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US 2023/0405116 A1Published application
Vectors, systems and methods for eukaryotic gene editing
AssigneeWake Forest University Health Sciences
InventorsBaisong LU, Anthony ATALA
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Abstract
Provided herein are compositions and methods for editing the genome of a eukaryotic cell.
Claims31
A mammalian expression plasmid comprising a eukaryotic promoter operably linked to a non-viral nucleic acid sequence, wherein the non-viral nucleic acid sequence comprises:
- (i) a nucleic acid sequence encoding an adenosine base pair editor (ABE), wherein the ABE is a fusion protein comprising an adenosine deaminase and a catalytically impaired CRISPR-associated endonuclease;
- and
- (ii) a guide RNA (gRNA) coding sequence,
- wherein the gRNA coding sequence comprises at least one aptamer coding sequence.
1 more element
A lentivirus-like particle comprising:
- a) a fusion protein comprising a nucleocapsid (NC) protein or a matrix (MA) protein wherein the NC protein or MA protein comprises at least one non-viral aptamer binding protein (ABP);
- and
- b) a ribonucleotide protein (RNP) complex comprising:
- (i) an adenine base editor (ABE), wherein the ABE is a fusion polypeptide comprising an adenine base editor and a catalytically impaired CRISPR-associated endonuclease;
- and (ii) a gRNA,
- wherein the lentivirus-like particle does not comprise a functional integrase protein.
3 more elements
Dependencies are inferred from the claim text.