Dr Carroll, who edited the article, has previously undertaken RNA - targeted Huntington's
disease gene silencing research, and is named in an associated patent.
Not exact matches
The
gene -
silencing strategy, in the form of RNA interference, has also showed promise against an uncommon neurodegenerative
disease called hereditary ATTR amyloidosis, as my colleague Sy Mukherjee wrote about in Fortune's year - end Investor's Guide.
«These particles, microRNA based SNAs, could also potentially be used for
gene silencing in other cancers and
diseases of genetic origin.»
RNA interference (RNAi), a technique for
silencing genes that shows potential for treating
diseases, has been like a hot baseball prospect who hasn't proven he can play in the big leagues.
Being able to detect and measure the amount of mutant huntingtin present in the nervous system will be a valuable way of seeing whether the
gene -
silencing drug is hitting its target and has the intended effect, lowering the amount of
disease causing mHTT protein.
Silencing of one of the «cell death»
genes may be the first step in finding treatments to help woman delay menopause or avoid the health problems — osteoporosis, heart
disease — associated with the cessation of estrogen production.
ASCB President Don Cleveland of the University of California, San Diego, said his team just published an animal study on a
gene silencing therapy for treating a form of Lou Gehrig's
disease that they now hope to move to clinical studies.
In September, Alnylam Pharmaceuticals announced that its small RNA molecules
silenced the
gene that causes the progressive
disease hereditary ATTR amyloidosis in a successful phase III trial.
Researchers have collected information on which
genes are activated or
silenced in certain
diseases and by certain drugs for many years.
Drugs capable of activating
silenced genes improve survival and growth outcomes in a mouse model of Prader - Willi syndrome (PWS), a rare and incurable childhood
disease, according to a study funded by the National Institutes of Health (NIH).
Biologically, hypermethylation often
silences genes that keep runaway cell growth in check, and its appearance in the DNA code of breast cancer - related
genes shed into the blood may indicate that a patient's cancer growth is increasing and the
disease has worsened.
Results with formulations of lipid nanoparticles as agents to deliver siRNAs in animals, including nonhuman primates, 28,29 have shown activity toward
silencing of
disease - modifying
genes expressed in the liver.
If what you've read here about zinc finger technology reminds you of «
gene silencing» or «huntingtin - lowering» methods for treating Huntington's
disease, give yourself a gold star.
Today brings news that the first Huntington's
Disease patients have been successfully dosed with
gene silencing drugs targeting the HD
gene.
Our proprietary technology can selectively inhibit any
gene in the genome, specifically
silencing the production of
disease - causing proteins.
The new CRISPR technique works by
silencing the
gene (DUX4) thought to be responsible for the genetic
disease.
Our technology is a highly specific and modular platform that enables us to inhibit or «
silence» the expression of
disease - causing
genes, allowing cells to revert to their healthy state.
After huge leaps forward in recent years, we're edging ever closer to human trials of huntingtin lowering or «
gene silencing» as a potential treatment for Huntington's
Disease.
The length of this «triplet repeat expansion» determines the degree of
gene silencing, and thus the severity of the
disease.
Cleveland and the members of his lab use these therapies, also known as designer DNA drugs, to
silence genes involved in the development of various neurodegenerative
diseases such as Alzheimer's, Huntington's and Lou Gehrig's
disease.
She leads a team at the UCL Huntington's
Disease Centre that is currently conducting a trial of a novel «
gene silencing» treatment for HD, sponsored by Ionis Pharmaceuticals.
We are interested in dissecting the mechanisms of epigenetic
gene silencing and understanding the functional relevance of DNA methylation in
diseases.
So far, we've seen
gene silencing drugs tested in several different animal models of Huntington's
disease, successfully delaying symptom onset or even reversing symptoms.
By injecting molecules called short interfering RNAs directly into tissue known as the trabecular meshwork, researchers have been able to
silence the action of
disease - relevant
genes.
Mipomersen (Kynamro) reduces low - density lipoprotein (LDL) cholesterol by
silencing a
gene that produces apolipoprotein B - 100, a building block of LDL; the drug can treat familial hypercholesterolemia, an inherited
disease that may cause heart attacks at an early age.
One possible implication of this work is for so - called «
gene -
silencing» therapies for Huntington's
disease, which aim to reduce production of the huntingtin protein, by sticking to its RNA message molecules and telling cells to get rid of them.
Transgenerational effects of caloric restriction on longevity, B - vitamins
silencing bad
genes, cognitive stimulation revering neurodegenerative
disease, exercise increases neurogenesis, and vitamin D controls aging.