Gene editing for HIV gene therapy
First Claim
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1. An isolated genetically modified cell that will not sustain HIV infection, wherein the genetic modifications comprise:
- (i) modification of an endogenous Trim5α
gene to increase its activity against HIV;
(ii) modification of an endogenous viral host factor gene by mutations, which mutations decrease its ability to support HIV infection; and
(iii) insertion of an exogenous transgene encoding a Trim5α
-cyclophilin A peptide (Trim5α
-Cyp) fusion protein into an endogenous safe harbor locus wherein the genetically modified cell will not sustain HIV infection.
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Abstract
The present disclosure is in the field of genome engineering, particularly targeted integration of anti-HIV transgenes into the genome of a cell for the treatment and/or prevention of HIV.
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Citations
14 Claims
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1. An isolated genetically modified cell that will not sustain HIV infection, wherein the genetic modifications comprise:
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(i) modification of an endogenous Trim5α
gene to increase its activity against HIV;(ii) modification of an endogenous viral host factor gene by mutations, which mutations decrease its ability to support HIV infection; and (iii) insertion of an exogenous transgene encoding a Trim5α
-cyclophilin A peptide (Trim5α
-Cyp) fusion protein into an endogenous safe harbor locus wherein the genetically modified cell will not sustain HIV infection. - View Dependent Claims (2, 3, 4, 5, 6, 7, 8, 9, 10, 11, 12, 13, 14)
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Specification