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.

Innovative genome editing approach could be the key to lifelong effective weight management

Weight-loss drugs have surged in popularity, promising rapid results with regular injections. Now, researchers from Japan report a way for the body to make its own weight-loss drugs, doing away with injections in favor of a one-time treatment.

9 Jul 2025

Chemist awarded NSF grant to study DNA nanoparticles for gene therapy

Case Western Reserve University chemist Divita Mathur was awarded a National Science Foundation (NSF) Faculty Early Career Development Program (CAREER) grant for her research in synthetic DNA nanoparticles, which have potential applications in gene therapy.

4 Jul 2025

Gene therapy brings back hearing in young and adult deaf patients

Gene therapy can improve hearing in children and adults with congenital deafness or severe hearing impairment, a new study involving researchers at Karolinska Institutet reports.

3 Jul 2025

Study unveils powerful strategy to rejuvenate effectiveness of CAR T cell therapy against glioblastoma

A team of researchers from the San Raffaele-Telethon Institute for Gene Therapy (SR-TIGET, Milan), led by Nadia Coltella and Luigi Naldini, has unveiled a powerful strategy to rejuvenate the effectiveness of chimeric antigen receptor (CAR) T cell therapy against glioblastoma, one of the most lethal and treatment-resistant brain tumors.

3 Jul 2025

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

Gene Therapy News and Research

Further Reading

Innovative genome editing approach could be the key to lifelong effective weight management

Chemist awarded NSF grant to study DNA nanoparticles for gene therapy

Gene therapy brings back hearing in young and adult deaf patients

Study unveils powerful strategy to rejuvenate effectiveness of CAR T cell therapy against glioblastoma

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

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

NANOSPRESSO device brings precision medicine to hospital pharmacies

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

Driving breakthroughs for the NF community at the 2025 NF Conference

New gene therapy strategy reactivates dormant genes to treat blood disorders

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

New nanoparticle platform breaks through MRSA biofilms

Gene therapy shows long-term success in hemophilia B patients

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

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

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

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

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

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

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

Ultimate guide to the essential tools for the characterization of proteins, polymers and nanoparticles eBook

The Critical Role of Sample Storage in Drug Discovery and Development

Advancing Neuroscience Research Through Collaboration and Patient-Centered Innovation

The impact of vibration on microscopy

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