The Brigham and Women's Hospital, Inc.

The Broad Institute, Inc.

Basudeb Maji

Amit Choudhary

Soumyashree Ashok Gangopadhyay

Miseon Lee

Debasish Manna

Bridget K Wagner

Kurt Cox

Elisa Franco

Peng Wu

Hari Subramanian

Mengchao Shi

The disclosure includes non-naturally occurring or engineered DNA- or RNA-guided nuclease systems, comprising CRISPR enzymes associated with at least one destabilization domain (DD) and photocaged stabilization ligands with at least one photocage molecule, along with compositions, systems and complexes involving such systems, nucleic acid molecules and vectors encoding the same, delivery systems involving the same, uses thereof.

Methods and compositions for optochemical control of CRISPR-CAS9

Compositions and methods for inhibiting the activity of RNA-guided endonucleases, and methods for identifying such compositions are included. Methods of treatment using the inhibitory compounds, formulations and methods are also detailed, as well as control of nucleic acid editing systems and methods using said compounds.

Inhibitors of RNA-guided nuclease target binding and uses thereof

The disclosure includes zinc prodrugs for targeted delivery of therapeutic, diagnostic or imaging agents to β-cells and methods of use therefor. The disclosure also includes targeted delivery of small molecules to β-cells that stabilize and activate CRISPR effector proteins comprising at least one destabilization domain, to enable CRISPR-based genome editing and transcriptional activation or repression in β-cells.

Targeted delivery to beta cells

The embodiments disclosed herein utilize fluorescence polarization based preliminary screen to identify a putative set of Cas inhibitors from an initial set of candidate inhibitors. The primary screening assay is followed by secondary screening assay to validate the putative set of inhibitors selected by the preliminary screen. In some embodiments, the present disclosure includes compositions and methods are provided for the inhibition of the function of RNA guided endonucleases, including the identification and use of such inhibitors.

CRISPR protein inhibitors

Growing community of inventors

Cambridge, MA, United States of America

Basudeb Maji