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.

Reviving the heart's regenerative capacities using microRNAs

Once the heart is fully formed, the cells that make up heart muscle, known as cardiomyocytes, have very limited ability to reproduce themselves. After a heart attack, cardiomyocytes die off; unable to make new ones, the heart instead forms scar tissue. Over time, this can set people up for heart failure.

23 Apr 2019

Study compares effectiveness and cost of gene therapy and HSCT in major beta-thalassemia

Gene therapy offers the promise of a cure for beta-thalassemia and a new study has shown that it is associated with fewer complications and hospital admissions over 2 years than treatment by allogeneic hematopoietic stem cell transplantation

18 Apr 2019

New gene therapy cures babies with fatal 'Bubble Boy' disease

A new gene therapy created by US researchers appears to have cured eight infant boys born with a rare genetic disorder referred to as “Bubble Boy” disease.

18 Apr 2019

Researchers identify cause of inherited metabolic disorder

A new study from BC Children's Hospital, the University of British Columbia (UBC) and an international team of researchers published in the New England Journal of Medicine is the first to identify a rarely-seen type of DNA mutation as the cause of an inherited metabolic disorder.

16 Apr 2019

Key gene could decrease detrimental brain swelling after stroke

Could a medication someday help the brain heal itself after a stroke, or even prevent damage following a blow to the head? A new USC study lends support to the idea.

15 Apr 2019

INTERPHEX 2019 Celebrates 40 years of Delivering Technology Innovation to Industry

INTERPHEX has been the leading pharmaceutical, biotechnology, device development, and manufacturing event for 40 years.

From Interphex 5 Apr 2019

Scientists identify biomarkers to predict risk of atrial fibrillation

Scientists at the University of Navarra (Spain), in collaboration with clinicians from the University Hospital of Donostia, have identified two biomarkers associated with the risk of suffering atrial fibrillation, a cardiac ailment that affects more than 33.5 million people in the world.

2 Apr 2019

Researchers discover natural gene therapy for intractable skin disease

Pathogenic gene mutations causing a type of intractable skin disease can be eliminated from some parts of patients' skin as they age, according to Hokkaido University researchers and their collaborators in Japan. This represents a form of natural gene therapy.

1 Apr 2019

Breakthrough study could lead to new treatments for sickle cell disease

In a breakthrough study of sickle cell disease, biomedical engineers in the University of Minnesota College of Science and Engineering have revealed that the building blocks of the disease are much less efficient at organizing than previously thought.

29 Mar 2019

Researchers develop new strategy to treat sickle cell disease and beta thalassemia

Researchers at Dana-Farber/Boston Children's Cancer and Blood Disorders Center and the University of Massachusetts Medical School have developed a strategy to treat two of the most common inherited blood diseases -- sickle cell disease and beta thalassemia -- applying CRISPR-Cas9 gene editing to patients' own blood stem cells.

29 Mar 2019

Tumor biomarker EpCAM shown to be promising target for cancer immunotherapy

Researchers have shown that cancer immunotherapy targeting the tumor biomarker epithelial cell adhesion molecule is safe and nontoxic in mice and can significantly delay tumor formation and growth.

28 Mar 2019

Gene transfer technique can combat diabetes-linked heart dysfunction

Researchers with the VA San Diego Healthcare System and the University of California San Diego have shown that a gene transfer technique can combat heart dysfunction caused by diabetes.

28 Mar 2019

Using gene therapy strategies to rejuvenate aging cells and treat osteoarthritis

Aging is a risk factor for the development of osteoarthritis (OA), a chronic disease characterized by degeneration of articular cartilage leading to pain and physical disability.

28 Mar 2019

Newly engineered nanoscale protein micelles can be tracked by MRI

A team of researchers from New York University has engineered nanoscale protein micelles capable of both delivering chemotherapeutic drugs and of being tracked by magnetic resonance imaging.

21 Mar 2019

Merck to collaborate with GenScript for plasmid and virus manufacturing in China

Merck, a leading science and technology company, today announced the signing of a non-binding Memorandum of Understanding with Chinese biotech company GenScript for a strategic alliance focusing on plasmid and viral vector manufacturing.

From Merck 20 Mar 2019

Promising gene therapy could restore vision in humans

It was surprisingly simple. University of California, Berkeley, scientists inserted a gene for a green-light receptor into the eyes of blind mice and, a month later, they were navigating around obstacles as easily as mice with no vision problems.

15 Mar 2019

New therapy could help prolong useful vision and delay total blindness

Millions of Americans are progressively losing their sight as cells in their eyes deteriorate, but a new therapy developed by researchers at the University of California, Berkeley, could help prolong useful vision and delay total blindness.

14 Mar 2019

Researchers develop new method to boost efficiency of CRISPR gene editing in DMD

Scientists have developed a method to boost the efficiency of CRISPR gene editing in Duchenne muscular dystrophy (DMD), according to a study that could have implications for optimizing gene therapies for other diseases.

7 Mar 2019

Liver tissue model can help investigate the effects of RNA interference

Novel therapies based on a process known as RNA interference hold great promise for treating a variety of diseases by blocking specific genes in a patient's cells. Many of the earliest RNAi treatments have focused on diseases of the liver, because RNA-carrying particles tend to accumulate in that organ.

6 Mar 2019

Oxford Genetics successfully negotiates six licensing deals

Mercia Technologies PLC, the national investment group focused on the identification, creation, funding and scaling of innovative technology businesses with high growth potential from the UK regions, today announces that its portfolio company, Oxford Genetics Limited, has successfully negotiated six licensing deals for its scalable gene therapy manufacturing technologies in the last twelve months.

From OXGENE 5 Mar 2019