Viral vectors dominate gene therapy, with lentivirus, adenovirus, and AAV being key players, each with unique advantages and limitations. Non-viral vectors, such as lipid nanoparticles and GalNAc, ...

The US Food and Drug Administration on Thursday approved the first gene therapy for inherited hearing loss, a one-time treatment that proved to be life-changing for a small number of children in a ...

Gene therapy approaches include gene replacement, suppression, and editing, each matched to specific genetic mechanisms in hereditary hearing loss. Preclinical studies in rodent models show promise, ...

Approval in severe-to-profound and profound OTOF-related hearing loss is based on pivotal results of the CHORD trial demonstrating 80% of participants achieved or surpassed a hearing level meeting the ...

Four decades have passed since cochlear implants gave infants born deaf the ability to hear. Now, gene therapy promises to restore natural hearing for those born with a rare form of deafness, and the ...

As cell and gene therapy leaders gathered in Maryland to discuss accelerating clinical trials in children, one “cutting edge” session focused on the need to expedite more bespoke gene editing ...

Jessica Rendall is a reporter with experience covering a variety of health and wellness topics, including chronic disease, infectious disease, telemedicine, mental health and health technology. Kelly ...

The search for gene therapies to treat rheumatoid arthritis (RA) has moved in fits and starts over the past two decades, with trials starting and stopping, and their focus shifting to osteoarthritis ...

Theresa Gaffney is the lead Morning Rounds writer and reports on health care, new research, and public policy, with a particular interest in mental health, gender-affirming care, and LGBTQ+ patient ...

Gene editing is now reaching the mainstream, ushering in a new era of genetic manipulation. Traditionally, inserting or deleting entire genes, regulating their expression, and altering specific ...