Latest Applied genetics Stories
Researchers believe they have found a way to avoid certain genetically inherited disorders through a special kind of in vitro fertilization (IVF). Ethical concerns abound, but experts say that the future of the procedure looks promising.In lab experiments with rhesus macaque monkeys, scientists from the Oregon Health and Science University were recently able to transfer DNA from the nucleus of an unhealthy egg cell into a normal egg cell without a nucleus, ultimately resulting in the...
 One year after a trio of young adults received gene therapy for an inherited form of blindness, researchers have documented that the patients are still experiencing the same level of remarkable vision improvements previously measured within weeks. This is the first study to report one-year gene therapy safety and efficacy results in treating young adults with Leber Congenital Amaurosis (LCA), a hereditary condition that causes severe vision impairment in infants and children. The findings...
Scientists have worked for 20 years to perfect gene therapy for the treatment of cystic fibrosis, which causes the body to produce dehydrated, thicker-than-normal mucus that clogs the lungs and leads to life threatening infections.Now University of North Carolina at Chapel Hill School of Medicine scientists have found what may be the most efficient way to deliver a corrected gene to lung cells collected from cystic fibrosis patients. They also showed that it may take this high level of...
In 1989 scientists identified the gene mutation that causes cystic fibrosis (CF), which led to the hope that CF lung disease could be 'cured' using gene therapy. The premise of gene therapy is that modified viruses or other gene-based systems could be used to deliver a corrected version of a gene into affected tissues. However, the projected cure has been hampered by the natural ability of the lung to limit the introduction of foreign genes into its cells. Now, University of North Carolina at...
Pancreatic cancer is a devastating disease with poor prognosis. This warrants the development of novel therapies including gene therapy. However, clinical studies have demonstrated poor efficacy of adenoviral gene therapy because of the absence of adenoviral binding sites on pancreatic cancer cells such as the coxsackie and adenovirus receptor (CAR). Circumventing CAR-mediated entry therefore seems a promising option to improve adenoviral entry into pancreatic cancer cells and to enhance the...
Strategy could have implications for multiple tumor typesResearchers at Nationwide Children's Hospital and Johns Hopkins University have discovered that delivering a small molecule that is highly expressed in normal tissues but lost in diseased cells can result in tumor suppression.MicroRNAs (miRNA) are a class of small RNA molecules that are highly expressed in normal tissues and are critical in gene expression and in maintaining normal cell development and cell balance. Dysfunction of...
A new study suggests that delivering small RNAs, known as microRNAs, to cancer cells could help to stop the disease in its tracks. microRNAs control gene expression and are commonly lost in cancerous tumors. Researchers have shown that replacement of a single microRNA in mice with an extremely aggressive form of liver cancer can be enough to halt their disease, according to a report in the June 12 issue of the journal Cell, a Cell Press publication. They delivered the microRNA to the mice...
South Korean medical scientists say a new lung cancer therapy employing a vaporized viral vector has shown early promise in a mouse model of lung cancer. The researchers at South Korea's Ministry of Education, Science and Technology said the vaporized viral vector is used to deliver a cancer-inhibiting molecule directly to lung tissue. Although gene therapy is an area of great promise, the scientists said delivery mechanisms have proven problematic for effective delivery of genetic therapy...
Phase 1 Data Demonstrate Safety and Evidence of Biological Activity in 9 of 12 Patients; Phase 2 Study Currently Enrolling in 17 Leading U.S. Medical Centers SAN DIEGO, May 28 /PRNewswire/ -- Celladon Corporation presented today Phase 1 data from the Calcium Up-Regulation by Percutaneous Administration of Gene Therapy in Cardiac Disease (CUPID), a First-in-Human Phase 1/2 Clinical Trial, in a scientific symposium at the 12th Annual American Society of Gene Therapy Meeting. The Phase 1...
AMSTERDAM, May 7 /PRNewswire-FirstCall/ -- Amsterdam Molecular Therapeutics (Euronext: AMT), a leader in the field of human gene therapy, announced today the treatment of the first patient in a preregistration clinical trial with Glybera(TM). This gene therapy product targets lipoprotein lipase deficiency (LPLD), a seriously debilitating and potentially lethal disease. The randomized controlled trial has been designed to gather additional data on the effects of Glybera on lipid...
