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.

New study assesses total economic impact of human genomics

Human genetics and genomifefcs contributed $265 billion to the U.S. economy in 2019 and has the potential to drive significant further growth given major new areas of application, according to a new report issued today by the American Society of Human Genetics (ASHG).

19 May 2021

Lack of functional GAS2 gene causes severe hearing impairment in mice

A gene called GAS2 plays a key role in normal hearing, and its absence causes severe hearing loss, according to a study led by researchers in the Perelman School of Medicine at the University of Pennsylvania.

10 May 2021

New gene therapy could help treat Duchenne muscular dystrophy

Researchers from UT Southwestern used a new kind of gene therapy to successfully treat mice with Duchenne muscular dystrophy (DMD), distinctively employing CRISPR-Cas9-based tools to restore a large section of the dystrophin protein missing in several DMD patients.

3 May 2021

National DNA Day 2021: An Interview with Professor George Church

In celebration of National DNA Day, AZoLifeSciences interviews renowned DNA expert Professor George Church about his life-long career in DNA research.

From Harvard Medical School 23 Apr 2021

Gene therapy protects eye cells in mice with retinitis pigmentosa

A gene therapy protects eye cells in mice with a rare disorder that causes vision loss, especially when used in combination with other gene therapies, shows a study published today in eLife.

15 Apr 2021

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.

From University College London 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.

From UC San Diego 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.

From University of Pittsburgh 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