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.

Genable Technologies, Spark Therapeutics to advance novel therapy for rare form of retinitis pigmentosa

Spark Therapeutics and Genable Technologies announced today that they have entered into a collaboration agreement for Genable's lead therapeutic to treat rhodopsin-linked autosomal dominant retinitis pigmentosa (RHO adRP), GT038.

25 Mar 2014

XBP1 gene plays pivotal role in the growth and progression of triple negative breast cancer

Scientists from Weill Cornell Medical College and Houston Methodist have found that a gene previously unassociated with breast cancer plays a pivotal role in the growth and progression of the triple negative form of the disease, a particularly deadly strain that often has few treatment options. Their research, published in this week's Nature, suggests that targeting the gene may be a new approach to treating the disease.

25 Mar 2014

Marina Cavazzana, Adrian J. Thrasher honored with Pioneer Award for gene therapy

Marina Cavazzana, MD, PhD, Paris Descartes University, France and Adrian J. Thrasher, MD, PhD, University College London Institute of Child Health, UK, have been honored with the Pioneer Award for basic and clinical gene therapy for immunodeficiency disorders.

25 Mar 2014

Researchers develop adjuvant which enhances effect of virus transduction

Lentiviruses, which belong to the family of retroviruses, are used as vectors to exchange genetic material in cells and can be used to replace a defective gene as defined by gene therapy.

5 Mar 2014

Abingworth announces final closing of £225M ($375M) fund

Abingworth, the international investment group dedicated to life sciences and healthcare, today announced the final closing of its £225m ($375m) fund, Abingworth Bioventures VI (ABV VI). ABV VI, Abingworth's 10th life sciences fund, exceeded its target of £200 million and invests in life sciences and healthcare both in Europe and the US.

28 Feb 2014

Vaccine targeting tumors could initiate immune response to fight cancer

Cincinnati Cancer Center (CCC) and UC Cancer Institute researchers have found that a vaccine, targeting tumors that produce a certain protein and receptor responsible for communication between cells and the body's immune system, could initiate the immune response to fight cancer.

28 Feb 2014

Study highlights the importance of personalized medicine for treating cancer

If a driver is traveling to New York City, I-95 might be their route of choice. But they could also take I-78, I-87 or any number of alternate routes. Most cancers begin similarly, with many possible routes to the same disease. A new study found evidence that assessing the route to cancer on a case-by-case basis might make more sense than basing a patient's cancer treatment on commonly disrupted genes and pathways.

26 Feb 2014

European Commission invests nearly $475 million in gene transfer and gene therapy projects

Over the past three funding stages, the European Commission has invested nearly $475 million in 100 projects in the gene transfer and gene therapy field.

25 Feb 2014

Cell therapy: A powerful treatment for cancer patients

Investigators from Memorial Sloan Kettering Cancer Center have reported more encouraging news about one of the most exciting methods of cancer treatment today. The largest clinical study ever conducted to date of patients with advanced leukemia found that 88 percent achieved complete remissions after being treated with genetically modified versions of their own immune cells.

24 Feb 2014

Study finds Cyclin A2 protein induces cardiac regeneration after myocardial infarction

After a heart attack, there is often permanent damage to a portion of the heart. This happens, in part, because cardiac muscle cells are terminally differentiated and cannot proliferate after blood flow is blocked off to the heart.

20 Feb 2014

NIH-funded session in Bethesda to heighten awareness of underactive bladder

Underactive bladder, estimated to affect more than 20 percent of the elderly population, is an unrecognized disease that has a major influence on the health and independence of seniors.

20 Feb 2014

Researcher receives Pioneer Award for engineering herpes simplex virus gene delivery systems

Joseph C. Glorioso, III, PhD (University of Pittsburgh School of Medicine, PA) devoted much of his research career to developing herpes viruses as efficient vectors for delivering therapeutic genes into cells.

20 Feb 2014

Cystinosis Research Foundation granted $2.15M in 2013 to researchers investigating cure for cystinosis

The Cystinosis Research Foundation awarded $2,153,048 in 2013 to researchers investigating better treatments and a cure for cystinosis, a rare metabolic and genetic disease that afflicts about 500 children and young adults in the United States and 2,000 worldwide.

19 Feb 2014

Duke researchers getting closer to generate replacement cartilage

By combining a synthetic scaffolding material with gene delivery techniques, researchers at Duke University are getting closer to being able to generate replacement cartilage where it's needed in the body.

19 Feb 2014

Researchers identify new type of regulatory blood cells that can combat brain inflammation

Hyperactivity of our immune system can cause a state of chronic inflammation. If chronic, the inflammation will affect our body and result in disease. In the devastating disease multiple sclerosis, hyperactivity of immune cells called T-cells induce chronic inflammation and degeneration of the brain. Researchers at BRIC, the University of Copenhagen, have identified a new type of regulatory blood cells that can combat such hyperactive T-cells in blood from patients with multiple sclerosis. By stimulating the regulatory blood cells, the researchers significantly decreased the level of brain inflammation and disease in a biological model.

17 Feb 2014

Horizon Discovery, Sirion Biotech ink supply and distribution agreement

Horizon Discovery, a leading provider of research tools to support translational genomics and the development of personalized medicines, today announced it has signed a supply and distribution agreement with Sirion Biotech GmbH.

17 Feb 2014

Researchers receive funding to develop treatments for Rett Syndrome

Researchers from Scotland and the US are taking a step towards reversing the effects of a debilitating genetic disease and bring hope to tens of thousands of sufferers worldwide.

14 Feb 2014

UMMS launches Voyager Therapeutics to develop life-changing treatments for fatal diseases

UMass Medical School (UMMS) recognizes the launch today of Voyager Therapeutics, a gene therapy company founded by four world leaders in the fields of AAV gene therapy, RNA biology and neuroscience, to develop life-changing treatments for fatal and debilitating diseases of the central nervous system (CNS).

13 Feb 2014

New approach for reducing levels of toxic protein fragments associated with Huntington's disease

An innovative therapeutic strategy for reducing the levels of toxic protein fragments associated with Huntington's disease uses a new approach called exon skipping to remove the disease-causing component of the essential protein, huntingtin.

11 Feb 2014

Researchers discover genetic mutations that cause deadly lung disease

A team of researchers, led by physicians and scientists at Intermountain Medical Center and ARUP Laboratories in Salt Lake City, has made a medical breakthrough by discovering genetic mutations that cause a rare and deadly lung disease.

4 Feb 2014

Gene Therapy News and Research

Further Reading

Genable Technologies, Spark Therapeutics to advance novel therapy for rare form of retinitis pigmentosa

XBP1 gene plays pivotal role in the growth and progression of triple negative breast cancer

Marina Cavazzana, Adrian J. Thrasher honored with Pioneer Award for gene therapy

Researchers develop adjuvant which enhances effect of virus transduction

Abingworth announces final closing of £225M ($375M) fund

Vaccine targeting tumors could initiate immune response to fight cancer

Study highlights the importance of personalized medicine for treating cancer

European Commission invests nearly $475 million in gene transfer and gene therapy projects

Cell therapy: A powerful treatment for cancer patients

Study finds Cyclin A2 protein induces cardiac regeneration after myocardial infarction

NIH-funded session in Bethesda to heighten awareness of underactive bladder

Researcher receives Pioneer Award for engineering herpes simplex virus gene delivery systems

Cystinosis Research Foundation granted $2.15M in 2013 to researchers investigating cure for cystinosis

Duke researchers getting closer to generate replacement cartilage

Researchers identify new type of regulatory blood cells that can combat brain inflammation

Horizon Discovery, Sirion Biotech ink supply and distribution agreement

Researchers receive funding to develop treatments for Rett Syndrome

UMMS launches Voyager Therapeutics to develop life-changing treatments for fatal diseases

New approach for reducing levels of toxic protein fragments associated with Huntington's disease

Researchers discover genetic mutations that cause deadly lung disease

Leveraging the power of automation to boost research

Automating research - solutions and best practices

How can microdialysis benefit drug development

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