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.

Eminent biologist resigns over allegations of gender discrimination and sexual harassment

Eminent biologist and cancer researcher Inder Verma has resigned from the Salk Institute for Biological Sciences in La Jolla, California according to an announcement made by the institute on the 11th of June 2018

13 Jun 2018

Penn research team identifies novel therapeutic target for heart disease

Changes in cellular struts called microtubules (MT) can affect the stiffness of diseased human heart muscle cells, and reversing these modifications can lessen the stiffness and improve the beating strength of these cells isolated from transplant patients with heart failure, found researchers from the Perelman School of Medicine at the University of Pennsylvania.

12 Jun 2018

CRISPR-Cas9 technique may raise cancer risk

Therapeutic use of gene editing with the so-called CRISPR-Cas9 technique may inadvertently increase the risk of cancer, according to a new study from Karolinska Institutet, Sweden, and the University of Helsinki, Finland, published in Nature Medicine.

11 Jun 2018

New way of delivering gene therapy shows promise in treating brain conditions

Researchers from the School of Immunology & Microbial Sciences have engineered a novel way of delivering gene therapy that they hope will mean scientists and doctors can target areas of the brain that were previously difficult to reach.

5 Jun 2018

Researchers use magnets to control tiny DNA-based robots

Researchers have devised a magnetic control system to make tiny DNA-based robots move on demand-;and much faster than recently possible.

4 Jun 2018

Gene therapy that reverses blindness in dogs could also help treat humans

A Michigan State University veterinary ophthalmologist has modified a gene therapy that reverses blindness in dogs that have a certain form of a disease known as progressive retinal atrophy, or PRA, and is now looking to advance the treatment for human use.

18 May 2018

Rocket Pharma partners with Stanford University to advance FA and PKD gene therapy research

Rocket Pharmaceuticals, Inc., a leading U.S.-based multi-platform gene therapy company, and the Stanford University School of Medicine today announced a strategic collaboration to support the advancement of Fanconi Anemia and Pyruvate Kinase Deficiency gene therapy research.

17 May 2018

Obsidian Therapeutics presents preclinical data on regulated IL12 and IL15 programs

Obsidian Therapeutics, Inc., a biotechnology company dedicated to the development of next-generation cell and gene therapies with pharmacologic operating systems, today announced that the company presented preclinical data on its regulated IL12 and IL15 programs at the Annual Meeting of the American Society of Gene and Cell Therapy in Chicago, IL.

17 May 2018

Researchers reverse Alzheimer's symptoms in flies by restoring epigenetic balance

Researchers from Drexel University reversed symptoms of Alzheimer's disease in fruit flies by restoring the balance between two epigenetic enzymes that regulate gene expression, a study shows.

15 May 2018

LogicBio team to present data on preclinical effectiveness of GeneRide platform

LogicBio Therapeutics, Inc., a genetic medicine company founded to develop safe medicines with lasting therapeutic benefit for children with life-threatening diseases, today announced it will be presenting data on the company's technology and robust preclinical pipeline at the annual meeting of the American Society of Gene & Cell Therapy at the Hilton Chicago, from May 16-19, 2018.

14 May 2018

Why the drugmaker that paid Trump’s lawyer $1.2 million is so worried

President Donald Trump didn't mention Novartis or other drugmakers by name last year when he said the industry is "getting away with murder."

11 May 2018

New case study shows CAR T therapy as viable option for AML patients

Chimeric Antigen Receptor T-cell therapy, also known as CAR T therapy, was named the biggest research breakthrough of 2017 by the American Society of Clinical Oncology. The personal gene therapy utilizes a patient's own immune cells to fight cancer.

9 May 2018

International research collaboration aims to prevent heart arrhythmias with gene therapy

The most common and potentially lethal complication following a heart attack is the heart's inability to do one of its most basic jobs: beat at a normal rate.

9 May 2018

Study shows advantages of using decellularized muscle to promote tissue regeneration

A new comparative study showed the advantages of using donor decellularized muscle to promote functional tissue regeneration at the site of bulk skeletal muscle loss due to trauma or surgery.

4 May 2018

Research team uncovers link between circadian clock and aggression center in the brain

Each year around many people worldwide are diagnosed with a form of dementia. Alzheimer's disease is one of them. The disease manifests itself in memory difficulties in particular, but can also result in personality changes and mood swings.

2 May 2018

New strategy found to delay onset of blindness in mice

More than 2 million people worldwide live with inherited and untreatable retinal conditions, including retinitis pigmentosa, which slowly erodes vision.

2 May 2018

Gene therapy offers potential cure for patients with lipoprotein lipase deficiency

During the first 18 months after treatment with ali-pogene tiparvovec, a gene therapy recently approved in Europe to treat lipoprotein lipase deficiency (LPLD), the first patient to receive the treatment had no abdominal pain or episodes of pancreatitis, following a history of 37 pancreatitis attacks.

2 May 2018

Gene therapy is the best stratagem to impact the course of GM2 gangliosidosis

Since its clinical description in 1881, Tay-Sachs disease has had almost totemic significance as a cruel and relentless genetic condition that destroys the developing brain of babies and young children.

28 Apr 2018

Researchers use CRISPR/Cas9 technique to turn off gene that regulates cholesterol levels in adult mice

Biomedical engineers at Duke University have used a CRISPR/Cas9 genetic engineering technique to turn off a gene that regulates cholesterol levels in adult mice, leading to reduced blood cholesterol levels and gene repression lasting for six months after a single treatment.

27 Apr 2018

Key signaling protein in Huntington's disease found to have deleterious effects on heart function, shows study

Researchers investigating a key signaling protein in Huntington's disease describe deleterious effects on heart function, going beyond the disease's devastating neurological impact.

25 Apr 2018

Gene Therapy News and Research

Further Reading

Eminent biologist resigns over allegations of gender discrimination and sexual harassment

Penn research team identifies novel therapeutic target for heart disease

CRISPR-Cas9 technique may raise cancer risk

New way of delivering gene therapy shows promise in treating brain conditions

Researchers use magnets to control tiny DNA-based robots

Gene therapy that reverses blindness in dogs could also help treat humans

Rocket Pharma partners with Stanford University to advance FA and PKD gene therapy research

Obsidian Therapeutics presents preclinical data on regulated IL12 and IL15 programs

Researchers reverse Alzheimer's symptoms in flies by restoring epigenetic balance

LogicBio team to present data on preclinical effectiveness of GeneRide platform

Why the drugmaker that paid Trump’s lawyer $1.2 million is so worried

New case study shows CAR T therapy as viable option for AML patients

International research collaboration aims to prevent heart arrhythmias with gene therapy

Study shows advantages of using decellularized muscle to promote tissue regeneration

Research team uncovers link between circadian clock and aggression center in the brain

New strategy found to delay onset of blindness in mice

Gene therapy offers potential cure for patients with lipoprotein lipase deficiency

Gene therapy is the best stratagem to impact the course of GM2 gangliosidosis

Researchers use CRISPR/Cas9 technique to turn off gene that regulates cholesterol levels in adult mice

Key signaling protein in Huntington's disease found to have deleterious effects on heart function, shows study

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Automating research - solutions and best practices

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