http://www.sciencedaily.com/news/health_medicine/gene_therapy/ Read all about gene therapy including current medical research on switching therapeutic genes on and off, light-activated gene therapy and gene silencing. en-us Sat, 26 May 2012 13:05:01 EDT Sat, 26 May 2012 13:05:01 EDT 60 http://www.sciencedaily.com/images/logosmall.gif http://www.sciencedaily.com/news/health_medicine/gene_therapy/ For more science articles, visit ScienceDaily. http://www.sciencedaily.com/releases/2012/05/120524123023.htm Scientists found that loss of the ADA gene directly contributes to B cell tolerance problems and that these defects are mostly corrected after gene therapy. Thu, 24 May 2012 12:30:30 EDT http://www.sciencedaily.com/releases/2012/05/120524123023.htm http://www.sciencedaily.com/releases/2012/05/120516152444.htm Taiwanese doctors have restored some movement in four children bedridden with a rare, life-threatening neurological disease using gene transfer. The first-in-humans achievement may also be helpful for more common diseases such as Parkinson's. Wed, 16 May 2012 15:24:24 EDT http://www.sciencedaily.com/releases/2012/05/120516152444.htm http://www.sciencedaily.com/releases/2012/05/120510114107.htm Imagine being able to control genetic expression by flipping a light switch. Researchers are using light-activated molecules to turn gene expression on and off. Their method enables greater precision when studying gene function, and could lead to targeted therapies for diseases like cancer. Thu, 10 May 2012 11:41:41 EDT http://www.sciencedaily.com/releases/2012/05/120510114107.htm http://www.sciencedaily.com/releases/2012/03/120329141923.htm A DICER protein, known to produce tiny RNAs in cells, also helps complete an important step in gene expression, according to research on Arabidopsis thaliana. The expression of a gene requires activation via a promoter or an external trigger. Plant research to be published in Science helps to show that later stages of transcription are just as important. This is likely to apply to other organisms, including humans. Thu, 29 Mar 2012 14:19:19 EDT http://www.sciencedaily.com/releases/2012/03/120329141923.htm http://www.sciencedaily.com/releases/2012/03/120329124613.htm The world's first bedside genetic test has been developed. Scientists reports on the use of a simple cheek swab test, the Spartan RX CYP2C19, performed by nurses at the patient's bedside. This revolutionary technology allows doctors to rapidly identify patients with a genetic variant known as CYP2C19*2. Thu, 29 Mar 2012 12:46:46 EDT http://www.sciencedaily.com/releases/2012/03/120329124613.htm http://www.sciencedaily.com/releases/2012/03/120327215700.htm Scientists have designed what appears to be a powerful gene therapy strategy that can treat both beta-thalassemia disease and sickle cell anemia. They have also developed a test to predict patient response before treatment. Tue, 27 Mar 2012 21:57:57 EDT http://www.sciencedaily.com/releases/2012/03/120327215700.htm http://www.sciencedaily.com/releases/2012/03/120318143904.htm Researchers have revealed how a mutation in a single gene is responsible for the inability of neurons to effectively pass along appetite suppressing signals from the body to the right place in the brain. What results is obesity caused by a voracious appetite. Sun, 18 Mar 2012 14:39:39 EDT http://www.sciencedaily.com/releases/2012/03/120318143904.htm http://www.sciencedaily.com/releases/2012/02/120201092918.htm Scientists have found that a rare dyslexia-linked genetic variant of the ROBO1 gene decreases normal crossing of auditory pathways in the human brain. The results link, for the first time, a dyslexia-susceptibility gene to a specific sensory function of the human brain. Wed, 01 Feb 2012 09:29:29 EST http://www.sciencedaily.com/releases/2012/02/120201092918.htm http://www.sciencedaily.com/releases/2012/01/120124113203.htm The time for commercial development of gene therapy has come. Patients with diseases treatable and curable with gene therapy deserve access to the technology, which has demonstrated both its effectiveness and feasibility, says researchers. Tue, 24 Jan 2012 11:32:32 EST http://www.sciencedaily.com/releases/2012/01/120124113203.htm http://www.sciencedaily.com/releases/2012/01/120123163412.htm A new gene therapy has the potential to treat a common form of blindness that strikes both youngsters and adults. The technique works by replacing a malfunctioning gene in the eye with a normal working copy that supplies a protein necessary for light-sensitive cells in the eye to function. Several complex steps remain before the gene therapy technique can be used in humans, but once at that stage, it has great potential to change lives. Mon, 23 Jan 2012 16:34:34 EST http://www.sciencedaily.com/releases/2012/01/120123163412.htm http://www.sciencedaily.com/releases/2012/01/120105141141.htm Researchers have solved the three-dimensional structure of a newly discovered type of gene-targeting protein that has shown to be useful as a DNA-targeting molecule for gene correction, gene therapy and gene modification. Thu, 05 Jan 2012 14:11:11 EST http://www.sciencedaily.com/releases/2012/01/120105141141.htm http://www.sciencedaily.com/releases/2011/12/111220172626.htm New research has investigated if nerve growth factor gene therapy can prevent diabetic heart failure and small vascular disease in mice. Tue, 20 Dec 2011 17:26:26 EST http://www.sciencedaily.com/releases/2011/12/111220172626.htm http://www.sciencedaily.com/releases/2011/12/111220154047.htm Women who are at risk for breast cancer may also be at greater risk for heart disease, new research has found. Tue, 20 Dec 2011 15:40:40 EST http://www.sciencedaily.com/releases/2011/12/111220154047.htm http://www.sciencedaily.com/releases/2011/12/111219135021.htm Gene therapy may someday in the future replace the use of implants in deaf people. The carrier for this gene medicine may be derived from shrimp shells. Mon, 19 Dec 2011 13:50:50 EST http://www.sciencedaily.com/releases/2011/12/111219135021.htm http://www.sciencedaily.com/releases/2011/12/111212153121.htm A new study has identified a gene that plays a major role in maintaining clarity of the cornea in humans and mice -- and could possibly be used as gene therapy to treat diseases that cause blindness. Mon, 12 Dec 2011 15:31:31 EST http://www.sciencedaily.com/releases/2011/12/111212153121.htm http://www.sciencedaily.com/releases/2011/12/111207175730.htm Researchers 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. Wed, 07 Dec 2011 17:57:57 EST http://www.sciencedaily.com/releases/2011/12/111207175730.htm http://www.sciencedaily.com/releases/2011/11/111130115822.htm New research demonstrates how a combination of two techniques improves the efficiency of experimental gene therapies, while reducing side effects. Scientists combined techniques involving site-specific recombinases that facilitate the exchange of genetic material between DNA strands, to guide where new genetic material is inserted into a cell's DNA. This approach to gene therapy represents an important advance, and has the potential to correct root causes of numerous illnesses. Wed, 30 Nov 2011 11:58:58 EST http://www.sciencedaily.com/releases/2011/11/111130115822.htm http://www.sciencedaily.com/releases/2011/11/111130115804.htm Researchers 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. Wed, 30 Nov 2011 11:58:58 EST http://www.sciencedaily.com/releases/2011/11/111130115804.htm http://www.sciencedaily.com/releases/2011/11/111114095721.htm Patients with non-small cell lung cancer who have mutations in the KRAS gene should respond well to the antifolate class of drugs, according to results of a recent study comparing human lung cancer cell lines and patients. Mon, 14 Nov 2011 09:57:57 EST http://www.sciencedaily.com/releases/2011/11/111114095721.htm http://www.sciencedaily.com/releases/2011/11/111103081434.htm 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. Thu, 03 Nov 2011 08:14:14 EDT http://www.sciencedaily.com/releases/2011/11/111103081434.htm http://www.sciencedaily.com/releases/2011/11/111102125357.htm Researchers have developed a viral vector designed to deliver a gene into the eyes of people born with an inherited, progressive form of blindness that affects mainly males. The vector is being tested in a clinical trial investigating the use of gene therapy to cure choroideremia, a disease that affects an estimated 100,000 people worldwide. Wed, 02 Nov 2011 12:53:53 EDT http://www.sciencedaily.com/releases/2011/11/111102125357.htm http://www.sciencedaily.com/releases/2011/11/111102093037.htm Scientists reveal the actions of a gene implicated in Batten disease, a rare, degenerative childhood disorder. Wed, 02 Nov 2011 09:30:30 EDT http://www.sciencedaily.com/releases/2011/11/111102093037.htm http://www.sciencedaily.com/releases/2011/10/111012124158.htm A new study could lead to improved gene therapies for conditions such as heart disease and cancer as well as more effective vaccines for tuberculosis, malaria and other diseases. Wed, 12 Oct 2011 12:41:41 EDT http://www.sciencedaily.com/releases/2011/10/111012124158.htm http://www.sciencedaily.com/releases/2011/09/110930153050.htm Scientists have achieved temporary functional preservation of photoreceptors in a mouse model for retinitis pigmentosa using novel bipartite gene therapy. Fri, 30 Sep 2011 15:30:30 EDT http://www.sciencedaily.com/releases/2011/09/110930153050.htm http://www.sciencedaily.com/releases/2011/09/110926104607.htm A genetic variant may explain why some people with asthma do not respond well to inhaled corticosteroids, the most widely prescribed medicine for long-term asthma control. Researchers found that asthma patients who have two copies of a specific gene variant responded only one-third as well to steroid inhalers as those with two copies of the regular gene. Mon, 26 Sep 2011 10:46:46 EDT http://www.sciencedaily.com/releases/2011/09/110926104607.htm http://www.sciencedaily.com/releases/2011/09/110908081005.htm Spontaneous ordering of DNA fragments in a special matrix holds the key to creating non-toxic gene therapy delivery vectors, according to a new study. Thu, 08 Sep 2011 08:10:10 EDT http://www.sciencedaily.com/releases/2011/09/110908081005.htm http://www.sciencedaily.com/releases/2011/09/110907132050.htm Researchers have identified a new drug target that may treat and/or prevent heart failure. The team evaluated failing human and pig hearts and discovered that SUMO1, a so-called "chaperone" protein that regulates the activity of key transporter genes, was decreased in failing hearts. When the researchers injected SUMO1 into these hearts via gene therapy, cardiac function was significantly improved. Wed, 07 Sep 2011 13:20:20 EDT http://www.sciencedaily.com/releases/2011/09/110907132050.htm http://www.sciencedaily.com/releases/2011/09/110906104837.htm Biomaterials may prove key to healing chronic wounds in diabetic patients, new research suggests. Tue, 06 Sep 2011 10:48:48 EDT http://www.sciencedaily.com/releases/2011/09/110906104837.htm http://www.sciencedaily.com/releases/2011/09/110905090001.htm Researchers have discovered a gene that when mutated can cause lymphedema (swollen limbs due to a failure of the lymph system), immune abnormalities, deafness and leukemia. The identification of the gene responsible for causing this rare combination of medical conditions, known as Emberger syndrome, could allow earlier identification and treatment of those at risk. Mon, 05 Sep 2011 09:00:00 EDT http://www.sciencedaily.com/releases/2011/09/110905090001.htm http://www.sciencedaily.com/releases/2011/08/110815172117.htm Low oxygen can silence the BRCA1 tumor suppressor gene and contribute to the progression of cancer, according to a new study. Silencing this particular gene is one of the steps on the malignant pathway to breast cancer. The research may ultimately lead to ways of reactivating this and other tumor suppressor genes, in order to thwart cancer. Mon, 15 Aug 2011 17:21:21 EDT http://www.sciencedaily.com/releases/2011/08/110815172117.htm http://www.sciencedaily.com/releases/2011/08/110815162233.htm An early phase clinical trial has shown that a form of gene therapy is safe even when combined with radiation therapy for treating brain tumors such as glioblastoma multiforme, the most common and dangerous form of brain cancer. The novel treatment uses an adenovirus vector that is taken up by cancer cells where it activates a drug that kills the cells. Mon, 15 Aug 2011 16:22:22 EDT http://www.sciencedaily.com/releases/2011/08/110815162233.htm http://www.sciencedaily.com/releases/2011/08/110815152037.htm In a kind of molecular gymnastics, scientists have devised a gene therapy cocktail that has the potential to treat some inherited diseases associated with "misfolded" proteins. Mon, 15 Aug 2011 15:20:20 EDT http://www.sciencedaily.com/releases/2011/08/110815152037.htm http://www.sciencedaily.com/releases/2011/08/110811094836.htm Scientists have mimicked the ways viruses infect human cells and deliver their genetic material. The research hopes to apply the approach to gene therapy -- a therapeutic strategy to correct defective genes such as those that cause cancer. Thu, 11 Aug 2011 09:48:48 EDT http://www.sciencedaily.com/releases/2011/08/110811094836.htm http://www.sciencedaily.com/releases/2011/08/110810101559.htm Genetically engineered spider silk could help overcome a major barrier to the use of gene therapy in everyday medicine, according to a new study. Wed, 10 Aug 2011 10:15:15 EDT http://www.sciencedaily.com/releases/2011/08/110810101559.htm http://www.sciencedaily.com/releases/2011/08/110809162014.htm Researchers have found that a cancer-causing fusion protein works by silencing the tumor suppressor gene IL-2R common gamma-chain. The results suggest news targets for lymphoma and other types of cancer. Tue, 09 Aug 2011 16:20:20 EDT http://www.sciencedaily.com/releases/2011/08/110809162014.htm http://www.sciencedaily.com/releases/2011/07/110725123543.htm Although spinal muscular atrophy (SMA) is caused by the loss of a specific gene, all infants and children with SMA have an untouched highly similar gene within their genetic make up. Activation of this copy gene has the potential to treat SMA, and now researchers report the strongest such activation yet observed with attendant benefit on mice genetically engineered to have SMA. Mon, 25 Jul 2011 12:35:35 EDT http://www.sciencedaily.com/releases/2011/07/110725123543.htm http://www.sciencedaily.com/releases/2011/06/110628163323.htm Researchers have found in a Phase II trial that a new gene therapy stabilized or improved cardiac function in people with severe heart failure. Patients receiving a high dose of the therapy, called SERCA2a, experienced substantial clinical benefit and significantly reduced cardiovascular hospitalizations, addressing a critical unmet need in this population. Tue, 28 Jun 2011 16:33:33 EDT http://www.sciencedaily.com/releases/2011/06/110628163323.htm http://www.sciencedaily.com/releases/2011/06/110627134532.htm Novel tools and methods for delivering therapeutic genes to cells in the central nervous system hold great promise for the development of new treatments to combat incurable neurologic diseases. Mon, 27 Jun 2011 13:45:45 EDT http://www.sciencedaily.com/releases/2011/06/110627134532.htm http://www.sciencedaily.com/releases/2011/06/110626145318.htm Using an innovative gene therapy technique called genome editing that hones in on the precise location of mutated DNA, scientists have treated the blood clotting disorder hemophilia in mice. This is the first time that genome editing, which precisely targets and repairs a genetic defect, has been done in a living animal and achieved clinically meaningful results. Sun, 26 Jun 2011 14:53:53 EDT http://www.sciencedaily.com/releases/2011/06/110626145318.htm http://www.sciencedaily.com/releases/2011/06/110623174140.htm Researchers have identified a natural mechanism that might one day be used to block the expression of the mutated gene known to cause Huntington's disease. Their experiments offer not an immediate cure, but a potential new approach to stopping or even preventing the development of this relentless neurodegenerative disorder. Thu, 23 Jun 2011 17:41:41 EDT http://www.sciencedaily.com/releases/2011/06/110623174140.htm http://www.sciencedaily.com/releases/2011/06/110617164128.htm A compound that for about 60 years has been used as a drug against tapeworm infection is also apparently effective against colon cancer metastasis, as studies using mice have shown. The compound silences a gene that triggers the formation of metastases in colon cancer. Researchers in Germany made this discovery in collaboration with researchers in the U.S. Plans are already underway to conduct a clinical trial. Fri, 17 Jun 2011 16:41:41 EDT http://www.sciencedaily.com/releases/2011/06/110617164128.htm http://www.sciencedaily.com/releases/2011/06/110606092738.htm An experimental cure for type 1 diabetes has a nearly 80 percent success rate in curing diabetic mice. The results offer possible hope of curing a disease that affects 3 million Americans. Mon, 06 Jun 2011 09:27:27 EDT http://www.sciencedaily.com/releases/2011/06/110606092738.htm http://www.sciencedaily.com/releases/2011/06/110601152059.htm Intensity modulated radiation therapy, a newer, more precise form of radiation therapy, causes fewer gastrointestinal side effects when combined with hormone therapy than using three-dimensional radiation therapy, according to a new study. Wed, 01 Jun 2011 15:20:20 EDT http://www.sciencedaily.com/releases/2011/06/110601152059.htm http://www.sciencedaily.com/releases/2011/05/110531155349.htm New research proves that a change in a particular gene can identify which patients with a specific kind of brain cancer will respond better to treatment. Testing for the gene can distinguish patients with a more- or less-aggressive form of glioblastoma, the most common and an often-fatal type of primary brain cancer, and this can help guide therapy. The prospective study looked for a change in the MGMT gene in tumors from 833 glioblastoma patients. Tue, 31 May 2011 15:53:53 EDT http://www.sciencedaily.com/releases/2011/05/110531155349.htm http://www.sciencedaily.com/releases/2011/05/110523124402.htm New research aimed to better identify the genetic factors that lead to breast cancer has uncovered a link between the obesity gene and a higher incidence of breast cancer. Mon, 23 May 2011 12:44:44 EDT http://www.sciencedaily.com/releases/2011/05/110523124402.htm http://www.sciencedaily.com/releases/2011/05/110512104216.htm A person's vulnerability to nicotine addiction appears to have a genetic basis, at least in part. A region in the midbrain called the habenula (from Latin: small reins) plays a key role in this process. Researchers have also shed light on the mechanism that underlies addiction to nicotine. Thu, 12 May 2011 10:42:42 EDT http://www.sciencedaily.com/releases/2011/05/110512104216.htm http://www.sciencedaily.com/releases/2011/05/110509122723.htm Men with prostate cancer whose disease has spread locally are primarily treated with radiation therapy. However, disease recurs in approximately half of these individuals. Strategies to enhance the efficacy of this treatment are clearly needed. Researchers have now developed an approach that enhances the therapeutic effects of radiation therapy in mice bearing human prostate cancer xenografts, which they hope can be developed for clinical use. Mon, 09 May 2011 12:27:27 EDT http://www.sciencedaily.com/releases/2011/05/110509122723.htm http://www.sciencedaily.com/releases/2011/04/110428112549.htm Researchers have developed an effective technique that uses gene therapy on stem cells to correct chronic granulomatous disease in cell culture, which could eventually serve as a treatment for this rare, inherited immune disorder, according to a new study. Thu, 28 Apr 2011 11:25:25 EDT http://www.sciencedaily.com/releases/2011/04/110428112549.htm http://www.sciencedaily.com/releases/2011/04/110414104219.htm Switching off an essential gene to study its function is problematic because shutting off its activity permanently will kill the organism before the gene's function can be determined. Researchers have overcome this problem by using RNAi technology to temporarily turn off any essential gene in adult mice and then turn it back on before the change kills the animals. Thu, 14 Apr 2011 10:42:42 EDT http://www.sciencedaily.com/releases/2011/04/110414104219.htm http://www.sciencedaily.com/releases/2011/04/110403090248.htm Scientists have identified a mutation in the DDR2 gene that may indicate which patients with squamous cell lung cancer will respond to dasatinib. Sun, 03 Apr 2011 09:02:02 EDT http://www.sciencedaily.com/releases/2011/04/110403090248.htm http://www.sciencedaily.com/releases/2011/03/110315103537.htm Scientists have shown in mice that a defect in a single gene, which is involved in cellular signaling, is sufficient to cause a dangerous brain tumor. Tue, 15 Mar 2011 10:35:35 EDT http://www.sciencedaily.com/releases/2011/03/110315103537.htm http://www.sciencedaily.com/releases/2011/03/110308173422.htm ADAM-12 is not only the name of a 1970s television police drama -- it's also the gene that researchers believe could be an important element in the fight against cancer, arthritis, and cardiac hypertrophy, or thickening of the heart's walls. Tue, 08 Mar 2011 17:34:34 EST http://www.sciencedaily.com/releases/2011/03/110308173422.htm http://www.sciencedaily.com/releases/2011/03/110303120852.htm Using recent advances in genomics, researchers have uncovered a genetic pathway that affects the development of breast cancer, work that could help predict which patients are at risk of relapse for the disease. Thu, 03 Mar 2011 12:08:08 EST http://www.sciencedaily.com/releases/2011/03/110303120852.htm http://www.sciencedaily.com/releases/2011/02/110227211021.htm Scientists have shown that they can deliver a gene directly into breast cancer cells causing them to self-destruct, using an innovative, miniscule gene transport system, according to new research. Sun, 27 Feb 2011 21:10:10 EST http://www.sciencedaily.com/releases/2011/02/110227211021.htm http://www.sciencedaily.com/releases/2011/02/110207124037.htm Gene therapy holds great promise for the cure of many diseases but synthetic DNA sequences which are introduced directly into the genome bear a significant risk of cancer. Researchers have now developed a new method which makes use of modified RNA and avoids both the risk of cancer and immune reactions provoked by conventional RNAs. Mon, 07 Feb 2011 12:40:40 EST http://www.sciencedaily.com/releases/2011/02/110207124037.htm http://www.sciencedaily.com/releases/2011/01/110126121059.htm An innovative genetic strategy for rendering T-cells resistant to HIV infection without affecting their normal growth and activity is described in a new research paper. Wed, 26 Jan 2011 12:10:10 EST http://www.sciencedaily.com/releases/2011/01/110126121059.htm http://www.sciencedaily.com/releases/2011/01/110125160854.htm Give caffeine to cells engineered to produce viruses used for gene therapy and the cells can generate three- to eight-times more virus, according to a new paper. Tue, 25 Jan 2011 16:08:08 EST http://www.sciencedaily.com/releases/2011/01/110125160854.htm http://www.sciencedaily.com/releases/2011/01/110110121701.htm Researchers report a gene therapy strategy that improves the condition of a mouse model of an inherited blood disorder, beta-thalassemia. Some of the stem cell lines do not inherit the disease gene and can thus be used for transplantation-based treatments of the same mice. The findings could hold promise for a new treatment strategy for autosomal dominant diseases like certain forms of beta-thalassemia, tuberous sclerosis or Huntington's disease. Mon, 10 Jan 2011 12:17:17 EST http://www.sciencedaily.com/releases/2011/01/110110121701.htm http://www.sciencedaily.com/releases/2011/01/110105121129.htm A new, simplified method for producing large amounts of viral vector cassettes capable of shuttling genes into host cells will help advance the promising field of gene therapy as applications move into large animal studies and human clinical trials. The novel adeno-associated virus (AAV) production method is described in a new article. Wed, 05 Jan 2011 12:11:11 EST http://www.sciencedaily.com/releases/2011/01/110105121129.htm http://www.sciencedaily.com/releases/2011/01/110104064605.htm Scientists have reported encouraging results in a new gene-based therapy for Duchenne muscular dystrophy (DMD), which at present has no known cure and affects one in 3,000 young boys. Tue, 04 Jan 2011 06:46:46 EST http://www.sciencedaily.com/releases/2011/01/110104064605.htm