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.

Pioneering the world’s first CRISPR medicine for sickle cell disease

When Vijay Sankaran was an MD-PhD student at Harvard Medical School in the mid-2000s, one of his first clinical encounters was with a 24-year-old patient whose sickle cell disease left them with almost weekly pain episodes.

3 Mar 2025

Study demonstrates successful gene therapy for maple syrup urine disease

A study led by UMass Chan researchers demonstrated that a gene therapy to correct a mutation that causes maple syrup urine disease (MSUD) prevented newborn death, normalized growth, restored coordinated expression of the affected genes and stabilized biomarkers in a calf as well as in mice.

28 Feb 2025

New deep-learning framework improves spatial transcriptomics analysis

Biological tissues are made up of different cell types arranged in specific patterns, which are essential to their proper functioning.

27 Feb 2025

New gene therapy strategy could cure COPA Syndrome

Scientists compared five families and multiple generations to find a gene variant that prevents COPA Syndrome and opens the door to a new gene therapy for the condition.

27 Feb 2025

New genetic medicine offers life-changing vision improvements for blind children

Four young children have gained life-changing improvements in sight following treatment with a pioneering new genetic medicine through UCL Institute of Ophthalmology and Moorfields Eye Hospital, with the support of MeiraGTx.

22 Feb 2025

Antioxidant-enzyme interactions and their role in non-communicable diseases

Oxidative stress, caused by an imbalance between reactive oxygen species (ROS) and antioxidant defenses, plays a central role in the development of non-communicable diseases (NCDs).

21 Feb 2025

New robotic surgery device aims to enhance precision in retinal procedures

When even the most highly trained surgeons perform procedures on the retina-one of the smallest, most delicate parts of the human body-the stakes are high.

21 Feb 2025

Inhalable gene therapy tested for cystic fibrosis treatment in the UK and Europe

An inhalable medicine with the potential to improve lung disease in people with cystic fibrosis, irrespective of their mutation type, is being tested in human trials in the UK and Europe.

20 Feb 2025

Breakthrough in gene therapy could help protect against heart arrhythmias

Cardiac arrhythmias affect millions across the world and are responsible for a fifth of all deaths in the Netherlands.

20 Feb 2025

Gene therapy breakthrough offers hope for severe Dravet syndrome cases

AAV-Navβ1 gene therapy effectively reduces seizures and prolongs life in a mouse model of SCN1B-linked Dravet syndrome, paving the way for future treatments.

18 Feb 2025

Breakthrough technology enables mRNA delivery across the blood-brain barrier

Scientists at the Icahn School of Medicine at Mount Sinai have developed a lipid nanoparticle system capable of delivering messenger RNA (mRNA) to the brain via intravenous injection, a challenge that has long been limited by the protective nature of the blood-brain barrier.

17 Feb 2025

Gene therapy shows promise in treating SCN1B-linked epileptic encephalopathies

Dravet syndrome and other developmental epileptic encephalopathies are rare but devastating conditions that cause a host of symptoms in children, including seizures, intellectual disability, and even sudden death.

14 Feb 2025

One shot to lower cholesterol for life? Scientists unlock a groundbreaking gene therapy

Researchers developed an epigenetic editing approach to silence the PCSK9 gene, achieving long-term cholesterol reduction without altering DNA, offering a potential one-time treatment for high cholesterol.

11 Feb 2025

Research highlights gender and age differences in eye gene therapy responses

Older women could be vulnerable to harmful inflammation from new gene therapies to treat incurable eye diseases, new research has found.

10 Feb 2025

Gene therapy developments and obstacles for neurologic disorders

Announcing a new article publication for BIO Integration journal. Neurologic disorders currently affect approximately 100 million people worldwide.

8 Feb 2025

UConn receives $400,000 grant for cutting-edge autism research

Just in time for the Super Bowl, researchers at UConn School of Medicine are part of a big win for autism research.

7 Feb 2025

MaxCyte® acquires SeQure Dx to broaden cell engineering offerings with on-target and off-target editing assessments

MaxCyte, Inc., a leading, cell-engineering focused company providing solutions to advance the discovery, development and commercialization of next-generation cell therapeutics announced today the acquisition of SeQure Dx, a market leader of on-target and off-target editing assessment services for cell and gene therapies.

From MaxCyte, Inc. 6 Feb 2025

New gene therapy shows promise for lifelong treatment of hypophosphatasia

For the last 10 years, the only effective treatment for hypophosphatasia (HPP) has been an enzyme replacement therapy that must be delivered by injection three-to-six times each week.

4 Feb 2025

USC receives $6 million grant for pioneering glioblastoma gene therapy

The California Institute for Regenerative Medicine has awarded a $6 million grant to USC investigators pioneering a new first-of-its-kind genetic therapy for glioblastoma, a severe form of brain cancer.

3 Feb 2025

New study confirms CAR T cell therapy does not cause secondary cancers

Within a large group of more than 700 patients treated with CAR T cell therapy, researchers found no evidence that the therapy itself caused any type of secondary cancer in the modified T cells, according to new analysis reported today in Nature Medicine from the Perelman School of Medicine at the University of Pennsylvania and Penn Medicine's Abramson Cancer Center.

3 Feb 2025