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HN Summary • Gene therapy is shifting medicine from treating symptoms to targeting disease at its genetic source, offering new hope for conditions such as Huntington’s disease, sic...
A new gene therapy has been used to successfully treat a deadly childhood liver disease in mice that model the disease, according to researchers at UCL and Great Ormond Street Hosp...
In May 2025, a young boy received an injection delivering a healthy copy of a gene called OTOF into the cochlea of his inner ear — one of the first patients with hereditary hearing...
A gene therapy aimed at correcting fragile X syndrome by restoring the missing FMR1 protein has demonstrated multiple disease-relevant improvements in a mouse model, according to a...
A 6-year-old boy with Cockayne syndrome — a rare genetic disorder that causes hearing loss, vision impairment, and early death — has become the first child to receive an experiment...
An experimental gene therapy called SynCav1 can protect the central nervous system from the cognitive decline linked to TDP-43-related proteinopathy, a primary driver of FTD, ALS,...
An 8-month-old infant with severe genetic epilepsy has become the first patient in the world to receive an experimental gene replacement therapy designed to restore the function of...
In a groundbreaking advance poised to reshape gene therapy and molecular medicine, researchers have unveiled a novel strategy for precise gene regulation via RNA splicing modulatio...
A gene therapy that instructs cells to produce more of an anti-ageing protein called klotho is about to be offered by a US company at overseas clinics to bypass FDA rules
A gorilla adenovirus originally developed for vaccines is showing unexpected promise as a cancer therapy—one whose therapeutic properties may be baked into its native biology rathe...
In a groundbreaking advancement in the treatment of fragile X syndrome (FXS), researchers have successfully deployed gene therapy targeting the FMR1 gene, restoring key neurologica...
You might recall that gene therapy to overexpress caveolin-1 in the brain was recently shown to reduce pathology in a mouse model of Alzheimer's disease. In today's open access pap...
A new treatment for children aged 2 or older with sickle cell disease has been approved by the U.S. Food & Drug Administration.
A new treatment for children aged 2 or older with sickle cell disease has been approved by the U.S. Food & Drug Administration.
A man is now the first person in Louisiana to be functionally cured of sickle cell disease through gene therapy.
Growing interest in in vivo cell and gene therapies is driving significant investment, given their potential to address some of the manufacturing and commercialization challenges a...
A man is now the first person in Louisiana to be functionally cured of sickle cell disease through gene therapy.
A new treatment platform developed by researchers at the University of Texas MD Anderson Cancer Center was able to deliver messenger RNA (mRNA) of the full-length DMD gene into pre...
Learn how gene editing is transforming sickle cell disease treatment and why expanding access to younger children matters.
The therapy restored FMRP expression in crucial brain regions, resulting in reduced audiogenic seizure susceptibility, normalized sensory hyperactivity, and complete reversal of el...
A new treatment for children aged 2 or older with sickle cell disease has been approved by the U.S. Food & Drug Administration.
In a groundbreaking study that could redefine treatment paradigms for spinal muscular atrophy (SMA), researchers have released a comprehensive long-term comparative analysis of ade...
As someone who has spent decades peering into skulls and pondering the fragile machinery inside, I have seen firsthand how our gray matter can falter under the weight of time, gene...
Med-El has acquired two gene therapy programs from Rescue Hearing Inc targeting mutations in the MYO7A and STRC genes, expanding the hearing implant company's pipeline into biologi...
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