Gene Therapy News and Research

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Gene therapy is an experimental technique that uses genes to treat or prevent disease. In the future, this technique may allow doctors to treat a disorder by inserting a gene into a patient’s cells instead of using drugs or surgery.

Scientists identify early life opportunity for in vivo stem cell gene therapy

A team of scientists from the San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget) in Milan, Italy, has identified a unique window shortly after birth in which circulating blood stem cells can be effectively targeted with gene therapy directly in the body.

28 May 2025

Building Bridges to Scalable Allogeneic Cell Therapy

Rui Tostoes, Chief Technology Officer at ImmuneBridge, shares how his team is redefining preclinical development and large-scale manufacturing for allogeneic cell therapies.

From Hanson Wade Group - 7th Allogeneic Cell Therapies Summit 27 May 2025

Study uncovers new molecular mechanisms linked to Alzheimer's cognitive decline

A research team at the University of Barcelona's Institute of Neurosciences (UBneuro) has discovered new molecular mechanisms related to the cognitive decline associated with Alzheimer's disease, the most common dementia.

26 May 2025

NUS researchers develop breakthrough gene delivery technology for immune cells

Researchers at the National University of Singapore (NUS) have developed a scalable, non-viral technology that efficiently delivers genetic material into human immune cells.

26 May 2025

Powerful new toolkit targets vision loss in advanced retinal degeneration

Inherited retinal degenerations (IRDs) are a group of genetic disorders that lead to progressive vision loss as the light-sensing cells of the eye-the photoreceptors-die due to mutations in genes needed for their function and survival.

23 May 2025

New gene delivery systems can reach cells in the brain and spinal cord with exceptional accuracy

Research teams funded by the National Institutes of Health (NIH) have created a versatile set of gene delivery systems that can reach different neural cell types in the human brain and spinal cord with exceptional accuracy.

21 May 2025

UArizona researchers uncover new way to ease postoperative pain in women

University of Arizona Health Sciences researchers identified a potential way to reduce female post-operative pain by inhibiting pituitary prolactin generated by pre-operative stress.

20 May 2025

Personalized gene editing corrects fatal infant metabolic disorder in world-first treatment

Researchers have used a personalised base-editing gene therapy to successfully treat an infant with carbamoyl-phosphate synthetase 1 (CPS1) deficiency, a rare and life-threatening inborn error of metabolism. This pioneering approach chemically corrects the underlying mutation without cutting DNA, showing promise for precision treatment of ultrarare genetic disorders.

20 May 2025

EMA designates allopurinol as first orphan drug for the treatment of Marfan syndrome

The European Medicines Agency (EMA) has designated allopurinol as the first orphan drug for the treatment of Marfan syndrome, a rare connective tissue disease which as no cure to date.

19 May 2025

New gene editor evoCAST enables precise insertion of complete genes

Ask scientists what gene editing tool is most needed to advance gene therapy, and they'd probably describe a system that's now close to realization in the labs of Samuel Sternberg at Columbia University Vagelos College of Physicians and Surgeons and David Liu at the Broad Institute of MIT and Harvard.

15 May 2025

Customized CRISPR gene editing therapy successfully treats infant with rare disease

In a historic medical breakthrough, a child diagnosed with a rare genetic disorder has been successfully treated with a customized CRISPR gene editing therapy by a team at Children's Hospital of Philadelphia (CHOP) and Penn Medicine.

15 May 2025

AAN releases Evidence in Focus Article on new gene therapy for Duchenne muscular dystrophy

To help neurologists, clinicians and families understand the current evidence for a new gene therapy for Duchenne muscular dystrophy called delandistrogene moxeparvovec, the American Academy of Neurology (AAN) has issued an Evidence in Focus article, published May 14, 2025, online in Neurology.

14 May 2025

Existing therapies may help control chronic muscle inflammation in Duchenne muscular dystrophy

A new study led by researchers at Hospital for Special Surgery (HSS) suggests that currently available therapies may help control chronic muscle inflammation in Duchenne muscular dystrophy (DMD), a severe condition that leads to muscle weakness and premature death.

30 Apr 2025

Clusterin identified as key marker for aging in blood stem cells

In recent years, there has been a growing trend of an aging patient population and an increasing prevalence of age-associated diseases, underscoring the need for advancing research into the biological mechanisms of aging.

30 Apr 2025

Nectin4-targeted VHH-CAR-T cells show potency against bladder cancer

Announcing a new article publication for BIO Integration journal. Bladder cancer (BLCA) is one of the most common malignancies and the second most frequent urogenital tract tumor.

29 Apr 2025

Gene therapy offers hope for children born with metachromatic leukodystrophy

If administered early, gene therapy has the potential to change the medical history of children born with metachromatic leukodystrophy (MLD), a rare and lethal neurodegenerative disease of genetic origin which leads to the progressive loss of the ability to walk, talk and interact.

25 Apr 2025