Gene Editing
Infant with rare disease receives world’s first personalized gene-editing therapy
By the second day of his life, Kyle and Nicole Muldoon knew their newborn son was very sick.
VIDEO: Translational studies may ‘move the field forward’ in rheumatoid arthritis
WASHINGTON — In this video, Jeffrey A. Sparks, MD, MMSc, discusses two translational studies focused on human tissue for the treatment of rheumatoid arthritis.
CRISPR gene-editing therapy shows promise as cardiac amyloidosis treatment in early study
CHICAGO — A CRISPR-Cas9-based therapy targeting the gene encoding transthyretin reduced transthyretin levels in patients with cardiac amyloidosis, researchers reported at the American Heart Association Scientific Sessions.
Gene editing therapy shows potential for CEP290-associated inherited retinal degeneration
A gene editing therapy showed safety and improvement in cone photoreceptor function in patients with CEP290-associated inherited retinal degeneration due to an IVS26 variant, according to a phase 1/2 study.
Commentary: It’s in your DNA
It is almost impossible to pick up a medical journal today without seeing an article or commentary on gene editing.
News you may have missed from AHA 2023: SELECT, artificial intelligence, gene editing, more
Healio | Cardiology Today compiled a list of the top news from the 2023 American Heart Association Scientific Sessions.
Positive interim results announced in phase 2 trial of leukoencephalopathy drug
Vigil Neuroscience Inc. has announced positive interim data from a phase 2 proof-of-concept clinical trial of a monoclonal antibody to treat those suffering from adult-onset leukoencephalopathy with axonal spheroids and pigmented glia.
VIDEO: Deepak L. Bhatt, MD, MPH, discusses gene editing, ORBITA-2, artificial intelligence
PHILADELPHIA — In this video exclusive, Deepak L. Bhatt, MD, MPH, provides his perspective on research presented at the American Heart Association Scientific Sessions.
Early study suggests gene editing can lower LDL cholesterol via PCSK9 inhibition
PHILADELPHIA — A novel single-dose CRISPR base-editing infusion for patients with heterozygous familial hypercholesterolemia and advanced CAD reduced serum PCSK9 and lowered LDL by up to 55% in one patient, a speaker reported.
Partnership to develop gene editing therapies for neurological, muscular diseases
Regeneron Pharmaceuticals Inc. and Intellia Therapeutics Inc. have announced an expanded research collaboration intended to develop additional CRISPR-based gene editing therapies focused on neurological and muscular diseases.