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.

Researchers develop cardiac tissue from human stem cells to find cures for heart diseases

When it comes to finding cures for heart disease, scientists at Icahn School of Medicine at Mount Sinai are working to their own beat. They may have developed a tissue model for the human heart that can bridge the gap between animal models and human clinical trial patients.

4 Feb 2014

Ronald G. Crystal wins Pioneer Award from Human Gene Therapy

In recognition of his seminal work on adenoviral vectors, which accelerated the translation of gene therapy from the research laboratory to the clinic, Ronald G. Crystal, MD (Weill Cornell Medical College, Cornell University, New York City), has received a Pioneer Award from Human Gene Therapy, a peer-reviewed journal from Mary Ann Liebert, Inc., publishers.

31 Jan 2014

FARA funds gene therapy-based research project to tackle Friedreich's ataxia

The gene therapy-based research project to tackle Friedreich's ataxia launched in November in labs at the Institute for Research in Biomedicine, in Barcelona, and the "Centro de Biolog-a Molecular Severo Ochoa", in Madrid, has received 100,000 US$ per year for two years from the Friedreich's Ataxia Research Alliance.

29 Jan 2014

Use of twice-daily pill could turn deadly blood cancer into highly treatable disease

Use of a twice-daily pill could turn a deadly blood cancer into a highly treatable disease, according to scientists at Weill Cornell Medical College who led a multinational research team.

24 Jan 2014

CUMC researchers identify gene that appears to play major role in motor neuron degeneration in ALS

Columbia University Medical Center researchers have identified a gene, called matrix metalloproteinase-9 (MMP-9), that appears to play a major role in motor neuron degeneration in amyotrophic lateral sclerosis, also known as Lou Gehrig's disease. The findings, made in mice, explain why most but not all motor neurons are affected by the disease and identify a potential therapeutic target for this still-incurable neurodegenerative disease. The study was published today in the online edition of the journal Neuron.

23 Jan 2014

Clinician scientist receives RPB Career Development Award to support eye research

The Harvard Medical School (HMS) Department of Ophthalmology has been granted a $250,000 Research to Prevent Blindness (RPB) Career Development Award to support eye research conducted by Jason I. Comander, M.D., Ph.D. The support will be provided over a four-year period.

22 Jan 2014

Chinese and American researchers introduce new approach to gene therapy

Resistance of tumor cells toward multiple cytostatic drugs is a serious problem in cancer treatment. In the journal Angewandte Chemie, a team of Chinese and American researchers has now introduced a new approach to gene therapy that could counter this problem: The gene that codes for resistance is “silenced” through the use of an ingenious nanocomplex.

22 Jan 2014

Gene therapy edges closer for Parkinson’s disease

Lentiviral vector-based gene therapy is well tolerated and may improve motor function in patients with Parkinson’s disease, shows an open-label study published in The Lancet.

20 Jan 2014

Gene therapies correct three forms of retinal degenerative diseases

Gene therapies developed by University of Pennsylvania School of Veterinary Medicine researchers have worked to correct different forms of blindness. While effective, the downside to these approaches to vision rescue is that each disease requires its own form of gene therapy to correct the particular genetic mutation involved, a time consuming and complex process.

17 Jan 2014

New gene therapy restores some sight in people born with inherited form of blindness

A new gene therapy has restored some sight in people born with an inherited, progressive form of blindness. The technique replaces a defective gene in the eye with a normal working copy of the gene using a single injection.

16 Jan 2014

Cedars-Sinai investigators identify new molecular abnormalities in diabetic cornea

Investigators at the Cedars-Sinai Regenerative Medicine Institute have identified new molecular abnormalities in the diabetic cornea that could contribute to eye problems in affected patients.

16 Jan 2014

Cedars-Sinai investigators identify new molecular abnormalities in diabetic cornea

Investigators at the Cedars-Sinai Regenerative Medicine Institute have identified new molecular abnormalities in the diabetic cornea that could contribute to eye problems in affected patients. With this new knowledge, investigators aim to accelerate the process of healing and repair in damaged corneas to ultimately reverse the effects of diabetes-induced eye complications.

15 Jan 2014

UMMS researchers commissioned by Alpha-1 Project to develop PiZ antibody

Researchers at the University of Massachusetts Medical School (UMMS) have been commissioned by the Alpha-1 Project (TAP) to develop a PiZ antibody. The antibody will be used to track the presence of mutant alpha-1 PiZ protein in human blood serum, an essential tool in testing potential therapies for Alpha-1 Antitrypsin Deficiency (Alpha-1).

13 Jan 2014

Weill Cornell Medical College received a $75M grant to expand, enhance cancer research, care programs

Weill Cornell Medical College announced today that it has received a $75 million gift from Sandra and Edward Meyer and the Sandra and Edward Meyer Foundation to expand and enhance the medical college's distinguished cancer research and care programs.

9 Jan 2014

Study sheds light on the biological underpinnings of obesity

A new neuroscience study sheds light on the biological underpinnings of obesity. The in vivo study, published in the January 8 issue of the Journal of Neuroscience, reveals how a protein in the brain helps regulate food intake and body weight. The findings reveal a potential new avenue for the treatment of obesity and may help explain why medications that are prescribed for epilepsy and other conditions that interfere with this protein, such as gabapentin and pregabalin, can cause weight gain.

7 Jan 2014

New ways to treat substance abuse, neurological diseases and mental illnesses

Researchers at the University at Buffalo have found a way to change alcohol drinking behavior in rodents, using the emerging technique of optogenetics, which uses light to stimulate neurons.

7 Jan 2014

Alliance for Cancer Gene Therapy grants nearly $25M to conduct cancer related gene therapy research

As the potential of gene and cell therapy in fighting cancers grows and oncologists across the nation explore the burgeoning science, doctors have uncovered a stunning development – cancers can be treated using patients' own cells as a weapon, and can eliminate the harrowing, often debilitating effects of chemotherapy, radiation and surgery.

28 Dec 2013

Stem cell-based gene therapy shows long-term outcomes for treatment of genetic skin diseases

Stem cell-based gene therapy holds promise for the treatment of devastating genetic skin diseases, but the long-term clinical outcomes of this approach have been unclear.

27 Dec 2013

Cell transplantation, gene therapy combination to open new therapeutic avenues for Alzheimer's disease

In a recent study published in the Neural Regeneration Research (Vol. 8, No. 33, 2013), Prof. Feng Li and team from Zhongshan School of Medicine, Sun Yat-sen University in China, synthesized a 19-nt oligonucleotide targeting BACE1, the key enzyme in amyloid beta protein (Aβ) production, and introduced it into the pSilenCircle vector to construct a short hairpin (shRNA) expression plasmid against the BACE1 gene.

24 Dec 2013

Researchers report new gene delivery method that targets tumor blood vessels

Working in mice, researchers at Washington University School of Medicine in St. Louis report developing a gene delivery method long sought in the field of gene therapy: a deactivated virus carrying a gene of interest that can be injected into the bloodstream and make its way to the right cells.

24 Dec 2013

Gene Therapy News and Research

Further Reading

Researchers develop cardiac tissue from human stem cells to find cures for heart diseases

Ronald G. Crystal wins Pioneer Award from Human Gene Therapy

FARA funds gene therapy-based research project to tackle Friedreich's ataxia

Use of twice-daily pill could turn deadly blood cancer into highly treatable disease

CUMC researchers identify gene that appears to play major role in motor neuron degeneration in ALS

Clinician scientist receives RPB Career Development Award to support eye research

Chinese and American researchers introduce new approach to gene therapy

Gene therapy edges closer for Parkinson’s disease

Gene therapies correct three forms of retinal degenerative diseases

New gene therapy restores some sight in people born with inherited form of blindness

Cedars-Sinai investigators identify new molecular abnormalities in diabetic cornea

Cedars-Sinai investigators identify new molecular abnormalities in diabetic cornea

UMMS researchers commissioned by Alpha-1 Project to develop PiZ antibody

Weill Cornell Medical College received a $75M grant to expand, enhance cancer research, care programs

Study sheds light on the biological underpinnings of obesity

New ways to treat substance abuse, neurological diseases and mental illnesses

Alliance for Cancer Gene Therapy grants nearly $25M to conduct cancer related gene therapy research

Stem cell-based gene therapy shows long-term outcomes for treatment of genetic skin diseases

Cell transplantation, gene therapy combination to open new therapeutic avenues for Alzheimer's disease

Researchers report new gene delivery method that targets tumor blood vessels

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