Back to results
US 12,735,711 B2Granted
Targeting the human CCR5 locus as a safe harbor for the expression of therapeutic proteins
AssigneeThe Board of Trustees of the Leland Stanford Junior University
InventorsNatalia Gomez-Ospina, Matthew H. Porteus, Samantha Glynne Scharenberg, Alvaro Amorin
Life of this patent
Estimating the term…
Abstract
The present disclosure provides methods and compositions for treating lysosomal storage disorders in subjects, comprising genetically modifying cells from the subjects ex vivo by integrating therapeutic transgenes into the CCR5 locus.
Claims13
A method of genetically modifying a cell from a subject with a lysosomal storage disorder (LSD), the method comprising:
- introducing into a cell isolated from the subject a single guide RNA (sgRNA) targeting the CCR5 locus, an RNA-guided nuclease, and a homologous donor template comprising a transgene encoding a therapeutic protein that is absent or deficient in the subject, wherein:
- the sgRNA binds to the nuclease and directs it to a target sequence at the CCR5 locus in the genome of the cell whereupon the nuclease cleaves the CCR5 locus at the target sequence, wherein:
- the homologous donor template comprises a sequence having 96% or greater identity to SEQ ID NO:
- 7 and the transgene is integrated into the genome by homology directed recombination (HDR) at the site of the cleaved CCR5 locus, and wherein
- the integrated transgene directs the expression of the therapeutic protein in the cell.
2 more elements
Dependencies are inferred from the claim text.