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.

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

EMA recognizes the value of EBMT patient registry for novel CAR T-cell therapies

CAR T-cell immunotherapies are advanced therapy medicinal products manufactured from human primary living cells procured by hospitals or blood banks.

4 Mar 2019

Researchers are developing and testing novel gene therapy to cure Crigler-Najjar syndrome

Crigler-Najjar syndrome affects about one in a million people at birth. Because of a defective gene, these individuals lack an essential enzyme in their liver to eliminate bilirubin, a toxic bile pigment that accumulates in all body tissues causing jaundice and potentially leading to irreversible and lethal neurological damage.

28 Feb 2019

Scientists identify cause of devastating pediatric brain disorder

An international effort led by physician-scientists at Rady Children's Institute for Genomic Medicine, in collaboration with a team at the Montreal Children's Hospital of the McGill University Health Centre, has identified the cause of a devastating pediatric brain disorder paving the way for the first step in developing potential therapies for this rare neurodegenerative condition.

25 Feb 2019

New intracellular delivery system using gold nano-stars

Researchers from Russian Academy of Sciences developed a new method for star-shaped nanoparticles synthesis based on laser irradiation.

21 Feb 2019

First gene therapy operation for macular degeneration is a success

A new treatment is the first to target the underlying genetic cause of AMD, which affects more than 600,000 people in the UK alone.

20 Feb 2019

Gene Therapy News and Research

Further Reading

INTERPHEX 2019 Celebrates 40 years of Delivering Technology Innovation to Industry

Scientists identify biomarkers to predict risk of atrial fibrillation

Researchers discover natural gene therapy for intractable skin disease

Breakthrough study could lead to new treatments for sickle cell disease

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

Tumor biomarker EpCAM shown to be promising target for cancer immunotherapy

Gene transfer technique can combat diabetes-linked heart dysfunction

Using gene therapy strategies to rejuvenate aging cells and treat osteoarthritis

Newly engineered nanoscale protein micelles can be tracked by MRI

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

Promising gene therapy could restore vision in humans

New therapy could help prolong useful vision and delay total blindness

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

Liver tissue model can help investigate the effects of RNA interference

Oxford Genetics successfully negotiates six licensing deals

EMA recognizes the value of EBMT patient registry for novel CAR T-cell therapies

Researchers are developing and testing novel gene therapy to cure Crigler-Najjar syndrome

Scientists identify cause of devastating pediatric brain disorder

New intracellular delivery system using gold nano-stars

First gene therapy operation for macular degeneration is a success

Leveraging the power of automation to boost research

Automating research - solutions and best practices

How can microdialysis benefit drug development

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