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 how pre-transplant chemotherapy facilitates the replacement of microglial cells

More than 50,000 bone marrow-derived stem cell transplants are performed worldwide each year to treat a wide range of conditions, including brain diseases.

21 Feb 2022

Drugs that increase fetal hemoglobin levels may help people with sickle cell disease

Sickle cell anemia is an inherited blood disorder where red blood cells become sickle/crescent shaped. It causes frequent infections, swelling in the hands and legs, pain, severe tiredness and delayed growth or puberty.

18 Feb 2022

Search for a treatment to stop nerve cell degeneration yields promising results

Researchers at the University of Illinois Chicago found promising results in their search for a treatment to stop nerve cell degeneration that happens in some types of disorders, such as hereditary spastic paraplegia and Parkinson's disease, which can cause significant disability.

17 Feb 2022

Study investigates what causes blood malignancies linked to gene therapy for sickle cell disease

Gene therapy trials for sickle cell disease have been showing great promise, even offering hope of a cure. But in early 2021, the trials ground to a halt after reports of blood malignancies in two people in a trial sponsored by bluebird bio.

16 Feb 2022

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.