Gene Therapy

Category: Gene Therapy

Treating the rare disease MPS I is a challenge. MPS I, caused by the deficiency of a key enzyme called IDUA, eventually leads to the abnormal accumulation of certain molecules and cell death.

Researchers from Salk Institute for Biological Studies, BGI, and other institutes for the first time evaluated the safety and reliability of the existing targeted gene correction technologies, and successfully developed a new method, TALEN-HDAdV, which could significantly increased gene-correction efficiency in human induced pluripotent stem cell (hiPSC). This study published online in Cell Stell Cell provides an important theoretical foundation for stem cell-based 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.

For the first time, researchers have combined gene therapy and stem cell transplantation to successfully reverse the severe, crippling bleeding disorder hemophilia A in large animals, opening the door to the development of new therapies for human patients.

National Institutes of Health-funded scientists have corrected sickle cell disease in adult laboratory mice by activating production of a special blood component normally produced before, but not after, birth.

A multi-center gene therapy trial for patients with advanced Parkinson’s disease demonstrated reduced symptoms of the progressive movement disorder, according to a new study published in Lancet Neurology. The study was designed to deliver the gene for glumatic acid decarboxylase (GAD) packaged in inert viral vectors into an area of the brain called the subthalamic nucleus. GAD makes an important inhibitory chemical called GABA. The subthalamic nucleus is abnormally activated in Parkinson’s disease and this activity leads to the debilitating movement problems. The idea of the gene therapy is that the billions of AAV-2 GAD viral vectors delivered into the subthalamic nucleus will increase GABA, thereby quieting this brain region.

Gene TherapyJanuary 12, 2011 01:23 AM

One of the challenges of gene therapy - a set of methodologies aimed at treating several nucleic acid diseases (DNA or RNA) - is to assure that this material arrives directly to the nucleus of the cell without losing a substantial amount along the way and without producing any undesired side effects. With this aim, scientists experiment with the use of different types of vectors, molecules capable of transporting genetic material to the correct place. Presently, natural "deactivated" viruses are the most commonly used vectors in clinical trials, their side effects however often limit therapeutic application.

It is common for cancer cells to find some way to disarm p53, also known as "guardian of the genome" due to its action in preventing defective cells from dividing. "The critical importance of the protective function of p53 is underscored by the diversity of molecular strategies employed by cancer cells to subvert p53 activity, such as overexpression of antagonistic proteins like HDM2 and HDMX," explains senior study author Dr. Loren D. Walensky from Harvard Medical School. "Restoration of p53 activity remains an important goal in the quest for more effective cancer therapeutics."

An immune reaction to dystrophin, the muscle protein that is defective in patients with Duchenne muscular dystrophy, may pose a new challenge to strengthening muscles of patients with this disease, suggests a new study appearing in the October 7, 2010, issue of The New England Journal of Medicine.

In one of only two studies of its kind, a study from researchers at Tufts University School of Medicine and the Sackler School of Graduate Biomedical Sciences at Tufts demonstrates that non-viral gene therapy can delay the onset of some forms of eye disease and preserve vision. The team developed nanoparticles to deliver therapeutic genes to the retina and found that treated mice temporarily retained more eyesight than controls. The study, published online in advance of print in Molecular Therapy, brings researchers closer to a non-viral gene therapy treatment for degenerative eye disorders.

Italian scientists pioneering a new gene transfer treatment for the blood disorder β-thalassemia have successfully completed preclinical trials, claiming they can correct the lack of beta-globin (ß-globin) in patients' blood cells which causes the disease. The research, published in EMBO Molecular Medicine, reveals how gene therapy may represent a safe alternative to current cures that are limited to a minority of patients.

Scientists have designed a nanoparticle that appears to effectively deliver genetic material into cells with minimal toxic effects.

EUREKA project E! 3371 Gene Transfer Agents has made great advances in the development of novel non-viral carriers able to introduce genetic material into the target cells. These new agents, derivatives of cationic amphiphilic 1,4-dihydropyridine (1,4-DHP), avoid the problems of the recipient's immune system reacting against a viral carrier. The project partners have developed methods to produce them in large amounts, which solves another of the problems with viral delivery. But the greatest advantage is that the new compounds are significantly more effective at delivering DNA into cell nuclei than other standard synthetic carriers; increasing the chance of the DNA successfully controlling the defective genes, and the disease.

Researchers have developed an experimental cure for Type 1 diabetes, a disease that affects about one in every 400 to 600 children and adolescents. They will present their results in a mouse model of Type 1 diabetes on Sunday at The Endocrine Society's 92nd Annual Meeting in San Diego.

Researchers at Mount Sinai School of Medicine have developed a new gene therapy that is safe and effective in reversing advanced heart failure. SERCA2a (produced as MYDICAR®) is a gene therapy designed to stimulate production of an enzyme that enables the failing heart to pump more effectively. In a Phase II study, SERCA2a injection through a routine minimally invasive cardiac catheterization was safe and showed clinical benefit in treating this patient population and decreasing the severity of heart failure. The data were presented this week at the Heart Failure Congress of the European Society of Cardiology in Berlin.

Blood vessel blockage, a common condition in old age or diabetes, leads to low blood flow and results in low oxygen, which can kill cells and tissues. Such blockages can require amputation resulting in loss of limbs. Now, using mice as their model, researchers at Johns Hopkins have developed therapies that increase blood flow, improve movement and decrease tissue death and the need for amputation. The findings, published online last week in the early edition of the Proceedings of the National Academy of Sciences, hold promise for developing clinical therapies.

Born with a retinal disease that made him legally blind, and would eventually leave him totally sightless, the nine-year-old boy used to sit in the back of the classroom, relying on the large print on an electronic screen and assisted by teacher aides. Now, after a single injection of genes that produce light-sensitive pigments in the back of his eye, he sits in front with classmates and participates in class without extra help. In the playground, he joins his classmates in playing his first game of softball.

Over-expressing a gene that lets brain cells communicate just a fraction of a second longer makes a smarter rat, report researchers from the Medical College of Georgia and East China Normal University.

Scientists from the Universities of Michigan and Minnesota show in a research report published online in the FASEB Journal (http://www.fasebj.org) that gene therapy may be used to improve an ailing heart's ability to contract properly. In addition to showing gene therapy's potential for reversing the course of heart failure, it also offers a tantalizing glimpse of a day when "closed heart surgery" via gene therapy is as commonly prescribed as today's cocktail of drugs.

An international team of scientists and clinicians from the United States and Saudi Arabia are working to develop gene therapy for treating a rare, hereditary retinal disease. The therapy has been shown to restore lost vision in animal models of retinitis pigmentosa (RP). Their work is being funded in part by a $1.5 million grant from the Prince Salman Center for Disability Research in Saudi Arabia, where the recessive gene mutation that leads to the eye disease RP has been found in children from several families.

University of Florida researchers have come up with a new gene therapy method to disrupt cancer growth by using a synthetic protein to induce blood clotting that cuts off a tumor's blood and nutrient supply.

With obesity reaching epidemic levels, researchers at the Ohio State University Medical Center are studying a potentially long-term treatment that involves injecting a gene directly into one of the critical feeding and weight control centers of the brain.

A new UCLA AIDS Institute study has found that gene therapy can be developed as a safe and active technique to combat HIV.

Long-term gene therapy resulted in improved cardiac function and reversed deterioration of the heart in rats with heart failure, according to a recent study conducted by researchers at Thomas Jefferson University's Center for Translational Medicine. The study was published online in Circulation.

Scientists at the University of Michigan have shown that gene therapy can be used to successfully stop the development of periodontal disease, the leading cause of tooth loss in adults.

The apparent success of a case in which German doctors cured a man of AIDS using a bone marrow transplant comes as no surprise to Gerhard Bauer, a UC Davis stem cell researcher. Bauer has been working for more than 10 years on a similar cure for AIDS based on replacing the devastated immune system of an HIV-infected patient with stem cells that have been engineered to resist human immunodeficiency syndrome.

Using a harmless virus to insert a corrective gene into mouse blood cells, scientists at St. Jude Children's Research Hospital have alleviated sickle cell disease pathology. In their studies, the researchers found that the treated mice showed essentially no difference from normal mice. Although the scientists caution that applying the gene therapy to humans presents significant technical obstacles, they believe that the new therapy will become an important treatment for the disease.

Massachusetts General Hospital (MGH) researchers have used gene therapy to restore useful vision to mice with degeneration of the light-sensing retinal rods and cones, a common cause of human blindness. Their report, appearing in the Oct. 14 Proceedings of the National Academy of Sciences, describes the effects of broadly expressing a light-sensitive protein in other neuronal cells found throughout the retina.


The gene transfer vector (left) is injected into the skin in the area of pain (red line, right). From the skin, the vector is carried into sensory nerves and releases the inhibitory neurotransmitter locally in the spinal cord (red oval). Credit: University of Michigan
This week, University of Michigan scientists will begin a phase 1 clinical trial for the treatment of cancer-related pain, using a novel gene transfer vector injected into the skin to deliver a pain-relieving gene to the nervous system.

A new approach to treating vision loss caused by Type 1 Usher syndrome (USH1), the most common condition affecting both sight and hearing, will be unveiled by a scientist at the annual conference of the European Society of Human Genetics tomorrow (Tuesday 3 June). Ms Annie Rebibo Sabbah, from the Genetics Department of the Rappaport Faculty of Medicine, Technion, Haifa, Israel, will tell the conference that preliminary results using a class of drugs called aminoglycosides, commonly used as antibiotics, had had promising effects in vitro and in cell culture.

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