The General Hospital Corporation

Harvard College

Massachusetts Institute of Technology

Boston University

Fox Chase Cancer Center

Beam Therapeutics Inc.

J Keith Joung

Benjamin Kleinstiver

Shengdar Q Tsai

Morgan Maeder

Jeffry D Sander

Vikram Pattanayak

James Angstman

Jason Michael Gehrke

Peter Cabeceiras

Jeffrey N Miller

Carl O Pabo

Karl Petri

Yanfang Fu

Rebecca Tayler Cottman

Alexander Sousa

Y Esther Tak

Shengdar Tsai

Erica A Golemis

Nicole Marie Gaudelli

Michael S Packer

Ilya G Serebriiskii

Ahmad S Khalil

Simon Dove

Minhee Park

Ann Hochschild

John Evans

Divya Israni

Julian Grunewald

David Bryson

Ronghao Zhou

Yan-fang Fu

Kanae Esther Sasaki

Human-derived virus-like particles (heVLPs), comprising a membrane comprising a phospholipid bilayer with one or more HERV-derived envelope proteins on the external side; one or more HERV-derived GAG proteins in the heVLP core, and a cargo molecule, e.g., a biomolecule and/or chemical cargo molecule, disposed in the core of the heVLP on the inside of the membrane, wherein the heVLP does not comprise a gag protein, except for gag proteins that are encoded in the human genome or gag proteins that are encoded by a consensus sequence that is derived from gag proteins found in the human genome, and methods of use thereof for delivery of the cargo molecule to cells.

Engineered human-endogenous virus-like particles and methods of use thereof for delivery to cells

Enhanced virus-like particles (eVLPs), comprising a membrane comprising a phospholipid bilayer with one or more virally-derived glycoproteins on the external side; and a cargo disposed in the core of the eVLP on the inside of the membrane, wherein the eVLP does not comprise an exogenous gag/pol protein, and methods of use thereof for delivery of the cargo to cells.

Enhanced virus-like particles and methods of use thereof for delivery to cells

The present invention relates to methods to improve the absolute rate of homology-directed repair (HDR) and/or to improve the relative rate of HDR compared with non-homologous end joining (NHEJ).

Methods for increasing efficiency of nuclease-induced homology-directed repair

Engineered CRISPR-Cas9 nucleases with altered and improved PAM specificities and their use in genomic engineering, epigenomic engineering, and genome targeting.

Engineered CRISPR-Cas9 nucleases with altered PAM specificity

Drug-inducible, tunable, and multiplexable Clustered Regularly Interspaced Short Palindromic Repeats fromand1 (Cpf1)-based activators, and methods of use thereof.

Inducible, tunable, and multiplex human gene regulation using CRISPR-Cpf1

Engineered CRISPR fromand1 (Cpf1) nucleases with improved targeting range and enhanced on-target activity, and their use in genomic engineering, epigenomic engineering, base editing, genome targeting, genome editing, and in vitro diagnostics.

Variants of Cpf1 (Cas12a) with altered PAM specificity

Fusion proteins comprising a DNA binding domain, e.g., a TAL effector repeat array or zinc finger, and a catalytic domain comprising a sequence that catalyzes hydroxylation of methylated cytosines in DNA, and methods of use thereof.

TAL-Tet1 fusion proteins and methods of use thereof

Provided herein are compositions and methods of using base editors comprising a polynucleotide programmable nucleotide binding domain and a nucleobase editing domain in conjunction with a guide polynucleotide. Also provided herein are base editor systems for editing nucleobases of target nucleotide sequences.

Methods of substituting pathogenic amino acids using programmable base editor systems

Unbiased, genomewide and highly sensitive methods for detecting mutations, e.g., off-target mutations, induced by engineered nucleases.

Genomewide unbiased identification of DSBs evaluated by sequencing (GUIDE-Seq)

Methods and constructs for RNA-guided targeting of heterologous functional domains such as transcriptional activators to specific genomic loci.

RNA-guided targeting of genetic and epigenomic regulatory proteins to specific genomic loci

Methods and compositions for performing highly sensitive in vitro assays to define substrate preferences and off-target sites of nucleic-acid binding, modifying, and cleaving agents.

Highly sensitive in vitro assays to define substrate preferences and sites of nucleic-acid binding, modifying, and cleaving agents

Engineered adenine base editor (ABE) variants with reduced RNA editing activity, and methods of using the same.

Adenine DNA base editor variants with reduced off-target RNA editing

Growing community of inventors

Winchester, MA, United States of America

J Keith Joung