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.

UCLA study shows stem cells can be reprogrammed to fight cancer

In a first-of-its-kind clinical trial, UCLA scientists have shown it's possible to reprogram a patient's blood-forming stem cells to generate a continuous supply of functional T cells, the immune system's most powerful cancer-killing agents.

4 Aug 2025

Unlocking the power of stereochemistry to improve mRNA drug delivery

A team from the Max-Planck-Institut für Kohlenforschung, Hokkaido University, and Osaka University has discovered that subtle differences in molecular structure can have a major impact on the performance of mRNA-based drugs.

1 Aug 2025

Virtual memory T cells provide nonspecific immunity for infants early in life

Findings from St. Jude Children's Research Hospital demonstrate that virtual memory T cells, a specialized group of immune cells, provide nonspecific immunity for infants early in life.

31 Jul 2025

Single injection of gene therapy at birth may offer lasting protection against HIV

A new study in Nature shows that delivering a single injection of gene therapy at birth may offer years-long protection against HIV, tapping into a critical window in early life that could reshape the fight against pediatric infections in high-risk regions.

30 Jul 2025

Researchers classify how specific genetic mutations correspond to rare disorders

The NF-κB signaling pathway plays a pivotal role in how our bodies fight infection, heal, and regulate inflammation. At the heart of this pathway is the NEMO protein, encoded by the IKBKG gene.

30 Jul 2025

New RNA therapy reverses symptoms of progeria in mouse models

Children who develop deep wrinkles, stunted growth, and rapidly aging bones and blood vessels as early as 1 to 2 years of age may be suffering from Hutchinson-Gilford Progeria Syndrome (HGPS), a rare and incurable genetic disorder that affects approximately one in eight million people.

29 Jul 2025

Reactivating silent genes offers hope for Rett syndrome treatment

Researchers led by UC Davis Health scientist Sanchita Bhatnagar have developed a promising gene therapy that could treat Rett syndrome.

23 Jul 2025

UC Davis researchers pioneer safer gene therapy for vision loss

The UC Davis Department of Ophthalmology has received a five-year, $3.6 million grant from the National Eye Institute to explore a new way to treat vision loss using gene therapy.

22 Jul 2025

Breakthrough gene therapy reverses genetic deafness across age groups

Using gene therapy to treat hereditary deafness is safe and effective in both children and adults, according to new findings from a multicenter clinical trial.

22 Jul 2025

Prime editing reverses symptoms of severe childhood neurological disease

Scientists use a precise form of gene editing called prime editing to correct the most common genetic mutations that cause alternating hemiplegia of childhood, a rare and severe neurological disorder that begins in infancy.

21 Jul 2025

Gene editing in the brain offers hope for ultra-rare neurological disease

Scientists have corrected gene mutations in mice causing an ultra-rare disease by editing DNA directly in the brain with a single injection, a feat with profound implications for patients with neurological diseases.

21 Jul 2025

Emerging therapies reshape the future of AMD treatment

Age-related macular degeneration (AMD) remains a leading cause of vision loss globally, with significant advancements in treatment options for both dry and wet AMD.

18 Jul 2025

Harvard researchers uncover how the brain directs blood flow in real time

All day long, our brains carry out complicated and energy-intensive tasks such as remembering, solving problems, and making decisions.

16 Jul 2025

New UAB-developed gene therapy for ALS receives orphan drug designation

The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to a new gene therapy for Amyotrophic Lateral Sclerosis (ALS) developed at the Universitat Autònoma de Barcelona and licensed to the U.S. company Klotho Neurosciences, Inc.

15 Jul 2025

New cellular entry pathway improves gene therapy outcomes

Scientists from the Centenary Institute and the University of Sydney have made a landmark discovery that could lead to safer and more effective gene therapies for a range of serious genetic disorders including Duchenne muscular dystrophy, Pompe disease and hemophilia.

15 Jul 2025

Sino Biological sets new industry standard with ProPure™ endotoxin-free proteins made in the USA

Sino Biological US, Inc. proudly unveils ProPure™, an industry-leading line of ultra-pure, endotoxin-free recombinant proteins, fully produced in the USA at its state-of-the-art Center for Bioprocessing (C4B) facility in Houston, Texas.

From Sino Biological Inc. 14 Jul 2025

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