- Cambridge MA, US Surbhi Goel - Winchester MA, US Sarah Beth Hesse - Arlington MA, US Troy Aaron Luster - Norfolk MA, US Birgit Schultes - Arlington MA, US Stephanie A. Yazinski - Lynnfield MA, US Pooja Vinay - St. Louis MO, US
Assignee:
Intellia Therapeutics, Inc. - Cambridge MA
International Classification:
C12N 15/11 C12N 9/22 C07K 14/725
Abstract:
Compositions and methods for editing, e.g., altering a DNA sequence, within the TRBC1, TRBC2 and/or TRAC genes are provided. Compositions and methods for immunotherapy are provided, for example.
Compositions And Methods For Hydroxyacid Oxidase 1 (Hao1) Gene Editing For Treating Primary Hyperoxaluria Type 1 (Ph1)
- Cambridge MA, US Shobu Odate - Arlington MA, US Bradley Andrew Murray - Saratoga CA, US Reynald Michael Lescarbeau - Medford MA, US Anette Huebner - Somerville MA, US Walter Strapps - Dedham MA, US Sarah Beth Hesse - Arlington MA, US
Assignee:
Intellia Therapeutics, Inc. - Cambridge MA
International Classification:
C12N 15/113 C12N 9/22 A61P 13/02
Abstract:
Compositions and methods for editing, e.g., introducing double-stranded breaks, within the HAO1 gene are provided. Compositions and methods for treating subjects having primary hyperoxaluria type 1 (PH1), are provided.
This application relates to compositions and methods for excising trinucleotide repeats (TNRs) contained within intron 3 of TCF4, such as is seen in subjects having Fuchs endothelial corneal dystrophy (FECD), PSC, and Schizophrenia. Compositions comprising guide sequences targeting the alpha 2 subunit of collagen VIII are also disclosed for treatment of mutations therein that may contribute to FECD.