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.

Transformational $104 million gift will help VCU to expand treatment options for liver diseases

Virginia Commonwealth University will radically expand treatment options for liver and liver-related metabolic diseases thanks to a historic, transformational $104 million gift from Richard Todd Stravitz, M.D., and his family's Barbara Brunckhorst Foundation.

15 Feb 2022

Researchers create a comprehensive atlas of cerebrovascular cell types

While neurons and glial cells are by far the most numerous cells in the brain, many other types of cells play important roles. Among those are cerebrovascular cells, which form the blood vessels that deliver oxygen and other nutrients to the brain.

15 Feb 2022

Reducing mucin secretion provides a way to control chemoresistance of colorectal cancers

Drugs which are commonly-used as the first line of treatment for colorectal cancer cause the tumor cells to oversecrete proteins known as mucins, according to a new study published today in the journal eLife.

9 Feb 2022

Defective mitochondrial DNA could be modified using gene-editing techniques

Defective mitochondria – the 'batteries' that power the cells of our bodies – could in future be repaired using gene-editing techniques.

8 Feb 2022

Gene therapy helps heart muscle cells electrically activate in live mice

Biomedical engineers at Duke University have demonstrated a gene therapy that helps heart muscle cells electrically activate in live mice.

4 Feb 2022

Gene therapy may be a curative treatment option for patients with transfusion-dependent beta-thalassemia

A novel gene therapy promoted transfusion independence in more than 90 percent of adult and pediatric patients with transfusion-dependent beta-thalassemia, according to a recent clinical trial published in The New England Journal of Medicine.

3 Feb 2022

New approach to CAR T-cell therapy for B-cell cancers triples the targeted antigens on cancer cells

Researchers at University Hospitals Seidman Cancer Center and Case Western Reserve University have developed a novel approach to chimeric antigen receptor (CAR) T-cell therapy for B-cell cancers that triples the targeted antigens on cancer cells – a "back up" approach that promises to greatly reduce the potential for antigen escape currently found in CAR T therapies that solely target CD19.

2 Feb 2022

Researchers develop new technique to open the blood-brain barrier in lab animals

University of Maryland School of Medicine researchers developed a technique in laboratory animals to consistently and reproducibly open the blood-brain barrier.

31 Jan 2022

Dexamethasone administration at a late time point could enhance transgene expression

Dexamethasone, a glucocorticoid with anti-inflammatory and immunosuppressive effects, can transiently increase the expression of therapeutic genes delivered using adeno-associated virus (AAV) vectors.

31 Jan 2022

Desipramine and L-cycloserine together reduce ceramide levels in the retina and enhance vision

Researchers from the University of California, Irvine have discovered that the absence of Adiponectin receptor 1 protein (AdipoR1), one of the principal enzymes regulating ceramide homeostasis in the retina, leads to an accumulation of ceramides in the retina, resulting in progressive photoreceptor cell death and ultimately vision loss.

28 Jan 2022

Gene therapy can provide long-lasting therapeutic benefit in Huntington's disease

Gene therapy targeting the messenger RNA (mRNA) of the mutated huntingtin gene (HTT) can provide long-lasting therapeutic benefit in Huntington's disease after a single administration.

25 Jan 2022

Low-cost test could open new avenues for earlier diagnosis, treatment of rare genetic disorders

A newly developed test to screen for three rare genetic disorders simultaneously in newborns was feasible, reliable and scalable, according to a new study.

24 Jan 2022

Novel gene therapy for diseased muscle fiber repair

In this interview, News-Medical speaks to Dr. Jyoti K. Jaiswal about his latest research into gene therapies for limb-girdle muscular dystrophy 2B.

24 Jan 2022

Eikonoklastes completes a license to add novel gene therapy for treating neurodegenerative diseases

Eikonoklastes Therapeutics, a preclinical stage biopharmaceutical company, today announced it has completed a license with the University of California San Diego to add a novel gene therapy for the treatment of neurodegenerative diseases.

20 Jan 2022

Newly developed gene therapy can treat a severe form of epilepsy and prolong survival

Research from the University of Virginia School of Medicine suggests how a newly developed gene therapy can treat Dravet syndrome, a severe form of epilepsy, and potentially prolong survival for people with the condition.

20 Jan 2022

Study: Nearly 30% of people with blinding retinal disease can safely stop eye injection therapy

In a preliminary study of 106 people with "wet" age-related macular degeneration, Johns Hopkins Medicine researchers report that as many as a third of those with the blinding retinal disease may someday be able to safely stop eye injection therapy without further vision loss.

18 Jan 2022

New study links anxiety and PTSD to increased myelin in brain's gray matter

A recent study links anxiety behavior in rats, as well as post traumatic stress disorder (PTSD) in military veterans, to increased myelin - a substance that expedites communication between neurons - in areas of the brain associated with emotions and memory.

6 Jan 2022

New gene therapy addresses primary cellular deficit associated with limb-girdle muscular dystrophy 2B

Children's National Hospital experts developed a new pre-clinical gene therapy for a rare disorder, known as limb-girdle muscular dystrophy (LGMD) 2B, that addresses the primary cellular deficit associated with this disease.

4 Jan 2022

Structural blueprint of nanoparticles developed to target neutrophils responsible for acute lung inflammation

The COVID-19 pandemic highlighted the devastating impact of acute lung inflammation (ALI), which is part of the acute respiratory distress syndrome (ARDS) that is the dominant cause of death in COVID-19.

28 Dec 2021

Researchers identify new mechanism for DNA invasion of adenoviral COVID-19 vaccines

Adenoviruses have a linchpin protein that stabilizes their DNA until it reaches the infected cell's nucleus. The protein then detaches from the viral genome, and the virus uncoats. Only then are the genes released into the nucleus, which is necessary for the production of new viruses.

22 Dec 2021