Social Media Ideas for Gene Therapy

Gene therapy is changing how we treat disease

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...

Gene Therapy Reverses Fragile X Traits in Preclinical Study

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...

Gene therapy shows promise in ARC syndrome, a deadly childhood liver disease

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...

Historic Gene Therapy Milestone for Cockayne Syndrome Carries Implications for Hearing Loss Community

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...

First-in-the-world gene therapy delivers missing gene directly to infant's brain

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...

Gene Therapy Proven to Shield Brain from TDP-43 Damage

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,...

Boston Children’s Hospital – Gene therapy for hearing loss

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...

Precise Gene Control Using FDA-Approved RNA Splicing Drug

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...

FMR1 Gene Therapy Rescues Fragile X Syndrome Traits in Mice

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...

Man functionally cured of sickle cell disease through gene therapy

A man is now the first person in Louisiana to be functionally cured of sickle cell disease through gene therapy.

Fifteenth In Vivo Lentiviral Vector-Based Therapeutic Technology Added to VIVEbiotech’s CGT Platform

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...

Gene Therapy Reverses Severe Traits of Fragile X Syndrome

The therapy restored FMRP expression in crucial brain regions, resulting in reduced audiogenic seizure susceptibility, normalized sensory hyperactivity, and complete reversal of el...

Gorilla Adenovirus Brings Natural Edge to Cancer Therapy

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...

Gene therapy for children with sickle cell disease approved by FDA

A new treatment for children aged 2 or older with sickle cell disease has been approved by the U.S. Food & Drug Administration.

How a CRISPR Gene Therapy Could Change Life for Young Children With Sickle Cell Disease

Learn how gene editing is transforming sickle cell disease treatment and why expanding access to younger children matters.

Caveolin-1 Gene Therapy Reduces TDP-43 Related Neurodegeneration in a Mouse Model

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...

Man functionally cured of sickle cell disease through gene therapy

A man is now the first person in Louisiana to be functionally cured of sickle cell disease through gene therapy.

Long-Term AAV9 Gene Therapy for Spinal Muscular Atrophy

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...

Gene therapy for children with sickle cell disease approved by FDA

A new treatment for children aged 2 or older with sickle cell disease has been approved by the U.S. Food & Drug Administration.

A gene therapy is rewriting Huntington’s disease

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...

Sickle cell breakthrough: World's first CRISPR therapy cures Louisiana man's lifelong disease

Gene therapy for children with sickle cell disease approved by FDA

A new treatment for children aged 2 or older with sickle cell disease has been approved by the U.S. Food & Drug Administration.

Novel gene therapy platform restores muscle function in Duchenne muscular dystrophy model

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...

Breakthrough Parkinson’s Drug Targets Disease at Its Genetic Roots

An experimental gene-silencing therapy safely lowered LRRK2 protein levels in an early Parkinson’s trial, but its effect on symptoms remains untested. An experimental medicine buil...