CRISPR-Cas9 genome editing exploits the CRISPR-Cas system to modify a genome in a targeted manner. Guided by RNA, the Cas9 endonuclease breaks DNA at a target sequence. Imprecise repair of the double ...
In a decision released today (May 12), the U. S. Court of Appeals for the Federal Circuit in Washington, D.C., ordered the U.S. Patent and Trademark Office’s Patent Trial and Appeal Board (PTAB) to ...
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 ...
A single dose of an experimental CRISPR therapy cut LDL cholesterol and triglycerides by about half in patients with difficult-to-treat lipid disorders. The reductions were still present one year ...
Researchers have identified critical spots on the genome where gene editing could cause an unwanted response, and they provide recommendations for safer approaches. CRISPR/Cas9 is a commonly used, ...
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 ...
Most approved gene therapies today, including those involving CRISPR-Cas9, work their magic on cells removed from the body, after which the edited cells are returned to the patient. This technique is ...
A Cleveland Clinic first-in-human clinical trial has shown that a one-time infusion of a gene-editing therapy using CRISPR-Cas9 was effective and safe in reducing LDL ("bad") cholesterol and ...
The practice of genetic modification is as old as humanity. For thousands of years, humans have bred crops, livestock and even pets that possess desirable traits. This selective process, which alters ...
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