/0023444 A1Published application
Gene Editing-Based Method of Attenuating the Beta-Amyloid Pathway
AssigneeWisconsin Alumni Research Foundation
InventorsSubhojit Roy, Jichao Sun
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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.
Claims12
A genetic construct comprising,
- i. a sequence encoding for a Cas9 nuclease;
- and
- ii. a sequence encoding a gRNA specific to amyloid precursor protein (APP),
- wherein a Cas9 nuclease/gRNA ribonucleoprotein directs cleavage of an APP gene to provide a C-terminal truncated APP having a length of 659, 670, 676, or 686 amino acids relative to SEQ ID NO:
- 12 (human) or SEQ ID NO:
- 14 (mouse).
3 more elements
A composition comprising a first AAV vector comprising a sequence encoding for a Cas9 nuclease and a second AAV vector comprising a sequence encoding a gRNA specific to amyloid precursor protein (APP), wherein a Cas9 nuclease/gRNA ribonucleoprotein directs cleavage of an APP gene to provide C-terminal truncated APP having a length of 659, 670, 676, or 686 amino acids relative to SEQ ID NO:
- 12 (human) or SEQ ID NO:
- 14 (mouse).
Dependencies are inferred from the claim text.