A single infusion of a CRISPR-based gene editing therapy significantly reduced LDL-C and PCSK9 levels in patients with heterozygous familial hypercholesterolemia (HeFH), based on findings from the VERVE-101 trial presented Nov. 12 at AHA 2023. In presenting the findings, Andrew Bellinger, MD, PhD, said they provide the first proof-of-concept for in vivo DNA base editing in humans.
A single IV infusion of NTLA-2001, a novel CRISPR/Cas9-based gene editing therapy, significantly reduced circulating transthyretin (TTR) protein levels in patients with ATTR amyloid cardiomyopathy, a progressive and fatal cause of heart failure, according to late-breaking research presented today at the American Heart Association’s Scientific Sessions 2022. The meeting, held in person in Chicago and
Improvements in technology and delivery techniques could revolutionize treatment of genetic disorders of vision In a new paper, University of California, Irvine researchers explain how precision genome editing agents have enabled precise gene correction and disease rescue in inherited retinal diseases (IRDs). The study, titled, “Precision genome editing in the eye,” was published this week
Sickle cell disease is the most common deadly genetic disorder. It results from a mutation that causes normally pliable red blood cells to become brittle and sickle shaped. This leads to pain, organ damage and early death. A team of scientists led by Mitchell J. Weiss, MD, PhD, and Jonathan Yen, PhD, at St. Jude, and David Liu,
Bill Gates calls for reducing health care inequities in a AAAS Annual Meeting plenary address. The number of child deaths around the world has declined by half in the past two decades, but that number could be cut even further with the help of artificial intelligence and gene editing tools, said Bill Gates at the
Researchers rapidly corrected finding through discussions on social media and preprints. Source Nature A study that raised questions over the future health of the world’s first gene-edited babies has been retracted because of key errors that undermined its conclusion. The research, published in June 2019 in Nature Medicine1, had suggested that people with two copies of a natural genetic mutation
The peptide nucleic acid-based technique offers an alternative to CRISPR-Cas9 A research team from Carnegie Mellon University and Yale University will advance their innovative, synthetic nucleic acid-based gene editing technique under a new grant from the National Institutes of Health’s (NIH’s) Somatic Cell Genome Editing (SCGE) Program. “Our PNA technique offers a promising avenue for treating — and possibly curing —
The report, sponsored by ARM, Intellia Therapeutics, and CRISPR Therapeutics, and endorsed by former MEP Maria Teresa Giménez Barbat, discusses the commercial, clinical, regulatory, and ethical landscape and framework for the development of therapeutic for genome editing in Europe.
Instead of augmenting super-soldiers, DARPA wants to boost troops’ natural defenses against engineered diseases — and even undo gene-editing altogether. The Pentagon’s research agency wants to explore the possibility of editing a soldier’s genetic makeup to protect against chemical and biological attacks. Defense Advanced Research Projects Agency director Steven Walker said Monday that he believes
Removal of the genetic defect that causes Friedreich’s ataxia (FA) in heart cells derived from a patient with heart disease associated with FA reversed the cells’ abnormal molecular profile, a study shows. This gene-editing strategy could be a useful tool for regenerative medicine aimed at treating heart defects associated with the condition. The study, “Excision of the
