Recombinant rabies viruses for gene therapy
AssigneeBeam Therapeutics Inc.
InventorsFei Ran, ChieYu Lin
Life of this patent
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Abstract
Provided herein are recombinant rabies virus genomes and recombinant rabies viruses and methods for their use in delivering a transgene into a target cell. Also provided are packaging systems and methods of using the packaging systems to produce recombinant rabies viruses.
Claims53
A recombinant rabies virus genome, comprising a nucleic acid encoding a therapeutic transgene, wherein:
- the genome lacks a G gene encoding for a rabies virus glycoprotein or a functional variant thereof;
- and/or
- the genome lacks an L gene encoding for a rabies virus polymerase or a functional variant thereof.
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A recombinant rabies virus particle, comprising:
- a rabies virus glycoprotein;
- and
- a recombinant rabies virus genome comprising a nucleic acid encoding a therapeutic transgene, wherein:
- the genome lacks a G gene encoding for a rabies virus glycoprotein or a functional variant thereof;
- and/or
- the genome lacks an L gene encoding for a rabies virus polymerase or a functional variant thereof.
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A method for expressing a nucleobase editor in a target cell, comprising transducing a target cell with a recombinant rabies virus particle, wherein the recombinant virus particle comprises:
- a rabies virus glycoprotein or variant thereof;
- and
- a recombinant rabies virus genome comprising a nucleic acid encoding a nucleobase editor comprising a polynucleotide programmable nucleotide binding domain and a nucleobase editing domain, wherein:
- the genome lacks a G gene encoding for a rabies virus glycoprotein or a functional variant thereof;
- and/or
- the genome lacks an L gene encoding for a rabies virus polymerase or a functional variant thereof.
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A packaging system for the recombinant preparation of a rabies virus particle, wherein the packaging system comprises:
- an N gene encoding for a rabies virus nucleoprotein or a functional variant thereof;
- a P gene encoding for a rabies virus phosphoprotein or a functional variant thereof;
- an L gene encoding for a rabies virus polymerase or a functional variant thereof;
- and
- a recombinant rabies virus genome, wherein:
- the genome lacks a G gene encoding for a rabies virus glycoprotein or a functional variant thereof;
- and/or
- the genome lacks an L gene encoding for a rabies virus polymerase or a functional variant thereof.
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A recombinant rabies virus particle packaging cell, comprising:
- an N gene encoding for a rabies virus nucleoprotein or a functional variant thereof;
- a P gene encoding for a rabies virus phosphoprotein or a functional variant thereof;
- and
- an L gene encoding for a rabies virus polymerase or a functional variant thereof.
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Dependencies are inferred from the claim text.