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.
Claims25
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A method for altering a target beta thalassemia-associated polynucleotide sequence in a cell comprising contacting the beta thalassemia-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 beta thalassemia-associated polynucleotide sequence, wherein the target beta thalassemia-associated polynucleotide sequence is cleaved, and wherein the efficiency of alteration is from about 50% to about 80%.
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