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.
Claims12
A method for treating or preventing a disorder associated with expression of a SCD-associated polynucleotide sequence in a subject, the method comprising (a) altering a target SCD-associated polynucleotide sequence in a cell ex vivo by contacting the SCD-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 SCD-associated polynucleotide sequence, wherein the target SCD-associated polynucleotide sequence is cleaved, and (b) introducing the cell into the subject, thereby treating or preventing a disorder associated with expression of the SCD-associated polynucleotide sequence.
A method for treating or preventing a disorder associated with expression of a SCD-associated polynucleotide sequence in a subject, the method comprising (a) altering a target SCD-associated polynucleotide sequence in a cell ex vivo by contacting the SCD-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 SCD-associated polynucleotide sequence, wherein the target SCD-associated polynucleotide sequence is cleaved;
- (b) contacting the cleaved target SCD-associated polynucleotide sequences with an exogenously introduced DNA repair template to initiate homology-directed repair of the target SCD-associated polynucleotide;
- and (c) introducing the cell into the subject, thereby treating or preventing a disorder associated with expression of the SCD-associated polynucleotide sequence.
A method for treating or preventing a disorder associated with expression of an HBB gene sequence in a subject, the method comprising (a) altering a target HBB gene sequence in a cell ex vivo by contacting the HBB 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 HBB gene sequence, wherein the target HBB gene sequence is cleaved, and (b) introducing the cell into the subject, thereby treating or preventing a disorder associated with expression of the HBB gene sequence.
Dependencies are inferred from the claim text.