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.

GenSight Biologics announces closing of €32 million Series A financing

GenSight Biologics, a biopharmaceutical company focused on the development of ophthalmic therapeutics using gene therapy, announced today the closing of a €32 million Series A financing.

8 Apr 2013

Liver transplants worth risk for people who have genetic liver conditions, study finds

Patients faced with the diagnosis of a life-threatening liver disease have to consider the seriousness of having a liver transplant, which can be a definitive cure for many acquired and genetic liver diseases.

6 Apr 2013

Penn Medicine scientist, trial participant detail experiences on leading edge of gene therapy

Harnessing the patient's own immune system to fight cancer through genetic modification of their own cells is rapidly emerging as a promising treatment option.

6 Apr 2013

Sanford-Burnham scientists identify how enzyme PKCzeta suppresses prostate tumor formation

Scientists at Sanford-Burnham Medical Research Institute have identified how an enzyme called PKCζ suppresses prostate tumor formation. The finding, which also describes a molecular chain of events that controls cell growth and metastasis, could lead to novel ways to control disease progression.

2 Apr 2013

Explosive growth in tissue engineering and regenerative medicine leads to innovative, promising applications

Explosive growth in the field of tissue engineering and regenerative medicine has led to innovative and promising applications and techniques, many of which are now being tested in human clinical trials.

30 Mar 2013

Study: Gene therapy can boost heart muscle function

In an animal study, researchers at the University of Washington show that it is possible to use gene therapy to boost heart muscle function. The finding suggests that it might be possible to use this approach to treat patients whose hearts have been weakened by heart attacks and other heart conditions.

27 Mar 2013

Study raises alarm over loss of individual 'genomic liberty' due to gene patents

Humans don't "own" their own genes, the cellular chemicsals that define who they are and what diseases they might be at risk for. Through more than 40,000 patents on DNA molecules, companies have essentially claimed the entire human genome for profit, report two researchers who analyzed the patents on human DNA. Their study, published March 25 in the journal Genome Medicine, raises an alarm about the loss of individual "genomic liberty."

27 Mar 2013

Novel cell therapy effective in treating aggressive form of childhood leukemia

Two children with an aggressive form of childhood leukemia had a complete remission of their disease—showing no evidence of cancer cells in their bodies—after treatment with a novel cell therapy that reprogrammed their immune cells to rapidly multiply and destroy leukemia cells.

26 Mar 2013

Two studies shed light on molecular biology of three blood disorders

Two studies led by investigators at Weill Cornell Medical College shed light on the molecular biology of three blood disorders, leading to novel strategies to treat these diseases.

26 Mar 2013

Researchers expand hematopoietic stem cells for bone marrow transplantation

More than 50,000 stem cell transplants are performed each year worldwide. A research team led by Weill Cornell Medical College investigators may have solved a major issue of expanding adult hematopoietic stem cells (HSCs) outside the human body for clinical use in bone marrow transplantation -- a critical step towards producing a large supply of blood stem cells needed to restore a healthy blood system.

25 Mar 2013

Study reveals role of SNX27 protein in brain function

What is it about the extra chromosome inherited in Down syndrome-chromosome 21-that alters brain and body development? Researchers at Sanford-Burnham Medical Research Institute have new evidence that points to a protein called sorting nexin 27, or SNX27.

25 Mar 2013

Weill Cornell researcher suggests ways to improve patient safety, high-level care

In its 2001 report Crossing the Quality Chasm, the Institute of Medicine outlined six domains of quality in medical care: safety, effectiveness, patient-centeredness, timeliness, efficiency and equity.

24 Mar 2013

Translational Research's April issue examines progress, outlook of gene therapy research

The April issue of Translational Research examines the progress and outlook of gene therapy research, with a specific focus on the clinical applicability of gene therapy today.

22 Mar 2013

Blocked bile ducts can be effectively treated with metal stents

A multi-center analysis, led by Weill Cornell Medical College and published in the Journal of Clinical Gastroenterology, shows the use of temporary "fully covered self-expanding metal stents" (FCSEMS) can effectively fix a painful and potentially life-threatening benign biliary stricture -- a severely blocked or narrowed bile duct.

21 Mar 2013

Cedars-Sinai surgeons perform more adult heart transplants in 2012, statistics show

For the third year in a row, the Cedars-Sinai Heart Institute and Comprehensive Transplant Center performed more adult heart transplants than any other U.S. medical center, according to statistics compiled by the United Network for Organ Sharing, the nonprofit organization that manages the nation's transplant system.

21 Mar 2013

Weill Cornell Medical College researchers call for clearer definition of population health

Accountable Care Organizations, a key feature of the Affordable Care Act, aim to control health care costs, enhance quality in health care and improve population health. But what does "improving population health" really mean? This is the question asked in a new viewpoint article by Weill Cornell Medical College researchers published in the March 20 issue of the Journal of the American Medical Association.

20 Mar 2013

New therapeutic approach for bladder pain syndrome

Severe chronic pain associated with conditions such as bladder pain syndrome/interstitial cystitis often require the use of opioid medication, with the risk of dependency and serious adverse reactions.

15 Mar 2013

Study identifies genetic cause of developmental delay in Amish individuals in USA

Researchers from the research group in growth factors and cell differentiation at the University of Barcelona (UB) and the IDIBELL and have participated in an international study that has identified the genetic cause of developmental delay observed in Amish individuals in the USA.

15 Mar 2013

Study demonstrates relationship between protein HERC2 and human diseases

Researchers from the research group in growth factors and cell differentiation at IDIBELL and the University of Barcelonahave participated in an international study that has identified the genetic cause of developmental delay observed in Amish individuals in the USA.

13 Mar 2013

Weill Cornell receives NIMH grant to discover novel biomarkers for chronic fatigue syndrome

Weill Cornell Medical College has been awarded more than $1.9 million by the National Institute of Mental Health of the National Institutes of Health to lead an innovative research study using advanced neuroimaging and clinical evaluations of patients with chronic fatigue syndrome (CFS).

8 Mar 2013