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CRISPR/Cas-related compositions and methods for treatment of Usher Syndrome and/or Retinitis Pigmentosa are disclosed herein.


Patent
Editas Medicine | Date: 2017-08-16

This application provides improved methods of genome editing. Cas9 molecules can be used to create a break in a genomic region of interest. To increase the likelihood that the break is repaired by HDR (homology-directed repair), the cell can be contacted with molecules that bring a template nucleic acid in close proximity to the break, under conditions that allow the cell to repair the break using the template nucleic acid.


CRISPR/CAS-related compositions and methods for treatment of Primary Open Angle Glaucoma (POAG) are disclosed.


CRISPR/CAS-related compositions and methods for treatment of Lebers Congenital Amaurosis 10 (LCAIO) are disclosed.


CRISPR/CAS-related compositions and methods for treatment of a subject at risk for or having a HIV infection or AIDS are disclosed.


CRISPR/CAS-related compositions and methods for treatment of HSV-1 are disclosed.


CRISPR/CAS-related compositions and methods for treatment of Cystic Fibrosis (CF).


CRISPR/Cas-related composition and methods for treatment of cancer, in particular by using gRNA molecules comprising a targeting domain which is complementary with a target domain from the FAS, BID, CTLA4, PDCD1, CBLB, PTPN6, TRAC or TRBC gene. In some embodiments, gRNAs are used with Cas9 enzymes to cause a cleavage event in said genes within engineered chimeric antigen receptor (CAR) T cells. In some embodiments, CAR T cells are modified to reduce, decrease or repress expression of said genes using gRNAs and Cas9 enzymes; said modified CAR T-cells are meant for therapeutic uses, especially for cancer.


CRISPR/CAS-related compositions and methods for treatment of Sickle Cell Disease (SCD) are disclosed.


CRISPR/CAS-related compositions and methods for treatment of a subject at risk for or having a HIV infection or AIDS are disclosed.

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