Patent classifications
C12N15/11
Therapeutic Editing
Disclosed herein are compositions that comprise engineered polynucleotides, pharmaceutical compositions comprising the same, methods of making the same, and methods of treatment comprising the compositions that comprise the engineered polynucleotides.
METHODS FOR TARGETED CELL DEPLETION
Described herein are compositions, kits and methods for shredding the genomes of selected cell types, for example, the genomes of selected cancer cell types.
METHODS FOR TARGETED CELL DEPLETION
Described herein are compositions, kits and methods for shredding the genomes of selected cell types, for example, the genomes of selected cancer cell types.
COMPOSITIONS COMPRISING A NUCLEASE AND USES THEREOF
The present invention relates to genes coding for nucleases, processes for characterizing the nucleases, cells comprising the nucleases, and methods of using the nucleases.
COMPOSITIONS COMPRISING A NUCLEASE AND USES THEREOF
The present invention relates to genes coding for nucleases, processes for characterizing the nucleases, cells comprising the nucleases, and methods of using the nucleases.
RETROTRANSPOSONS AND USE THEREOF
Systems and methods for targeted gene modification, targeted insertion, perturbation of gene transcripts, and nucleic acid editing. Novel nucleic acid targeting systems comprise components of CRISPR systems and non-LTR retrotransposon elements.
RETROTRANSPOSONS AND USE THEREOF
Systems and methods for targeted gene modification, targeted insertion, perturbation of gene transcripts, and nucleic acid editing. Novel nucleic acid targeting systems comprise components of CRISPR systems and non-LTR retrotransposon elements.
DEVELOPMENT OF DUAL-gRNA APPROACH WITH UNDETECTABLE OFF-TARGET EFFECT TO CORRECT C9ORF72 REPEAT EXPANSION AND C9ORF72 PATHOLOGY
A group of gRNAs is provided, and the group of gRNAs comprises a first gRNA molecule; and a second gRNA molecule capable of defining a region in a genome sequence with the first gRNA molecule, wherein the region in the genome comprises a target sequence in need of removal.
DEVELOPMENT OF DUAL-gRNA APPROACH WITH UNDETECTABLE OFF-TARGET EFFECT TO CORRECT C9ORF72 REPEAT EXPANSION AND C9ORF72 PATHOLOGY
A group of gRNAs is provided, and the group of gRNAs comprises a first gRNA molecule; and a second gRNA molecule capable of defining a region in a genome sequence with the first gRNA molecule, wherein the region in the genome comprises a target sequence in need of removal.
ADOPTIVE CELL THERAPY WITH ZBTB20 SUPPRESSION
Provided are methods, compositions, and cells for use in adoptive cell therapy for the treatment of cancer. The methods involve administering an effective amount of cells to a subject, wherein the cells are modified ex vivo to suppress endogenous Zbtb20 expression and/or activity within the modified cells. The cells may comprise a dominant negative Zbtb20 capable of suppressing endogenous Zbtb20 activity, at least one shRNA capable of suppressing endogenous Zbtb20 expression, or at least one sgRNA capable of suppressing endogenous Zbtb20 expression. The cells may further comprise an exogenous TCR and/or CAR suitable for treating cancer. The method can further involve administering one or more additional cancer therapies, such as cells which express at least one exogenous TCR and/or CAR suitable for treating cancer. The method can provide various advantages, such as a reduction and/or elimination of an amount of cancer cells in the subject.