Transthyretin amyloidosis with cardiomyopathy (ATTR-CM) is a progressive, often fatal disease. Nexiguran ziclumeran (nex-z) is an investigational therapy based on CRISPR-Cas9 (clustered regularly ...
Crispr Therapeutics (CRSP) is downgraded to "Hold" as current valuation already reflects optimism for CTX310 Phase I results and pipeline progress. Intellia Therapeutics (NTLA) maintains a "Buy" ...
A single infusion of an experimental gene-editing drug appears safe and effective for cutting cholesterol, possibly for life, according to a small early study released Saturday. The study, which ...
For the first time, doctors have treated a baby born with a rare, life-threatening genetic disorder with a gene-editing therapy scientists tailored to specifically repair his unique mutation. The baby ...
Angiopoietin-like protein 3 (ANGPTL3) inhibits lipoprotein and endothelial lipases. ANGPTL3 loss-of-function genetic variants are associated with decreased levels of low-density lipoprotein ...
Like the human immune system, bacteria learn from past infections. CRISPR sequences—short snippets of DNA from previous viruses—guide destructive enzymes towards invading bacteriophages that express ...
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