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.

Researchers discover regulatory map of the human EphB4 gene

Pierluigi Scalia and coworkers at the Sbarro Institute for Cancer Research and Molecular Medicine at Temple University in Philadelphia, and the affiliated Italian research organization ISOPROG, have announced the regulatory map of the human EphB4 gene.

3 Dec 2019

Sheffield research receives £800k to develop new gene therapy to treat CVD

Cardiac research in Sheffield has received a major boost from the British Heart Foundation in the form of an £800,000 award to develop a new gene therapy to treat cardiovascular disease (CVD).

26 Nov 2019

New partnership aims to explore, cultivate innovations in cell and gene therapy

Some patients who have not responded to traditional medicines are now experiencing remarkable recoveries thanks to next-generation immunotherapies. These therapies equip a patient's own immune cells to recognize, target, and destroy cancer cells.

25 Nov 2019

University of Sheffield receives Queen's Anniversary Prize for innovation in neuroscience

Neuroscience research at the University of Sheffield has been recognized by Her Majesty The Queen for delivering real benefits in improving patient outcomes for people living with some of the most devastating neurodegenerative diseases.

22 Nov 2019

Tailored T-cells safe, may be effective in preventing and treating multiple viral infections

Tailored T-cells specially designed to combat a half dozen viruses are safe and may be effective in preventing and treating multiple viral infections, according to research led by Children's National Hospital faculty.

18 Nov 2019

Improved gene editing technique can successfully cut out undesirable genetic information

City of Hope researchers may have found a way to sharpen the fastest, cheapest and most accurate gene editing technique, CRISPR-Cas9, so that it can more successfully cut out undesirable genetic information.

7 Nov 2019

CRISPR-Cas9 protein can help increase targeting accuracy in genome editing process

A team of researchers from City University of Hong Kong and Karolinska Institutet has recently developed a new protein that can help increase the targeting accuracy in the genome editing process. It is believed that it would be useful for future gene therapies in human, which require high precision.

6 Nov 2019

Newly identified gene variant may offer potential treatment for Alzheimer's disease

Defying the odds, an individual at high risk for early-onset Alzheimer's disease remained dementia-free for many years beyond what was anticipated.

4 Nov 2019

Researchers develop new type of transposase protein with improved biochemical properties

The team, comprising researchers from the European Molecular Biology Laboratory, the Universitätsklinikum Würzburg and the Paul-Ehrlich-Institut, managed to design a new variant of the Sleeping Beauty transposase with dramatically improved biochemical properties.

4 Nov 2019

Three UCLA researchers receive multimillion-dollar grant from state's stem cell agency

Three researchers at the Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research at UCLA have received awards totaling more than $18 million from the California Institute for Regenerative Medicine, the state's stem cell agency.

31 Oct 2019

OHSU receives $2.1 million to develop new vaccine additives for tropical viruses

Sometimes vaccines need a little help to protect people against infectious diseases.

31 Oct 2019

Study identifies a kinase as potential target to treat heart failure

An unexplored kinase in heart muscle cells may be a good target to treat heart failure, a disease that is only incrementally delayed by existing therapies.

31 Oct 2019

Researchers develop new therapeutic concept for treatment of temporal lobe epilepsy

Teams of researchers from Charité - Universitätsmedizin Berlin and the Medical University of Innsbruck have developed a new therapeutic concept for the treatment of temporal lobe epilepsy.

30 Oct 2019

New collaborative effort focuses on therapeutic research for CDKL5 Deficiency Disorder

CDKL5 Deficiency Disorder is a devastating neurodevelopmental disorder caused by mutations in the CDKL5 gene.

30 Oct 2019

Researchers make progress toward gene-based strategies to treat inherited neurometabolic diseases

Researchers are making great strides toward developing gene-based strategies to treat a variety of inherited neurometabolic diseases characterized by severe neurological involvement.

29 Oct 2019

Gut microbiome altered by burn injury and influenced by fluid resuscitation, study shows

Major burns lead to changes in the gastrointestinal tract bacteria, and these alterations of the gut microbiome are influenced by resuscitation with intravenous (IV) fluids, according to animal studies reported in SHOCK: Injury, Inflammation, and Sepsis: Laboratory and Clinical Approaches, Official Journal of theShockSociety. The journal is published in the Lippincott portfolio by Wolters Kluwer.

25 Oct 2019

Discovery in monkeys could pave way for treatment of Bardet-Biedl Syndrome

A genetic mutation that leads to a rare, but devastating blindness-causing syndrome has been discovered in monkeys for the first time. The finding offers a promising way to develop gene and cell therapies that could treat the condition in people.

25 Oct 2019

New NPO formed to accelerate development of therapies for Shankopathies

Parents of children affected by Phelan-McDermid syndrome (PMS) announce the formation of CureSHANK, a nonprofit organization on a mission to accelerate drug development in PMS and other diseases related to the SHANK genes for which there are currently no FDA approved treatments.

23 Oct 2019

New collaboration aims to develop gene-based cures for sickle cell disease and HIV

The National Institutes of Health plans to invest at least $100 million over the next four years toward an audacious goal: develop affordable, gene-based cures for sickle cell disease and HIV.

23 Oct 2019

New research center will encompass a myriad of muscle science and disease investigations

With collaborating labs across the University of Washington campus and at other Seattle-area institutions and beyond, the Center for Translational Muscle Research will encompass a myriad of muscle science and disease investigations.

17 Oct 2019

Gene Therapy News and Research

Further Reading

Researchers discover regulatory map of the human EphB4 gene

Sheffield research receives £800k to develop new gene therapy to treat CVD

New partnership aims to explore, cultivate innovations in cell and gene therapy

University of Sheffield receives Queen's Anniversary Prize for innovation in neuroscience

Tailored T-cells safe, may be effective in preventing and treating multiple viral infections

Improved gene editing technique can successfully cut out undesirable genetic information

CRISPR-Cas9 protein can help increase targeting accuracy in genome editing process

Newly identified gene variant may offer potential treatment for Alzheimer's disease

Researchers develop new type of transposase protein with improved biochemical properties

Three UCLA researchers receive multimillion-dollar grant from state's stem cell agency

OHSU receives $2.1 million to develop new vaccine additives for tropical viruses

Study identifies a kinase as potential target to treat heart failure

Researchers develop new therapeutic concept for treatment of temporal lobe epilepsy

New collaborative effort focuses on therapeutic research for CDKL5 Deficiency Disorder

Researchers make progress toward gene-based strategies to treat inherited neurometabolic diseases

Gut microbiome altered by burn injury and influenced by fluid resuscitation, study shows

Discovery in monkeys could pave way for treatment of Bardet-Biedl Syndrome

New NPO formed to accelerate development of therapies for Shankopathies

New collaboration aims to develop gene-based cures for sickle cell disease and HIV

New research center will encompass a myriad of muscle science and disease investigations

Leveraging the power of automation to boost research

Automating research - solutions and best practices

How can microdialysis benefit drug development

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