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.

Protein marker detects early Huntington's disease before brain damage

Before motor or cognitive impairments appear and before brain volume loss becomes evident, some individuals carrying the genetic mutation that causes Huntington's disease already show signs of neuronal injury.

10 Nov 2026

New mRNA therapy reawakens heart muscle cells to repair damage

Scientists have developed a treatment that 'reawakens' heart muscle cells so they start to divide and help repair damaged muscle.

9 Nov 2026

Gene therapy successfully corrects genetic defect in alpha-1 antitrypsin deficiency

In a landmark achievement in the field of genetic medicine, researchers have reported the very first published case in a peer-reviewed journal of a successful clinical treatment of a patient with alpha-1 antitrypsin deficiency (PiZZ) using in vivo gene therapy.

2 Nov 2026

$1 million grant advances research into RNA-DNA virus evolution

Much remains unknown about the origin of viruses and how they evolve - but hunting for answers has been the focus of Portland State biology professor Ken Stedman's eXtreme Virus Lab for the past 25 years.

27 Oct 2026

Baylor researchers discover how glioblastoma hijacks nearby brain cells

Researchers at Baylor College of Medicine have discovered a new strategy that glioblastoma, the most common and aggressive form of adult brain cancer, uses to fuel its own growth.

27 Oct 2026

Proteins linked to Alzheimer's found to assist sperm cells

Unexpectedly, a kind of protein that is usually linked with diseases in the brain may play a vital role in semen, helping shepherd genetic material into sperm cells, scientists at Columbia's Zuckerman Institute report today in the research journal Molecular Cell.

26 Oct 2026

Dual-targeted therapy shows promise against metastatic triple-negative breast cancer

A new preclinical study suggests that attacking triple-negative breast cancer (TNBC) in two ways at once can kill cancer cells, slow tumor growth and limit the cancer's spread. Researchers at The Ohio State University Comprehensive Cancer Center – Arthur G. James Cancer Hospital and Richard J. Solove Research Institute (OSUCCC – James) developed the approach.

23 Oct 2026

Touchlight announces leadership transition, with Will Downie appointed interim CEO

Touchlight today announces that Karen Fallen is stepping down as CEO and that the Board of Directors has appointed Will Downie as Interim Chief Executive Officer.

From Touchlight 21 Oct 2026

Experts weigh risks of using gene therapies for common diseases

Gene therapy is no longer the future of medicine - it's reality. Treatments that replace, silence or edit genes are already changing clinical care.

20 Oct 2026

Researchers investigate molecular mechanisms linking high-calorie diets to cognitive decline

The molecular mechanisms linking a high calorie diet to cognitive impairment remain unclear. Researchers at Institute of Science Tokyo, Japan, investigated this association in mice, finding that the diet reduced nuclear protein PQBP1 expression, disrupting the alternative splicing of genes involved in synaptic function.

20 Oct 2026

Experimental gene therapy offers non-surgical option for hydrocephalus

Researchers at the University of Cincinnati College of Medicine have published the first proof-of-concept study showing the potential of an experimental gene therapy for hydrocephalus.

14 Oct 2026

Novel gene rejuvenation therapy preserves cell identity while reversing age

Every organ and tissue progressively loses function and integrity as it ages. In fact, researchers have identified "hallmarks of aging" that not only present specific processes impacted by age, but are also thought of as levers that, if successfully targeted, could help turn back the aging clock and result in tissue rejuvenation.

14 Oct 2026

$5.8 million study explores gut-brain inflammation in people with HIV

Oregon Health & Science University researchers have secured a $5.8 million, five-year federal award to investigate whether damage to the gastrointestinal tract contributes to neuroinflammation in people living with HIV - raising the potential for new treatments to preserve brain function in people with HIV and older adults.

13 Oct 2026

Gene therapy and special goggles help some blind adults detect objects again

In a phase 1 study of 10 adults with advanced retinitis pigmentosa, researchers found that optogenetic gene therapy combined with light-stimulating goggles was generally well tolerated, although one participant experienced a severe but transient ocular adverse event. Seven participants showed improved light sensitivity, while four of eight who completed behavioral testing demonstrated modest gains in visual function, providing preliminary evidence of partial vision restoration that requires confirmation in larger controlled trials.

12 Oct 2026