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.

Tacere Therapeutics submits TT-034 clinical trial application to Recombinant DNA Advisory Committee

RNAi-based therapeutics company Benitec Biopharma Limited today announced that the company's wholly owned US subsidiary, Tacere Therapeutics Inc., has submitted an application to the US National Institutes of Health's Recombinant DNA Advisory Committee.

18 Apr 2013

Death of heart tissue could be prevented with new medicines, say TUSM scientists

A major factor in the advance of heart disease is the death of heart tissue, a process that a team of scientists at Temple University School of Medicine's Center for Translational Medicine think could be prevented with new medicines.

15 Apr 2013

New combination therapy could help restore vision

Researchers have discovered that using two kinds of therapy in tandem may be a knockout combo against inherited disorders that cause blindness. While their study focused on man's best friend, the treatment could help restore vision in people, too.

10 Apr 2013

Weill Cornell Medical College sets up new Feil Family Brain and Mind Research Institute

Weill Cornell Medical College has established the new Feil Family Brain and Mind Research Institute, a unique, multidisciplinary translational neuroscience research hub. Named in honor of long-time benefactors Gertrude and Louis Feil, the institute was created with a generous $28 million gift from the Feil Family.

9 Apr 2013

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.