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US 2026/0209730 A1Published application
Compositions and methods of genome editing
AssigneeEpigenic Therapeutics Pte. Ltd.
InventorsChangyang ZHOU, Yidi SUN, Shaoshuai MAO, Wenbo PENG
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Abstract
This disclosure provides CRISPR/Cas9 based fusion molecules and guide RNAs for use in in vivo targeted reduction or elimination of VEGFA gene products. This disclosure also relates to formulations, methods of production and methods of use thereof.
Claims20
A construct of Formula III:
- wherein:
- one of A and B is a polynucleotide encoding DNMT3A;
- and the other of A and B is a polynucleotide encoding DNMT3L;
- CasN is a polynucleotide encoding a N-terminal portion of dCas9;
- CasC is a polynucleotide encoding a C-terminal portion of dCas9;
- E is 5′-(Am5-Bm6)n3-Kr-Dq-3′;
- K is a polynucleotide encoding KRAB;
- D is a polynucleotide encoding a modulator of gene expression;
- T comprises (a) a polynucleotide encoding an epitope capable of binding to an antibody or antigen binding fragment thereof, and further comprises (b) a polynucleotide encoding a self cleaving peptide at the 3′ end of the polynucleotide in (a), and at the 3′ end of the nucleotide encoding the self cleaving peptide in (b) further comprises (c) polynucleotide encoding an antibody or antigen binding fragment thereof that is capable of binding to the epitope;
- m1, m2, m3, m4, m5, and m6 are each independently 1;
- n2 is 0;
- n1 is 1 and n3 is 0;
- or n1 is 0 and n3 is 1;
- p is 1;
- q is 0;
- r is 1.
13 more elements
Dependencies are inferred from the claim text.