In the Baulcombe lab, Voinnet analyzed the in vivo
dynamics of gene silencing using transgenic plants, recombinant viruses, and fluorescence microscopy.
At almost the same time, he stumbled across what would later become his research field: the first
observations of gene silencing, which had started to trickle through via transgenic plant experiments.
2015 will see the start of the first human clinical
trial of a gene silencing or huntingtin - lowering drug, which specifically aims to reduce production of mutant huntingtin in the brains of HD patients.
It is, in a sense, the
opposite of gene silencing, but it's another proof point that validates the general concept of creating drugs that mute or amplify defective genes.
This
form of gene silencing is triggered by a short piece of double - stranded RNA (the chemical cousin to DNA) that matches the gene.
Toni Bedalov investigates drug candidates for anticancer therapy in breast - cancer malignancies, illuminates the molecular process of gene silencing
Ullu was recognized for her lab's work with the protozoan parasite Trypanosoma brucei, which causes African sleeping sickness, to uncover a novel
mechanism of gene silencing known as RNA interference.
The length of this «triplet repeat expansion» determines the
degree of gene silencing, and thus the severity of the disease.
Now, a major deal between Isis Pharmaceuticals and drug giant Roche promises to support the development of one
kind of gene silencing drug, called ASOs, taking them through to clinical trials in patients as quickly and efficiently as possible.
Since IGF2R is imprinted (i.e. one
copy of the gene silenced by DNA methylation), this finding thrusted me into the rapidly growing research field of epigenetics.