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.

New research on diagnosis and treatment of leukemia to be presented at 53rd ASH meeting

Despite major research advances over the last several decades that have helped deliver improved therapeutic options for leukemia, the condition remains deadly. Specialists are in need of new options to help diagnose the condition earlier and new therapies that will extend patients' lives. New research addressing important updates on the diagnosis and treatment of leukemia will be presented today at the 53rd Annual Meeting of the American Society of Hematology.

12 Dec 2011

Gene therapy can reduce painful bleeding episodes in patients with hemophilia B

Symptoms improved significantly in adults with the bleeding disorder hemophilia B following a single treatment with gene therapy developed by researchers at St. Jude Children's Research Hospital in Memphis and demonstrated to be safe in a clinical trial conducted at the University College London (UCL) in the U.K.

12 Dec 2011

New gene editing technique can heal sickle cell patients with their own cells

Researchers at the Salk Institute for Biological Studies have developed a way to use patients' own cells to potentially cure sickle cell disease and many other disorders caused by mutations in a gene that helps produce blood hemoglobin.

8 Dec 2011

Research: lncRNA affects apoptosis and plays a role in RBC development

A long non-coding RNA (lncRNA) regulates programmed cell death during one of the final stages of red blood cell differentiation, according to Whitehead Institute researchers. This is the first time a lncRNA has been found to play a role in red blood cell development and the first time a lncRNA has been shown to affect programmed cell death.

8 Dec 2011

ASH recognizes abstract presenters at 53rd annual meeting in San Diego

The American Society of Hematology (ASH) recognizes the following abstract presenters at the 53rd ASH annual meeting in San Diego, CA, with the highest scoring abstracts in the categories of undergraduate student, medical student, graduate student, resident physician, and postdoctoral fellow.

2 Dec 2011

New method reveals complete set of aberrant signaling pathways that give rise to cancers

One major obstacle in the fight against cancer is that anticancer drugs often affect normal cells in addition to tumor cells, resulting in significant side effects. Yet research into development of less harmful treatments geared toward the targeting of specific cancer-causing mechanisms is hampered by lack of knowledge of the molecular pathways that drive cancers in individual patients.

2 Dec 2011

Enrollment complete in Ceregene's CERE-120 Phase 2b trial for Parkinson's

Ceregene, Inc. reported today that it has completed enrollment in its Phase 2b trial of CERE-120, a gene therapy product expressing the neurturin (NRTN) gene for the treatment of Parkinson's disease.

1 Dec 2011

Study reveals no clear advantage of one implant type over another

More than 270,000 Americans get hip replacement surgeries every year -- a number that is projected to double in the next decade as the population ages. With various options for implants, including metal-on-polyethylene, metal-on-metal and ceramic-on-ceramic, there have been questions about which kind works best.

1 Dec 2011

Combination of two techniques promises to improve effectiveness of experimental gene therapies

yeast or phages act not only to tag and target but also to exchange specific genes in DNA A combination of two techniques promises to improve the efficiency and effectiveness of experimental gene therapies, while also reducing potential side effects says a new research report published in the December 2011 issue of the FASEB Journal.

1 Dec 2011

New gene therapy approach safe for patients with muscular dystrophy

Researchers at the University of North Carolina at Chapel Hill have shown that it is safe to cut and paste together different viruses in an effort to create the ultimate vehicle for gene therapy. In a phase I clinical trial, the investigators found no side effects from using a "chimeric" virus to deliver replacement genes for an essential muscle protein in patients with muscular dystrophy.

1 Dec 2011

Cases of two AIDS patients renew hope of AIDS cure for many scientists, New York Times reports

The New York Times profiles two AIDS patients whose cases "suggest to many scientists that curing AIDS may be achievable," according to the newspaper. "One man, the so-called Berlin patient, apparently has cleared his HIV infection, albeit by arduous bone marrow transplants," and the other, "a 50-year-old man in Trenton, N.J., underwent a far less difficult gene therapy procedure. While he was not cured, his body was able to briefly control the virus after he stopped taking the usual antiviral drugs, something that is highly unusual," the newspaper writes.

29 Nov 2011

Scientists use genetic methods to successfully repair cleft lips in mice embryos

Scientists at Weill Cornell Medical College used genetic methods to successfully repair cleft lips in mice embryos specially engineered for the study of cleft lip and cleft palate. The research breakthrough may show the way to prevent or treat the conditions in humans.

29 Nov 2011

Study reveals specific functional deficit in inner ear mechanotransduction channels

National Institutes of Health-funded researchers have identified two proteins that may be the key components of the long-sought after mechanotransduction channel in the inner ear-the place where the mechanical stimulation of sound waves is transformed into electrical signals that the brain recognizes as sound.

22 Nov 2011

Gene-therapy trial to restore hearing in deaf mice

Researchers have found long-sought genes in the sensory hair cells of the inner ear that, when mutated, prevent sound waves from being converted to electric signals - a fundamental first step in hearing. The team, co-led by Jeffrey Holt, PhD, in the department of otolaryngology at Children-s Hospital Boston, and Andrew Griffith, MD, PhD, of the NIH-s National Institute on Deafness and other Communication Disorders (NIDCD), then restored these electrical signals in the sensory cells of deaf mice by introducing normal genes.

22 Nov 2011

Older home health care patients are prescribed PIMs three times higher than others

Older adults receiving home health care may be taking a drug that is unsafe or ineffective for someone their age. In fact, nearly 40 percent of seniors receiving medical care from a home health agency are taking at least one prescription medication that is considered potentially inappropriate to seniors, a new study in the Journal of General Internal Medicine has revealed.

22 Nov 2011

Modified spider silk proteins for cancer gene therapy

A new episode in the American Chemical Society's (ACS) award-winning "Global Challenges/Chemistry Solutions" podcast series spins a real-life tale in which spider silk shows promise for overcoming a major barrier to the use of gene therapy in everyday medicine.

17 Nov 2011

Aurora kinase inhibitor may treat neuroendocrine prostate cancer

An international team of researchers led by clinicians at Weill Cornell Medical College have discovered a genetic Achilles' heel in an aggressive type of prostate cancer -- a vulnerability they say can be attacked by a targeted drug that is already in clinical trials to treat other types of cancers.

17 Nov 2011

CF Foundation, Genzyme announce new drug research agreement

Genzyme, a Sanofi company, and Cystic Fibrosis Foundation Therapeutics Inc., the nonprofit affiliate of the Cystic Fibrosis Foundation, today announced a research agreement to support the discovery of new drugs to treat people with the most common mutation found in patients with CF, Delta F508.

17 Nov 2011

EMA grants orphan designation to AMT for hemophilia B gene therapy

Amsterdam Molecular Therapeutics, a leader in the field of human gene therapy, announced today that the European Medicines Agency (EMA) has granted orphan designation for its gene therapy program for the treatment of hemophilia B. Orphan designation in the European Union provides several benefits including 10 years of market exclusivity from product launch and access to the central authorization procedure.

16 Nov 2011

EPO-secreting engineered vessels could reverse anemia

Patients who rely on recombinant, protein-based drugs must often endure frequent injections, often several times a week, or intravenous therapy. Researchers at Children's Hospital Boston demonstrate the possibility that blood vessels, made from genetically engineered cells, could secrete the drug on demand directly into the bloodstream.

15 Nov 2011

Gene Therapy News and Research

Further Reading

New research on diagnosis and treatment of leukemia to be presented at 53rd ASH meeting

Gene therapy can reduce painful bleeding episodes in patients with hemophilia B

New gene editing technique can heal sickle cell patients with their own cells

Research: lncRNA affects apoptosis and plays a role in RBC development

ASH recognizes abstract presenters at 53rd annual meeting in San Diego

New method reveals complete set of aberrant signaling pathways that give rise to cancers

Enrollment complete in Ceregene's CERE-120 Phase 2b trial for Parkinson's

Study reveals no clear advantage of one implant type over another

Combination of two techniques promises to improve effectiveness of experimental gene therapies

New gene therapy approach safe for patients with muscular dystrophy

Cases of two AIDS patients renew hope of AIDS cure for many scientists, New York Times reports

Scientists use genetic methods to successfully repair cleft lips in mice embryos

Study reveals specific functional deficit in inner ear mechanotransduction channels

Gene-therapy trial to restore hearing in deaf mice

Older home health care patients are prescribed PIMs three times higher than others

Modified spider silk proteins for cancer gene therapy

Aurora kinase inhibitor may treat neuroendocrine prostate cancer

CF Foundation, Genzyme announce new drug research agreement

EMA grants orphan designation to AMT for hemophilia B gene therapy

EPO-secreting engineered vessels could reverse anemia

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