CRISPR in the Clinic
Type
Blog Post
Published
Nov. 2, 2023, 1:15 p.m.
...amyloidosis (hATTR)
hATTR is caused by a variety of single nucleotide polymorphisms (SNPs) in the TTR gene and... the liver. Thus, it has been used for hATTR therapy, since TTR is primarily made in the liver. Similarly...is molecularly characterized by misfolding of the TTR protein, which contributes to the disease phenotype...phenotype. Intellia initiated a CRISPR therapy for hATTR with the first-ever systemic CRISPR therapy. They ... expected soon on how this therapy is affecting hATTR patient outcomes.
Bacterial infection and HIV
Most...