CRISPR/CAS-RELATED METHODS AND COMPOSITIONS FOR KNOCKING OUT C5

Organization Name

Regeneron Pharmaceuticals, Inc.

Inventor(s)

Kishor Devalaraja-narashimha of Yorktown Heights NY (US)

Lori Morton of Chappaqua NY (US)

Evangelos Pefanis of Somers NY (US)

Suzanne Hartford of Putnam Valley NY (US)

Arti Mahendra Prakash Kanjolia of Malden MA (US)

Sarah Hesse of Arlington MA (US)

CRISPR/CAS-RELATED METHODS AND COMPOSITIONS FOR KNOCKING OUT C5

This abstract first appeared for US patent application 18704681 titled 'CRISPR/CAS-RELATED METHODS AND COMPOSITIONS FOR KNOCKING OUT C5

Original Abstract Submitted

Guide RNAs and CRISPR/Cas systems targeting a C5 locus or gene, lipid nanoparticles or viral vectors comprising such guide RNAs or CRISPR/Cas systems, and cells or animals comprising such guide RNAs or systems are provided. Methods of modifying or knocking down or knocking out a C5 locus or gene using the CRISPR/Cas systems are also provided, as well as use of the CRISPR/Cas systems in prophylactic and therapeutic applications for treatment and/or prevention of a disease, disorder, or condition associated with C5 and/or for ameliorating at least one symptom associated with such disease, disorder, or condition.