Pathology

In vivo

Endonuclease

Amyloidosis

Biology

Gene

Transthyretin

Genetic enhancement

Endocrinology

Genome editing

Cas9

Molecular biology

Computational biology

CRISPR

Genetics

Biochemistry

Medicine

New England Journal of Medicine

Transthyretin amyloidosis, also called ATTR amyloidosis, is a life-threatening disease characterized by progressive accumulation of misfolded transthyretin (TTR) protein in tissues, predominantly the nerves and heart. NTLA-2001 is an in vivo gene-editing therapeutic agent that is designed to treat ATTR amyloidosis by reducing the concentration of TTR in serum. It is based on the clustered regularly interspaced short palindromic repeats and associated Cas9 endonuclease (CRISPR-Cas9) system and comprises a lipid nanoparticle encapsulating messenger RNA for Cas9 protein and a single guide RNA targeting TTR.

CRISPR-Cas9 In Vivo Gene Editing for Transthyretin Amyloidosis