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.

Sense of smell may play key role in how the body deals with calories

Our sense of smell is key to the enjoyment of food, so it may be no surprise that in experiments at the University of California, Berkeley, obese mice who lost their sense of smell also lost weight.

5 Jul 2017

Researchers uncover novel mechanism of beta-blockers in preventing progression of heart failure

Beta-blocker drugs serve a key role in the treatment of heart failure, preventing bombardment of the heart by catecholamines - substances like epinephrine and norepinephrine - which overexcite and stress the heart.

4 Jul 2017

Study sheds light on how key protein allows hearing to work

Although the basic outlines of human hearing have been known for years – sensory cells in the inner ear turn sound waves into the electrical signals that the brain understands as sound – the molecular details have remained elusive.

29 Jun 2017

International team to set how gene therapies in hemophilia should be measured for effectiveness

An international team involving dozens of stakeholders from patients and policy makers to payers and government agencies are getting together to set how gene therapies in hemophilia should be measured for effectiveness.

20 Jun 2017

Altered virus could help more patients to become eligible for human gene therapy trials

For many patients, participating in gene therapy clinical trials isn't an option because their immune system recognizes and fights the helpful virus used for treatment.

13 Jun 2017

Study reveals role of mast cells in severe forms of dengue infection

Why mosquito-borne dengue virus causes more severe disease in some individuals, including hemorrhagic fever with or without shock, remains controversial and researchers are focusing on the factors related to the interaction between the virus and the host immune system, including the role of mast cells.

12 Jun 2017

Researchers find long-lasting benefits of gene therapy in dog model of myotubular myopathy

Researchers who previously showed that a gene therapy treatment could save the lives of dogs with a deadly disease called myotubular myopathy—a type of muscular dystrophy that affects the skeletal muscles—have found that the therapy is long-lasting.

7 Jun 2017

New immunotherapy sends multiple myeloma into remission

Researchers are reporting the impressive results of a new cell and gene therapy that has achieved unprecedented success in treating multiple myeloma patients. Although the study is an early one and the population only included 35 people, every one of the patients responded to the treatment and 33 (94%) were in some degree of clinical remission within two months.

6 Jun 2017

New breakthrough offers hope for allergy sufferers

Scientists from The University of Queensland believe they have discovered a single treatment that could be a life-long cure for potentially fatal allergies such as asthma.

5 Jun 2017

Gene therapy could give protection from severe allergies

A single treatment giving life-long protection from severe allergies such as asthma could be made possible by immunology research at The University of Queensland.

2 Jun 2017

Gene editing technology can introduce hundreds of unintended mutations, study shows

As CRISPR-Cas9 starts to move into clinical trials, a new study published in Nature Methods has found that the gene-editing technology can introduce hundreds of unintended mutations into the genome.

29 May 2017

Experimental gene therapy proven safe for preserving vision of people with AMD

In a small and preliminary clinical trial, Johns Hopkins researchers and their collaborators have shown that an experimental gene therapy that uses viruses to introduce a therapeutic gene into the eye is safe and that it may be effective in preserving the vision of people with wet age-related macular degeneration.

17 May 2017

Spanish scientists develop promising gene therapy method to treat Wilson's disease

Scientists at the Center for Applied Medical Research at the University of Navarra have designed a promising gene therapy method to treat Wilson's disease, a rare pathology caused by the lack of or malfunction of a gene.

16 May 2017

Preclinical data reinforces potential efficacy, durability of investigational gene therapy for DMD

Solid Biosciences announced today that new data from two preclinical studies reinforce the potential of its investigational microdystrophin gene therapy, SGT-001, to be an effective treatment approach for Duchenne muscular dystrophy.

11 May 2017

Sangamo Therapeutics and Pfizer team up for development of Hemophilia A gene therapy

Sangamo Therapeutics, Inc. and Pfizer Inc. today announced an exclusive, global collaboration and license agreement for the development and commercialization of gene therapy programs for Hemophilia A, including SB-525, one of Sangamo's four lead product candidates, which Sangamo expects will enter the clinic this quarter.

11 May 2017

Study: New gene editing strategy effectively targets cancer-causing ‘fusion genes’ in mouse models

A novel gene therapy using CRISPR genome editing technology effectively targets cancer-causing "fusion genes" and improves survival in mouse models of aggressive liver and prostate cancers, University of Pittsburgh School of Medicine researchers report in a study published online today in Nature Biotechnology.

2 May 2017

$Women at highest genetic risk for fracture benefit most from hormone therapy, study finds$

Women at highest genetic risk for fracture benefit most from hormone therapy, study finds

Women at the highest genetic risk for fracture benefit the most from hormone therapy, according to a first-of-its-kind study led by researchers at the University at Buffalo.

24 Apr 2017

Latest advances in antibody delivery mediated by rAAV for treatment of chronic and infectious diseases

Monoclonal antibody-based therapies have revolutionized treatments of cancer and autoimmune diseases because of their specificity and limited toxicity.

21 Apr 2017

Researchers use gene-editing tool to restore visual function in mouse models of retinitis pigmentosa

Using the gene-editing tool CRISPR/Cas9, researchers at University of California San Diego School of Medicine and Shiley Eye Institute at UC San Diego Health, with colleagues in China, have reprogrammed mutated rod photoreceptors to become functioning cone photoreceptors, reversing cellular degeneration and restoring visual function in two mouse models of retinitis pigmentosa.