The present invention includes compositions and methods for identifying cancer driver mutations through use of an AAV-CRISPR library and molecular inversion sequencing probes (MIPs).

The present invention includes novel compositions and methods for identifying driver mutations in glioblastoma. In one aspect, the invention includes an AAV-CRISPR library for identifying driver mutations in, and thus treatments for glioblastoma.

Described herein are methods and vectors for rational, multiplexed manipulation of chromosomes within open reading frames (e.g., in protein libraries) or any segment of a chromosome in a cell or population of cells, in which various CRISPR systems are used.

The present disclosure describes the fusogenic promoting activity of the Myomixer protein. This polypeptide, when expressed in non-muscle cells with the Myomaker protein, is able to increase fusion of the cell with a muscle cell, but not with other non-muscle cells, as compared to cells only expression the Myomaker protein. The use of this protein and cells expressing it in the delivery of exogenous genetic material to muscle cells also is described.

Described herein are method for generating a vector for editing a cell. The method comprises ligating into a vector that encodes a portion of a gRNA a cassette comprising at least one editing cassette, a promoter, and a gene encoding another portion of the gRNA. Upon ligation, the portion of the gRNA from the editing cassette and the other portion of the gRNA are ligated and form a functional gRNA.

The present disclosure provides compositions and methods for inhibiting viral pathogenesis by targeting long noncoding ribonucleic acids.

Cas-protein-ready tau biosensor cells, CRISPR/Cas synergistic activation mediator (SAM)-ready tau biosensor cells, and methods of making and using such cells to screen for genetic modifiers of tau seeding or aggregation are provided. Reagents and methods for sensitizing such cells to tau seeding activity or tau aggregation or for causing tau aggregation are also provided.

A guide RNA comprising: a gRNA spacer sequence at the 5 end of the guide RNA, wherein the spacer sequence is complementary to a target gene, a scaffold sequence that binds to Cas9, and an RNA capture and sequencing domain comprising: a barcode sequence, and a primer binding sequence; nucleic acids and vectors encoding the guide RNA; cells expressing the guide RNA; and a library comprising a plurality of guide RNAs. Also disclosed are methods of introducing a genetic perturbation into a cell, methods of assessing an effect of at least one genetic perturbation on RNA expression in a cell, methods of identifying nucleic acid sequences associated with a disease state and a method of identifying candidate therapeutic agents.

The disclosure provides novel personalized therapies, kits, transmittable forms of information and methods for use in treating patients having cancer, wherein the cancer is amenable to therapeutic treatment with an inhibitor, e.g., an inhibitor of any of the targets disclosed herein. Kits, methods of screening for candidate inhibitors, and associated methods of treatment are also provided.

The present disclosure provides methods for treating a disease or disorder or sensitizing a cell to phagocytosis. The methods comprise contacting a cell with an inhibitor of Adipocyte Plasma Membrane Associated Protein (APMAP), an agonist of fatty-acid G-protein coupled receptor GPR84, or a combination thereof. The methods may further comprise contacting the cell with at least one or both of a tumor antigen (TA)-targeting antibody and a CD47 blocking antibody. The present disclosure also provides methods for determining cellular regulators of phagocytosis.