Massachusetts Institute of Technology

The Broad Institute, Inc.

Harvard College

Beam Therapeutics Inc.

The Board Institute, Inc.

Ian Slaymaker

Bernd Zetsche

Feng Zhang

Omar O Abudayyeh

Jonathan S Gootenberg

Yi Yu

Nicole Marie Gaudelli

Michael S Packer

Seung-Joo Lee

David A Born

Linyi Gao

Johnathan S Gootenberg

David Benjamin Turitz Cox

Randall Jeffrey Platt

Jason Michael Gehrke

Yvonne Aratyn

Shaunna Berkovitch

Genesis Lung

Francine Gregoire

Angelica Messana

Natalie Petrossian

Nicole Gaudelli

Omar Abudayyeh

The invention provides for systems, methods, and compositions for targeting nucleic acids. In particular, the invention provides non-naturally occurring or engineered DNA-targeting systems comprising a novel DNA-targeting CRISPR effector protein and at least one targeting nucleic acid component like a guide RNA. Methods for making and using and uses of such systems, methods, and compositions and products from such methods and uses are also disclosed and claimed.

CRISPR enzymes and systems

The invention provides for systems, methods, and compositions for targeting nucleic acids. In particular, the invention provides non-naturally occurring or engineered DNA-targeting systems comprising a novel DNA-targeting CRISPR effector protein and at least one targeting nucleic acid component like a guide RNA. Aspects of the invention in particular relate to Cpf1 mutants having altered PAM specificity.

CRISPR enzymes and systems with modified pam specificity

Disclosed and claimed are mutation(s) or modification(s) of the CRISPR enzyme, for example a Cas enzyme such as a Cas9, which obtain an improvement, for instance a reduction, as to off-target effects of a CRISPR-Cas or CRISPR-enzyme or CRISPR-Cas9 system or complex containing or including such a mutated or modified Cas or CRISPR enzyme or Cas9. Methods for making and using and uses of such mutated or modified Cas or CRISPR enzyme or Cas9 and systems or complexes containing the same and products from such methods and uses are also disclosed and claimed.

CRISPR enzyme mutations reducing off-target effects

The invention provides for systems, methods, and compositions for targeting nucleic acids. In particular, the invention provides non-naturally occurring or engineered DNA-targeting systems comprising a novel DNA-targeting CRISPR effector protein and at least one targeting nucleic acid component like a guide RNA Methods for making and using and uses of such systems, methods, and compositions and products from such methods and uses are also disclosed and claimed.

Crispr enzymes and systems

The present invention features compositions and methods for editing deleterious mutations associated with hemoglobinopathies, such as sickle cell disease (SCD). In particular embodiments, the invention provides methods for correcting mutations in a beta globin polynucleotide using modified adenosine base editors termed 'ABE8' having unprecedented levels (e.g., >60-70%) of efficiency.

Compositions and methods for treating hemoglobinopathies

The present invention features compositions and methods for editing deleterious mutations associated with hemoglobinopathies, such as sickle cell disease (SCD). In particular embodiments, the invention provides methods for correcting mutations in a beta globin polynucleotide using modified adenosine base editors termed 'ABE8' having unprecedented levels (e.g., &#x3e;60-70%) of efficiency.

Methods and compositions for inducing a plurality of mutations in transgenic Cas9 eukaryotes to model a neuronal disease or disorder. The invention further comprehends testing putative treatments with such models, e.g., testing putative chemical compounds that may be pharmaceutically relevant for treatment or gene therapy that may be relevant for treatment, or combinations thereof. The invention allows for the study of genetic diseases and putative treatments to better understand and alleviate a genetic disease or a condition, e.g., autism, autism-spectrum disease or disorder, obsessive-compulsive disorder, or psychiatric disorders.

Use and production of CHD8+/&#x2212; transgenic animals with behavioral phenotypes characteristic of autism spectrum disorder

The disclosure provides compositions comprising novel adenosine base editors (e.g., ABE8) that have increased efficiency and methods of using these adenosine deaminase variants for editing a target sequence.

Adenosine deaminase base editors and methods of using same to modify a nucleobase in a target sequence

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Cambridge, MA, United States of America

Ian Slaymaker