Editas Medicine, Inc.

Luis A Barrera

Jennifer Leah Gori

Edouard Aupepin De Lamothe-Dreuzy

Jack Heath

Anne Helen Bothmer

Cecilia Cotta-Ramusino

Georgia Giannoukos

Dawn Ciulla

Cecilia Fernandez

Terence Ta

Genome editing systems, guide RNAs, dead guide RNAs, and CRISPR-mediated methods are provided for altering portions of a target nucleic acid.

CRISPR/RNA-guided nuclease systems and methods

Genome editing systems, guide RNAs, and CRISPR-mediated methods are provided for altering portions of the HBG1 and HBG2 loci, portions of the erythroid specific enhancer of the BCL11A gene, or a combination thereof, in cells and increasing expression of fetal hemoglobin.

Systems and methods for the treatment of hemoglobinopathies

The present disclosure is directed to methods of producing a modified nucleic acid comprising a precise deletion in a target nucleic acid in a cell comprising generating, within the cell, a first single strand break on a first strand of the target nucleic acid and a second single strand break on a second strand of the target nucleic acid, thereby forming a double strand break in the target nucleic acid having a first 3' overhang and a second 3&#x2032; overhang; processing the first 3&#x2032; overhang and the second 3&#x2032; overhang with an exonuclease molecule, thereby deleting the segment of the target nucleic acid that was located between the first single strand break and the second single strand break, and forming a processed double strand break; and allowing the processed double strand break to be repaired by at least one DNA repair pathway, thereby producing the modified nucleic acid comprising the precise deletion in the target nucleic acid in the cell. Gene editing systems, vectors, polynucleotides, and methods of treatment are also disclosed herein.

Use of exonucleases to improve CRISPR/CAS-mediated genome editing

The present disclosure relates to methods of assessing a sample of guide RNAs (gRNAs).

Growing community of inventors

Somerville, MA, United States of America

Luis A Barrera