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.

Gene therapy shows promise against deadly childhood liver disease

A new gene therapy has been used to successfully treat a deadly childhood liver disease using mice that model this disease by researchers at UCL and Great Ormond Street Hospital.

23 Jun 2026

Gene therapy successfully treats deadly childhood liver disease in mice

A new gene therapy has been used to successfully treat a deadly childhood liver disease using mice that model this disease by researchers at UCL and Great Ormond Street Hospital.

19 Jun 2026

Insilico Medicine to showcase AI-driven drug breakthroughs at BIO 2026 International Convention

Insilico Medicine will attend the BIO 2026 International Convention, held from June 22 to 25 at the San Diego Convention Center.

18 Jun 2026

Novel gene therapy appears to be safe for Friedreich ataxia cardiomyopathy

A new gene therapy appears to be safe in patients diagnosed with Friedreich ataxia cardiomyopathy, a progressive and fatal inherited cardiac disease, according to a phase 1 clinical trial led by Weill Cornell Medicine researchers.

17 Jun 2026

Salt-loaded nanoparticles help deliver fragile gene therapies into cells

Researchers at the University of Houston's College of Pharmacy have discovered an unexpected simple strategy to improve the performance of mRNA vaccines and gene therapeutics: adding salt.

16 Jun 2026

Single-dose gene therapy extends healthy lifespan in older mice

A research team from the Universitat Autònoma de Barcelona (UAB) has shown that a one-time administration of a gene therapy expressing the metabolic factor FGF21 can prolong health span in old mice.

15 Jun 2026

Renowned physician‑scientist to lead Houston Methodist's cell and gene therapy research

Malcolm Brenner, an internationally recognized physician‑scientist renowned for his leadership in cell and gene therapy, has been tapped to lead the newly formed Houston Methodist Center for Cell and Gene Therapy.

12 Jun 2026

Amsterdam UMC researchers overturn long-standing biological pacemaker dogma

Researchers from Amsterdam UMC have overturned a key assumption in the biological pacemaker field.

11 Jun 2026

New treatment platform delivers full-length mRNA for Duchenne muscular dystrophy

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 preclinical models of Duchenne muscular dystrophy, successfully restoring the production of an important muscle protein, dystrophin, and dramatically improving muscle strength, endurance and function in vivo.

11 Jun 2026

First-in-the-world gene therapy delivers missing gene directly to infant’s brain, marking historic milestone in precision medicine

An eight-month-old baby from Israel has become the first human ever to receive an experimental gene therapy designed to replace a missing gene responsible for a devastating neurological disease that until now offered no hope of survival.

11 Jun 2026

Low-methionine diet shows promise against aggressive brain tumors

An unexpected lab observation led a team of scientists to discover how diet can influence survival in animal models of glioma, one of the most aggressive and deadly forms of brain cancer.

10 Jun 2026

Silver nanoparticles enable precise DNA cutting and assembly

DNA is composed of long chains that act as the blueprint for living organisms. In genetic engineering, scientists cut DNA at specific sites and join the resulting fragments to other DNA sequences, enabling applications such as advanced crop breeding, genetic disease treatment, and the generation of animal models for drug discovery.

10 Jun 2026

Laverock Therapeutics reports key oncology research milestones

Laverock Therapeutics ('Laverock'), a biotechnology company developing disease-responsive advanced therapies through its unique, programmable gene control technology, today announced key in-vivo functional milestones across its T-cell and macrophage oncology programs for solid tumor indications.

From Laverock Therapeutics 10 Jun 2026

Scientists use inactive virus to safe-deliver spasticity-reversing spinal genes

Muscle spasticity is a common and often debilitating consequence of spinal cord injury that can significantly affect mobility and quality of life. Spasticity is a condition in which muscles become abnormally stiff or tight because of disrupted nerve signaling, often causing exaggerated reflexes, involuntary muscle spasms and difficulty with movement.

9 Jun 2026

Infant receives world's first gene therapy for WOREE syndrome

An eight-month-old infant with a severe genetic epilepsy has become the first patient in the world to receive an experimental gene replacement therapy designed to restore the function of the WWOX gene directly in the brain.

8 Jun 2026

Stem cell transplantation is the most cost-effective sickle cell treatment

Stem cell transplantation is the most cost-effective option for long-term care of sickle cell disease in adults when compared with gene therapy and standard of care treatment, according to new research published in Blood.

2 Jun 2026

New ultrasound pacemaker design promises minimal recovery time for cardiac patients

MIT engineers have developed a noninvasive pacemaker that stimulates the heart using ultrasound. The design could one day provide a surgery-free alternative to traditional cardiac implants.

2 Jun 2026

Pediatric experts reveal the 10 research areas set to shape child health

Pediatric research leaders identified 10 high-promise areas likely to shape child health over the next 5 to 10 years, including genomics, mental health, vaccines, artificial intelligence, perinatal medicine, precision therapeutics, health equity, nutrition, environmental health, and cancer treatment. The paper argues that these opportunities will only translate into better outcomes if pediatric research receives sustained funding, public trust, workforce support, advocacy, and equitable implementation.

1 Jun 2026

New SMArT platform improves safety of CRISPR gene editing

A team of researchers led by Luigi Naldini at the San Raffaele Telethon Institute for Gene Therapy (SR-Tiget) has developed a new strategy to significantly improve the precision and safety of CRISPR-Cas9 gene editing in human blood stem cells, potentially overcoming one of the major barriers limiting broader clinical application of genome editing therapies.

1 Jun 2026

Hypoxia-gated AAV enables targeted treatment for advanced NSCLC

Adeno‑associated virus (AAV) vectors offer safety and durable expression but lack tumor specificity. This hypothesis proposes an NSCLC‑directed, hypoxia‑responsive AAV platform that integrates MGS4 peptide‑guided capsid targeting with dual hypoxia‑responsive element (HRE)‑gated promoters driving Q‑CXCL9‑Fc (chemokine) and miR‑30‑based shRNA against mesothelin (MSLN).

1 Jun 2026