The current ASO trial in humans is a huntingtin lowering, or
gene silencing therapy, which works to disable both copies of the HD gene in short bursts.
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 this episode, Dr. Carlos Faraco speaks with Dr. Don Cleveland of the University of California San Diego regarding his work on drug - based
gene silencing therapies.
Not exact matches
Sutherland says the
genes she studies are already
silenced in healthy adult tissue, so theoretically researchers should be able to design
therapies that re-silence them in cancer cells without side effects.
Study shows memories formed by the same
gene -
silencing tool used in embryonic development; a finding could set the stage for new
therapies for schizophrenia
«Experimental
therapy for Prader - Willi syndrome shows promise in mice: Strategy activates
silenced genes.»
Toni Bedalov investigates drug candidates for anticancer
therapy in breast - cancer malignancies, illuminates the molecular process of
gene silencing
«With the new stem cell model, however, we hope eventually to put together a much more detailed picture of this process of repeat expansion and
gene silencing, and that should put us in a good position to devise optimal
therapies,» Gottesfeld said.
«With the new stem cell model, we hope eventually to put together a much more detailed picture of this process of repeat expansion and
gene silencing, and that should put us in a good position to devise optimal
therapies,» says Professor Joel Gottesfeld.
RNAi is a type of
gene therapy that «
silences»
genes by disrupting the protein - making process.
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.
Like many scientists, we think that
gene silencing is the most promising approach to developing meaningful
therapies for HD families, and it's gratifying that large companies are willing to make a large financial investment in the
therapy.
Van Meir cautions: «We need to have a better understanding of all the
genes that are turned on or off by
silencing MBD2 in a given cancer before we can envision to use this approach for
therapy.»
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.