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.

Xenotransplantation – organ transplants from animals may soon be a reality

Researchers have been able to create genetically modified piglets that are cleaned of the viruses that could causes diseases in humans. This could open up new avenues in organs transplants from pigs to humans. The process of transplanting organs or tissues from animals to humans is called xenotransplantation. The report was published this Tuesday in the journal Science.

11 Aug 2017

Clinical trial shows safety, effectiveness of pre-prepared cells to treat drug-resistant viral infections

Patients who receive a hematopoietic stem cell transplant are at risk of developing potentially lethal viral infections. Some of the infections can be treated with available drugs, but they are not always effective and side effects can be significant.

8 Aug 2017

Biological bypass found safe for patients with coronary artery disease

A new gene therapy that targets the heart and requires only one treatment session has been found safe for patients with coronary artery disease, according to a successful trial carried out in Finland.

8 Aug 2017

Nanotechnology wonders: Organ healing with a single touch!

Scientists from the Ohio State University Wexner Medical Centre have come up with what sounds like a science fiction - a nanotechnology device that can switch the cell functions in such a way that the failing organs are revived using a single touch.

7 Aug 2017

Newly discovered biomarker linked to rare metabolic disorders may facilitate better diagnosis

A newly discovered biomarker associated with a rare metabolic disorder may facilitate better diagnosis and identification of new drugs for clinical trials for the disease, according to researchers in the Perelman School of Medicine at the University of Pennsylvania.

7 Aug 2017

Gene-therapy administered through skin transplants could enable treatment of many diseases

A research team based at the University of Chicago has overcome challenges that have limited gene therapy and demonstrated how their novel approach with skin transplantation could enable a wide range of gene-based therapies to treat many human diseases.

3 Aug 2017

Study shows new method for repairing disease-causing mutation in human embryos

Scientists have demonstrated an effective way of using a gene-editing tool to correct a disease-causing gene mutation in human embryos and stop it from passing to future generations.

3 Aug 2017

Bringing highly potent cell therapy products to market

There are two main challenges and both relate to manufacturing. The first one is to make sure you can manufacture therapies with reasonable costs and the second one is to make sure that you can balance the capacity you need with the demands.

31 Jul 2017

Researchers compare bone-healing effects of three low-dose growth factors

Researchers compared the effects of three bone growth factors to bone morphogenetic protein 2 (BMP2) -- the most commonly used agent for repair of large bone defects, which is not without risks at the doses required--and showed significant bone-healing effects including the formation of new blood vessels at low doses relative to BMP2.

28 Jul 2017

Preclinical study demonstrates efficacy of gene therapy in treatment of Duchenne muscular dystrophy

Researchers from Genethon, the AFM-Telethon laboratory, Inserm (UMR 1089, Nantes) and the University of London (Royal Holloway) demonstrated the efficacy of an innovative gene therapy in the treatment of Duchenne muscular dystrophy.

25 Jul 2017

Genetic therapy for two inherited neurologic diseases moves closer to human clinical trials

Researchers at University of Massachusetts Medical School and Auburn University College of Veterinary Medicine are nearing human clinical trials on a genetic therapy for two rare neurological diseases that are fatal to children.

19 Jul 2017

Developing software for cell and gene therapy supply chain tracking

The first thing is, to always remember that we're dealing with a living drug, cells, which are very sensitive to environmental conditions, such as temperature and humidity etc. All of those things can impact on the viability of the drug and its effectiveness.

18 Jul 2017

'Organic gene therapy' could be effective treatment approach for serious blood disorders

Genome therapy with beneficial natural mutation could lead to new treatment for life-threatening blood disorders.

17 Jul 2017

MGH researchers identify novel mechanism behind resistance to angiogenesis inhibitors

A Massachusetts General Hospital research team has identified a novel mechanism behind resistance to angiogenesis inhibitors - drugs that fight cancer by suppressing the formation of new blood vessels.

14 Jul 2017

Scientists uncover new gene therapy treatment routes for motor neurone disease

Scientists investigating the genetic causes and altered functioning of nerve cells in motor neurone disease (MND) have discovered a new mechanism that could lead to fresh treatment approaches for one of the most common forms of the disease.

14 Jul 2017

FDA panel recommends new breakthrough gene therapy for B-cell acute lymphoblastic leukemia (ALL)

A Food and Drug Administration (FDA) panel created history by recommending a gene therapy for patients with cancer. In this therapy the patient’s own genes would be modified to fight the cancer cells. The patient’s cells which are thus altered to treat the cancer would thus be termed as “living drugs” as they act as soldiers to treat the cancer. These cells boost up the immune system and help the body fight the cancer.

14 Jul 2017

FDA advisory committee recommends Novartis' CAR-T therapy for young leukemia patients

Today the FDA's Oncologic Drugs Advisory Committee, an independent panel of experts, voted unanimously to recommend to the FDA approval of Novartis' experimental CAR-T therapy called Tisagenlecleucel, also known as CTL019.

13 Jul 2017

New partnership to advance novel airway-delivered gene therapy for treating pulmonary hypertension

Mount Sinai has partnered with Theragene Pharmaceuticals, Inc. to advance a novel airway-delivered gene therapy for treating pulmonary hypertension (PH), a form of high blood pressure in blood vessels in the lungs that is linked to heart failure.

13 Jul 2017

Synthetic compound that reads mitochondrial DNA could lead to treatments for nerve, muscle diseases

For the first time, a synthetic compound has been made that can bind to DNA in the cells' energy powerhouses, suppressing a gene associated with nerve and muscle disease.

11 Jul 2017

Loss of smell linked to weight loss in mice despite same food intake, study reveals

Experiments conducted at the University of California, Berkeley, suggest that the odor of food has a direct link with how the body processes the calories. The new study conducted on mice published in the first week of July in the journal Cell Metabolism implicates that the ability to detect the smell of food is related to whether the calories are burnt rather than stored.

6 Jul 2017

Gene Therapy News and Research

Further Reading

Xenotransplantation – organ transplants from animals may soon be a reality

Clinical trial shows safety, effectiveness of pre-prepared cells to treat drug-resistant viral infections

Biological bypass found safe for patients with coronary artery disease

Nanotechnology wonders: Organ healing with a single touch!

Newly discovered biomarker linked to rare metabolic disorders may facilitate better diagnosis

Gene-therapy administered through skin transplants could enable treatment of many diseases

Study shows new method for repairing disease-causing mutation in human embryos

Bringing highly potent cell therapy products to market

Researchers compare bone-healing effects of three low-dose growth factors

Preclinical study demonstrates efficacy of gene therapy in treatment of Duchenne muscular dystrophy

Genetic therapy for two inherited neurologic diseases moves closer to human clinical trials

Developing software for cell and gene therapy supply chain tracking

'Organic gene therapy' could be effective treatment approach for serious blood disorders

MGH researchers identify novel mechanism behind resistance to angiogenesis inhibitors

Scientists uncover new gene therapy treatment routes for motor neurone disease

FDA panel recommends new breakthrough gene therapy for B-cell acute lymphoblastic leukemia (ALL)

FDA advisory committee recommends Novartis' CAR-T therapy for young leukemia patients

New partnership to advance novel airway-delivered gene therapy for treating pulmonary hypertension

Synthetic compound that reads mitochondrial DNA could lead to treatments for nerve, muscle diseases

Loss of smell linked to weight loss in mice despite same food intake, study reveals

Leveraging the power of automation to boost research

Automating research - solutions and best practices

How can microdialysis benefit drug development

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