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.

Study shows beneficial effects of gene therapy can be observed even after several decades

A new study shows that the advantageous effects of gene therapy can be observed several decades after the body clears the transplanted blood stem cells.

18 Mar 2021

Study shows how human viruses are better adapted to infect certain tissues

The world’s first systematic study has explained how SARS-CoV-2 and other human viruses are more adapted to infect specific types of tissues..

17 Mar 2021

First-in-human clinical trial to test safety, efficacy of gene therapy for Alzheimer's disease

Researchers at University of California San Diego School of Medicine have launched a first-in-human Phase I clinical trial to assess the safety and efficacy of a gene therapy to deliver a key protein into the brains of persons with Alzheimer's disease (AD) or Mild Cognitive Impairment (MCI), a condition that often precedes full-blown dementia.

19 Feb 2021

New understanding of tumor microenvironment could help enhance immunotherapy

A paper published today in Nature shows how chemicals in the areas surrounding tumors--known as the tumor microenvironment--subvert the immune system and enable cancer to evade attack. These findings suggest that an existing drug could boost cancer immunotherapy.

16 Feb 2021

New AI-based method produces new diversity of AAV capsids for optimized gene therapy

Dyno Therapeutics has demonstrated the use of AI to created an unparalleled range of adeno-associated virus capsids that can evade the immune system.

15 Feb 2021

Severe genetic immunodeficiency reversed by a rare DNA change

Researchers have revealed how a rare DNA change rebalanced the immune system of patients with a life-threatening genetic immunodeficiency.

2 Feb 2021

Gene therapy helps paralyzed mice to walk again

For the first time, scientists have successfully used gene therapy to make mice walk again after these animals suffered a complete cross-sectional injury.

18 Jan 2021

Innovative gene therapy approach for regeneration of functional new neurons

Spinal cord injury (SCI) often causes disability and seriously compromises quality of life. While decades of research have made significant progress in axonal regeneration after SCI, most of the interventions have not been translated into clinical therapies.

18 Dec 2020

New gene therapy approach can improve uterine blood flow in placental insufficiency

A new study identified an adenovirus gene therapy vector carrying a VEGF isoform. It can improve uterine blood flow in placental insufficiency, as reported in the peer-reviewed journal Human Gene Therapy.

16 Dec 2020

Gene therapy and medication can provide certain beneficial effects of physical exercise

Jose Bianco Moreira from NTNU is now convinced that gene therapy can be used to achieve the positive health benefits of physical exercise.

7 Dec 2020

A Gene Mutation That Protects Against Disease

Researchers think they have found a fountain of youth, and it is unique to a few French Canadian families.

3 Dec 2020

New gene therapy can protect visual function in patients with dominant optic atrophy

Researchers have created a novel gene therapy approach that shows promise for someday treating an eye disease that results in progressive vision loss.

27 Nov 2020

Researchers use metal-organic frameworks to target prostate cancer cells

Scientists have used metal-organic frameworks to effectively deliver the CRISPR/Cas9 genetic snipping tool into human cancer cells.

23 Nov 2020

Researchers identify rare genetic marker that protects against cardiovascular disease

According to researchers from McMaster University and the Montreal Clinical Research Institute, they have identified a “fountain of youth” in a rare genetic marker that is unique to a few French-Canadian families.

20 Nov 2020

Somatic mutation acts as natural gene therapy in patients with GATA2 deficiency

Researchers affiliated with the Center for Cell-Based Therapy (CTC) in Ribeirão Preto, Brazil, have identified for the first time a non-hereditary mutation in blood cells from a patient with GATA2 deficiency, a rare autosomal disease caused by inherited mutations in the gene that encodes GATA-binding protein 2 (GATA2).

18 Nov 2020

Light-activated liposomes could help deliver CRISPR gene therapy

Light-activated liposomes could help to deliver CRISPR gene therapy - and the method could prove safer and more direct than current methods.

16 Nov 2020

New approach can prevent toxicity linked to neurological gene therapy

Scientists have deigned a new method to inhibit a toxicity observed in the sensory neurons of DRG following gene therapy to treat neurological diseases.

12 Nov 2020

Dietary induction of hepatocyte regeneration could help enhance gene repair in the liver

The thyroid hormone used to promote the proliferation of hepatocytes improved the efficiency of CRISPR/Cas9-mediated gene repair in the mouse liver.

11 Nov 2020

Research projects receive BTCA awards to develop new drugs

Five research projects with exceptional promise to deliver new life-changing and health-altering therapies have received the inaugural Blavatnik Therapeutics Challenge Awards (BTCA) at Harvard Medical School.

30 Oct 2020

New DNA-cutting tool could advance gene editing for novel treatments

Gene editing for the development of new treatments, and for studying disease as well as normal function in humans and other organisms, may advance more quickly with a new tool for cutting larger pieces of DNA out of a cell's genome, according to a new study by UC San Francisco scientists.

21 Oct 2020