July 28, 2014 – 8:42 am
Creating a successful vaccine against two members of the family, the sexually transmitted herpes simplex virus 1 (HSV-1) and 2 (HSV-2), has proven to be challenging. A clinical trial being conducted by a branch of the National Institutes of Health, now enrolling patients, is based on an HSV-2 vaccine developed by David Knipe, Higgins Professor of Microbiology and Molecular Genetics at Harvard Medical School.
Meanwhile, Knipe and his colleagues are continuing to fight these viruses on another front. Seizing an approach used against HIV, they are studying possible treatments that could also combat herpes virus infections.
July 25, 2014 – 10:09 am
Clinical stage biotech firm Celladon Corporation announced that it has signed an exclusive, global license from Enterprise Partners Venture Capital for the membrane-bound form of the Stem Cell Factor gene (mSCF) as treatment for cardiac ischemia. The license agreement will allow Celladon to use mSCF in gene therapy applications.
Stem Cell Factor is an important cytokine which plays a role in cell migration and survival as well as survival of cardiac stem cells. Scientists from the Cardiovascular Research Center at Icahn School of Medicine at Mount Sinai have successfully used mSCF gene therapy to undo heart damage in animal models after myocardial infarction. According to the study’s senior investigator Roger J. Hajjar, Director of the Cardiovascular Research Center at Icahn School of Medicine at Mount Sinai, “mSCF gene therapy promoted a regenerative response in damaged hearts similar to that observed with stem cell therapy and may be one of the first non-cell therapies to increase cardiac muscle precursors in the heart.” The findings were published in Circulation Research.
July 23, 2014 – 9:29 am
Researchers at the University of Iowa have developed a vaccine that can combat dust-mite allergies by naturally switching the body's immune response. In animal tests, the nano-sized vaccine package lowered lung inflammation by 83 percent despite repeated exposure to the allergens, according to the paper, published in the AAPS (American Association of Pharmaceutical Scientists) Journal. One big reason why it works, the researchers contend, is because the vaccine package contains a booster that alters the body's inflammatory response to dust-mite allergens.
July 22, 2014 – 10:09 am
For the first time, researchers in Philadelphia have found a way to completely delete the HIV virus from human cells by ‘snipping’ them out. The process could also provide a cure for other latent infections.
In a study published by the Proceedings of the National Academy of Sciences, Dr Khalili and colleagues detail how they created molecular tools to delete the HIV-1 proviral DNA. When deployed, a combination of a DNA-snipping enzyme called a nuclease and a targeting strand of RNA called a guide RNA (gRNA) hunt down the viral genome and remove the HIV-1 DNA.
From there, the cell's gene repair machinery takes over, soldering the loose ends of the genome back together – resulting in virus-free cells.
July 21, 2014 – 9:17 am
Aduro BioTech, Inc., a clinical-stage biotechnology company, today announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation for its pancreatic cancer combination treatment that consists of its CRS-207 and GVAX Pancreas immunotherapies. According to the FDA, a breakthrough therapy designation is for a drug that treats a serious or life-threatening condition, and preliminary clinical evidence indicates that the drug may demonstrate substantial improvement on a clinically significant endpoint over available therapies.
July 18, 2014 – 9:15 am
CRISPR, a system of genes that bacteria use to fend off viruses, is involved in promoting antibiotic resistance in Francisella novicida, a close relative of the bacterium that causes tularemia. The finding contrasts with previous observations in other bacteria that the CRISPR system hinders the spread of antibiotic resistance genes.
The results are scheduled for publication in PNAS Early Edition.
July 17, 2014 – 9:18 am
By injecting a gene into a pig’s heart, scientists have created a “biological pacemaker” that can regulate heartbeats, an achievement that eventually may lead to an alternative to electronic pacemakers in some people.
The technique may also prove to be a promising example of gene therapy, which so far has shown glimmers of success in just a few other conditions.
Researchers at the Cedars-Sinai Heart Institute in Los Angeles reported Wednesday that they had injected a gene into a tiny section of pigs’ hearts and were able to temporarily reprogram ordinary heart cells into rhythm-generating cells. Human trials of the technique are at least three years away, and if successful, the approach would be, at least at first, limited to a small subset of pacemaker users.
July 15, 2014 – 9:17 am
Researchers at the University of Sheffield have moved one step closer to a gene therapy that could silence the faulty SOD1 gene responsible for triggering a form of motor neuron disease (MND) also known as amyotrophic lateral sclerosis (ALS).
Scientists from the Sheffield Institute for Translational Neuroscience (SITraN) have begun groundbreaking research to develop a gene therapy that, if successful, could cure one form of the devastating neurological disease which affects more than 5,000 people in the UK.
July 11, 2014 – 11:17 am
A potential new dengue vaccine - the first to reach phase 3 clinical testing - has demonstrated that it protects 56% of subjects from the disease, according to research published in The Lancet. Currently, there are no licensed vaccines or specific treatments that prevent dengue infection. The main problem with developing a vaccine capable of preventing dengue is that the disease is caused by four separate dengue viruses, so the vaccine must be able to target all four serotypes.
July 10, 2014 – 9:33 am
Columbia University Medical Center (CUMC) researchers have created a way to develop personalized gene therapies for patients with retinitis pigmentosa (RP), a leading cause of vision loss. The approach, the first of its kind, takes advantage of induced pluripotent stem (iPS) cell technology to transform skin cells into retinal cells, which are then used as a patient-specific model for disease study and preclinical testing.
Using this approach, researchers led by Stephen H. Tsang, MD, PhD, showed that a form of RP caused by mutations to the gene MFRP (membrane frizzled-related protein) disrupts the protein that gives retinal cells their structural integrity. They also showed that the effects of these mutations can be reversed with gene therapy. The approach could potentially be used to create personalized therapies for other forms of RP, as well as other genetic diseases. The paper was published recently in the online edition of Molecular Therapy, the official journal of the American Society for Gene & Cell Therapy.
