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 to develop new stem cell-based strategies for treating vision disorders

A team from the University of Pennsylvania, Children's Hospital of Philadelphia, and University of Wisconsin--Madison are launching a project to develop new strategies for treating vision disorders using cells implanted in the retina.

17 Oct 2018

Researchers develop two-pronged approach for targeting Ebola virus

In a new study researchers have developed a two-pronged approach for targeting Ebola virus infection using linked nucleic acid (LNA) antisense oligonucleotides (ASOs)designed to interfere both genes essential for translation of Ebola virus genes and to block production of an intracellular human protein needed for the virus to enter cells.

12 Oct 2018

Study suggests new approach to treat life-threatening blood disorder in children

Pediatric researchers studying the life-threatening blood disorder Fanconi anemia have devised a method to block the abnormal biological signals that drive the disease.

12 Oct 2018

Lab grown retinal cells provide clues to color blindness treatment

Researchers have grown human retinal cells in the laboratories and thus attempted to understand the basis of color vision and its development in humans. The results showed that certain hormones are essential for the development of color vision in humans and were published in the latest issue of the journal Science.

11 Oct 2018

Research team awarded $3.6 million to assess safety of genome editing

As genome editing technologies quickly advance toward clinical therapies, they are raising hopes of a completely new way to treat disease. They are also bringing to light a number of challenges that need to be addressed before potential treatments can be widely used in patients.

11 Oct 2018

Micropeptide molecule could be promising gene therapy target to treat heart failure

Researchers have discovered a micropeptide molecule that can restore normal heart function in mice, according to a study in eLife.

10 Oct 2018

Bradford and GE Healthcare collaborate to build new AI-powered hospital Command Center in Europe

Bradford Teaching Hospitals NHS Foundation Trust is collaborating with GE Healthcare to build a Command Center - like an air traffic control - at the Bradford Royal Infirmary, UK. A first of its kind in Europe, the Command Center will transform how care is delivered and organized as the number of patients at the hospital continues to increase.

4 Oct 2018

Working with Next Health World Innovation Forum to build a healthier future

The Innovation Forum is partnering with the Milner Therapeutics Institute in 2019 for the Next Health World Innovation Forum – a new international healthcare conference taking place from 26-27 June 2019, in Cambridge, UK.

4 Oct 2018

Study: Reducing mutated Huntington disease protein restores psychiatric impairments in mice

New research from the University of British Columbia suggests that reducing mutated Huntington disease protein in the brain can restore cognitive and psychiatric impairments in mice.

4 Oct 2018

Hopkins gastroenterologists use endoscopic procedure to deliver therapeutic genes to the liver

Fixing or replacing faulty genes has emerged as a key to unlocking cures for numerous devastating diseases. But if the new, engineered genes can't find their way into the patient's genomic sequence, they won't help.

3 Oct 2018

Carnegie professor receives NIH Director's New Innovator Award

Kathryn Whitehead, assistant professor of chemical engineering at Carnegie Mellon University, has been awarded a 2018 National Institutes of Health (NIH) Director's New Innovator Award for her project titled "Fate, Function, and Genetic Engineering of Breast Milk Cells for Infant Therapy."

3 Oct 2018

Your Vision Matters: How to Protect Your Eyesight

Dr. Neil Ebenezer from the charity Fight for Sight discusses the steps you can take to protect your eyesight into old age, and why research into ophthalmic diseases is desperately needed.

24 Sep 2018

Gene therapy approach could help treat mitochondrial diseases

Researchers have developed a genome-editing tool for the potential treatment of mitochondrial diseases: serious and often fatal conditions which affect 1 in 5,000 people.

24 Sep 2018

LineaRx signs agreement with Takis/Evvivax to develop linear-DNA based anti-cancer vaccines

Applied DNA Sciences Inc. announced today that LineaRx, Inc., its wholly-owned subsidiary focused on next-generation biotherapeutics, has signed a Joint Development Agreement with Takis S.R.L. and Evvivax S.R.L., biotechnology companies focused on the discovery and development of DNA based anti-cancer vaccines for the human and animal markets, respectively.

20 Sep 2018

NIH and FDA call for eliminating involvement of RAC in human gene therapy experiments

Recently, the U.S. National Institutes of Health and Food and Drug Administration called for the eliminating involvement of the Recombinant DNA Advisory Committee in human gene therapy experiments, marking the end of an era of federal government oversight

20 Sep 2018

First U.S. patient treated with innovative gene therapy at Bascom Palmer Eye Institute

A Puerto Rican patient with X-linked retinitis pigmentosa (XLRP) is hoping to save his vision after an innovative gene therapy procedure at Bascom Palmer Eye Institute at the University of Miami Miller School of Medicine.

19 Sep 2018

Promising gene therapy for 'day blind' sheep now safe for clinical trials in human patients

Back in 2009, a group of Israeli researchers identified a herd of Awassi sheep suffering from "day blindness". As its name implies, these sheep were blind during the day (in bright light) but could see at night, in low-light conditions.

17 Sep 2018

Previously unknown virus acts as 'driver' for interstitial nephropathy

An international research team led by Wolfgang Weninger, who has been Head of MedUni Vienna's Department of Dermatology since 1 September, has discovered a previously unknown virus that acts as a "driver" for certain kidney diseases (interstitial nephropathy).

14 Sep 2018

Discovering Prion-like Proteins in Eukaryotic Viruses

Dr. George Tetz discusses the discovery of prion-like domains in eukaryotic viruses, and the implications of this study on gene therapies and common neurological diseases such as Alzheimer's Disease.

6 Sep 2018

UCLA Mattel Children's Hospital recognized as one of the most innovative children's hospitals in the U.S.

Parents magazine has named UCLA Mattel Children's Hospital one of the most innovative children's hospitals in the United States.

6 Sep 2018

Gene Therapy News and Research

Further Reading

Researchers to develop new stem cell-based strategies for treating vision disorders

Researchers develop two-pronged approach for targeting Ebola virus

Study suggests new approach to treat life-threatening blood disorder in children

Lab grown retinal cells provide clues to color blindness treatment

Research team awarded $3.6 million to assess safety of genome editing

Micropeptide molecule could be promising gene therapy target to treat heart failure

Bradford and GE Healthcare collaborate to build new AI-powered hospital Command Center in Europe

Working with Next Health World Innovation Forum to build a healthier future

Study: Reducing mutated Huntington disease protein restores psychiatric impairments in mice

Hopkins gastroenterologists use endoscopic procedure to deliver therapeutic genes to the liver

Carnegie professor receives NIH Director's New Innovator Award

Your Vision Matters: How to Protect Your Eyesight

Gene therapy approach could help treat mitochondrial diseases

LineaRx signs agreement with Takis/Evvivax to develop linear-DNA based anti-cancer vaccines

NIH and FDA call for eliminating involvement of RAC in human gene therapy experiments

First U.S. patient treated with innovative gene therapy at Bascom Palmer Eye Institute

Promising gene therapy for 'day blind' sheep now safe for clinical trials in human patients

Previously unknown virus acts as 'driver' for interstitial nephropathy

Discovering Prion-like Proteins in Eukaryotic Viruses

UCLA Mattel Children's Hospital recognized as one of the most innovative children's hospitals in the U.S.

Leveraging the power of automation to boost research

Automating research - solutions and best practices

How can microdialysis benefit drug development

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