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.

Newly developed gene therapy helps decelerate aging process

Aging is a leading risk factor for a number of debilitating conditions, including heart disease, cancer and Alzheimer's disease, to name a few. This makes the need for anti-aging therapies all the more urgent. Now, Salk Institute researchers have developed a new gene therapy to help decelerate the aging process.

20 Feb 2019

Gene therapy restores hearing in mice with congenital genetic deafness

In collaboration with the universities of Miami, Columbia and San Francisco, scientists from the Institut Pasteur, Inserm, CNRS, Collège de France, Sorbonne University and the University of Clermont Auvergne have managed to restore hearing in an adult mouse model of DFNB9 deafness - a hearing disorder that represents one of the most frequent cases of congenital genetic deafness.

19 Feb 2019

Unique gene therapy approach paves new way to tackle rare, inherited diseases

Nonsense mutations are single-letter errors in the genetic code that prematurely halt the production of critical proteins. These unfinished proteins are unable to function normally, and nonsense mutations cause 10-15 percent of all inherited genetic diseases, including Duchenne muscular dystrophy, spinal muscular atrophy, cystic fibrosis and polycystic kidney disease.

19 Feb 2019

Human cells can change their original function to battle against diabetes

For the first time, researchers have shown that ordinary human cells can change their original function. This may give new hope for diabetes patients.

14 Feb 2019

New process efficiently reprograms stem cells in one step using measles virus vector

Induced pluripotent stem cells, the workhorse of many regenerative medicine projects, start out as differentiated cells that are reprogrammed to pluripotent stem cells by exposure to a complex set of genetic cocktails.

13 Feb 2019

Simple drug cocktail converts neighboring glial cells into functional new neurons

A simple drug cocktail that converts cells neighboring damaged neurons into functional new neurons could potentially be used to treat stroke, Alzheimer's disease, and brain injuries.

8 Feb 2019

New DNA-based nanomachines can be used for gene therapy of cancer

Scientists from ITMO in collaboration with their international colleagues proposed new DNA-based nanomachines that can be used for gene therapy of cancer.

8 Feb 2019

Researcher studies how sickle cell disease affects blood flow and oxygen delivery

Sickle Cell Disease is rare but devastating. Due to a genetic mutation affecting hemoglobin, the component of the red blood cell that carries oxygen, a reduced amount of oxygen is supplied to vital tissues and organs.

7 Feb 2019

A new treatment for Chlamydia via Canadian researchers

A new way to prevent and treat one of the commonest sexually transmitted infections (STIs) has been developed by the researchers at the University of Manitoba and the University of Waterloo. This new method is more of a gene therapy than antibiotic therapy against the infection.

6 Feb 2019

Researchers develop new way to prevent and treat Chlamydia

Researchers at the University of Waterloo have developed a new way to prevent and treat Chlamydia, the most common sexually transmitted bacterial infection in the world.

6 Feb 2019

Study shows promise for future prostate cancer treatment

A new 'seek-and-destroy' gene therapeutic system could have the potential to treat prostate cancer in the future, after it halted the majority of tumors in laboratory models at the University of Strathclyde and the Beatson Institute.

4 Feb 2019

Oxford Genetics signs licensing agreement with Aldevron to market lentiviral packaging plasmids

Oxford Genetics, a leading innovator of biopharmaceutical technologies, has signed a licensing agreement with world-class service organization, Aldevron to bring to market a standardized range of its plasmids for lentiviral manufacture.

1 Feb 2019

Megaphages detected in the intestinal tract of humans who eat high-fiber, low-fat diet

Viruses plague bacteria just as viruses like influenza plague humans. Some of the largest of these so-called bacteriophages have now been found in the human gut, where they periodically devastate bacteria just as seasonal outbreaks of flu lay humans low, according to a new study led by University of California, Berkeley, scientists.

29 Jan 2019

Stand Up To Cancer creates Dream Team focused on finding therapies for T-cell lymphoma

Stand Up To Cancer announced here today the creation of an $8 million Dream Team of top scientists focused on finding therapies for T-cell lymphoma, a rare cancer of the blood and immune system, using chimeric antigen receptors (CARs) to trigger the immune system to attack and destroy cancerous cells.

29 Jan 2019

NIH grants awarded to Nanoscope to recruit talent and develop optogenetic delivery platforms

Retinal dystrophies such as Age-related macular degeneration (AMD) are a major cause of vision loss in aging populations. National Eye Institute (NEI) estimates that the prevalence of advanced AMD will grow to nearly 3 million by 2020.

29 Jan 2019

New class of antisense oligonucleotide therapeutics tested in humans

A first-in-human study with a new class of antisense oligonucleotide therapeutics showed the ability to target the RNA-silencing drug to the liver, resulting in improved potency and safety at therapeutic doses. The design and results of this trial, conducted in healthy human volunteers are reported in Nucleic Acid Therapeutics, a peer-reviewed journal from Mary Ann Liebert, Inc. publishers.

28 Jan 2019