THERAPEUTIC USES OF GENOME EDITING WITH CRISPR/Cas SYSTEMS
AssigneesPresident and Fellows of Harvard College, Children's Medical Center Corporation
InventorsKiran Musunuru, Chad A. Cowan, Derrick J. Rossi
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Abstract
Disclosed herein are methods, compositions, and kits for high efficiency, site-specific genomic editing of cells for treating or preventing genetic blood disorders.
Claims10
A method for altering a target severe combined immune deficiency disease (SCID)-associated polynucleotide sequence in a cell ex vivo comprising contacting the SCID-associated polynucleotide sequence with a clustered regularly interspaced short palindromic repeats-associated (Cas) protein and from one to two ribonucleic acids, wherein the ribonucleic acids direct Cas protein to and hybridize to a target motif of the target SCID-associated polynucleotide sequence, wherein the target SCID-associated polynucleotide sequence is cleaved.
A method for altering a target sever combined immune deficiency disease (SCID)-associated polynucleotide sequence in a cell ex vivo comprising (a) contacting the SCID-associated polynucleotide sequence with a clustered regularly interspaced short palindromic repeats-associated (Cas) protein and from one to two ribonucleic acids, wherein the ribonucleic acids direct Cas protein to and hybridize to a target motif of the target SCID-associated polynucleotide sequence, wherein the target SCID-associated polynucleotide sequence is cleaved, and (b) contacting the cleaved target SCID-associated polynucleotide sequences with an exogenously introduced DNA repair template to initiate homology-directed repair of the target SCID-associated polynucleotide.
A method for altering a target IL2RG gene sequence in a cell ex vivo comprising (a) contacting the IL2RG gene sequence with a clustered regularly interspaced short palindromic repeats-associated (Cas) protein and from one to two ribonucleic acids, wherein the ribonucleic acids direct Cas protein to and hybridize to a target motif of the target IL2RG gene sequence, wherein the target IL2RG gene sequence is cleaved, and (b) contacting the cleaved target IL2RG gene sequences with an exogenously introduced DNA repair template to initiate homology-directed repair of the target IL2RG gene sequence.
Dependencies are inferred from the claim text.