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.

TET2 mutations in blood stem cells linked to lower Alzheimer's risk

A study published in Cell Stem Cell reveals that some mutations in blood stem cells might help protect against late-onset Alzheimer's disease.

2 Jul 2025

Gene therapy shows promise for reducing stroke risk in patients with sickle cell disease

Gene therapy for sickle cell disease may help improve a major contributing factor to stroke risk in patients, reports a new study from St. Jude Children's Research Hospital.

30 Jun 2025

NANOSPRESSO device brings precision medicine to hospital pharmacies

A new gene therapy delivery device could let hospital pharmacies make personalized nanomedicines to order.

26 Jun 2025

Genetically engineered skin grafts help heal chronic wounds in patients with epidermolysis bullosa

Skin grafts genetically engineered from a patient's own cells can heal persistent wounds in people with an extremely painful dermatologic disease, a Stanford Medicine-led clinical trial has shown.

23 Jun 2025

Driving breakthroughs for the NF community at the 2025 NF Conference

A new era is unfolding for the NF community. With two FDA-approved treatments now on the market - including the first-ever therapy for adults with NF1 - and cutting-edge advances in AI, drug repositioning, and precision medicine, momentum is building like never before.

20 Jun 2025

New gene therapy strategy reactivates dormant genes to treat blood disorders

Researchers have found a promising new method for gene therapy. They successfully restarted inactive genes by bringing them closer to genetic switches on the DNA called enhancers.

19 Jun 2025

Mass photometry recognized by U.S. Pharmacopeia in new AAV reference standards for gene therapy quality

Refeyn, the company behind pioneering mass photometry technology, announces that mass photometry has been recognized by the U.S. Pharmacopeia (USP)—and referenced in the new draft USP general chapter <1067>—as a key orthogonal method for characterizing USP's new adeno-associated virus (AAV) reference standards.

19 Jun 2025

New nanoparticle platform breaks through MRSA biofilms

A major cause of hospital-acquired infections, the super bacteria Methicillin-resistant Staphylococcus aureus (MRSA), not only exhibits strong resistance to existing antibiotics but also forms a dense biofilm that blocks the effects of external treatments.

14 Jun 2025

Gene therapy shows long-term success in hemophilia B patients

A gene transfer approach to treating the bleeding disorder hemophilia B remains safe and effective long-term, as scientists from St. Jude Children's Research Hospital and University College London today report thirteen years of follow-up data.

12 Jun 2025

New CRISPR tool enables precise chemical modification of RNA in living cells

CRISPR-Cas13, a powerful RNA-targeting technology is gaining increasing attention as a next-generation gene therapy platform due to its precision and reduced side effects.

12 Jun 2025

Alzheimer's gene therapy could help protect the brain from damage and preserve cognitive function

Researchers at University of California San Diego School of Medicine have developed a gene therapy for Alzheimer's disease that could help protect the brain from damage and preserve cognitive function.

5 Jun 2025

Gene editing can trigger inflammatory, senescence-like responses in blood stem cells

Scientists at the San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), Milan, have found that gene editing using CRISPR-Cas9 in combination with AAV6 vectors can trigger inflammatory and senescence-like responses in blood stem cells, compromising their long-term ability to regenerate the blood system.

5 Jun 2025

FDA approves ENCELTO implant to preserve sight in people with macular telangiectasia type 2

For people diagnosed with macular telangiectasia type 2 (MacTel)-a rare and slowly progressing retinal disease-there have been no FDA-approved (or other) treatments to slow or stop vision loss.

4 Jun 2025

Study uncovers how HSV-1 disables the brain's antiviral defense

Infections caused by herpes simplex virus type 1 (HSV-1) can lead to HSV-1 encephalitis-a rare but deadly condition that inflames the brain.

3 Jun 2025

Engineered viruses and gene therapy halt tumor growth and extend survival in mice

This study reveals the potential of engineered phage-IL-12 therapy in melanoma, enhancing immune response and tumor destruction in aggressive cancer models.

2 Jun 2025

Non-drug therapies show real promise in calming agitation in Alzheimer’s

Non-drug therapies like music and light outperform medications in managing dementia-related agitation, enhancing patient care and safety for caregivers.

28 May 2025

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