The disclosure features methods of correcting a mutation in the human beta-globin (HBB) gene in a cell or population of cells. The disclosure also features methods of increasing repair of a DNA double stranded break (DSB) in an HBB gene by the homology-directed repair (HDR) pathway. The disclosure also features compositions for use in the methods.

Disclosed herein are cells including cells expressing CD24 and related methods of their use and generation. In some embodiments, the cells disclosed herein do not express one or more MHC I and/or MHC II human leukocyte antigens. In some embodiments, the cells are hypoimmunogenic.

The present disclosure provides for systems, methods, and compositions for targeting nucleic acids. In particular, the invention provides Cas proteins and their use in modifying target sequences.

As described below, the present invention features genetically modified immune cells having enhanced anti-neoplasia activity, resistance to immune suppression, and decreased risk of eliciting a graft versus host reaction, or a combination thereof. The present invention also features methods for producing and using these modified immune effector cells.

Provided herein are systems and methods for detecting genomic structural variants using a non-application gene-editing sample preparation followed by long-read sequencing.

Provided herein are compositions and methods for improving the genome-wide specificities of targeted base editing technologies.

The present disclosure provides RNA-guided CRISPR-Cas effector proteins, nucleic acids encoding same, and compositions comprising same. The present disclosure provides ribonucleoprotein complexes comprising: an RNA-guided CRISPR-Cas effector protein of the present disclosure; and a guide RNA. The present disclosure provides methods of modifying a target nucleic acid, using an RNA-guided CRISPR-Cas effector protein of the present disclosure and a guide RNA.

Provided herein is a method of delivering a biological material, including a genome-editing agent, to the interior of a plant cell wherein the method includes contacting the plant cell with a complex comprising the biological material and a fluorous agent. Also provided herein are compositions and reagents for practicing the method.

The present invention provides methods of treating, preventing or delaying the onset of bone marrow failure in Fanconi Anemia patients.

A Class 2, Type VI clustered regularly interspaced short palindromic repeat (CRISPR) RNA (crRNA) which comprises a direct repeat (DR) stem loop sequence and a guide or spacer sequence, is provided characterized by a DR selected from those of Table 9. Also described is are methods for generating, selecting, characterizing and optimizing a clustered regularly interspaced short palindromic repeats (CRISPR) RNA (crRNA) for use in the CRISPR-Cas13d system described herein. Also provided is a screening method to identify crRNA particularly suited for use with specified targets. Further, the invention includes non-naturally occurring, synthesized or engineered crRNAs as described herein along with nucleic acid molecule, vectors, RNPs, cells, libraries, and compositions comprising the same, and uses thereof in treating a disease or in functionally screening a gene. A method for blocking an RNA target without degradation and a method for modification of multiple RNA targets using the same CRISPR effector protein are also disclosed.