New gene-silencing platform that can lead to treatments for diseases resistant to current RNAi methods

London, February 16 (ANI): Rutgers University researchers in New Jersey, US, say that they have created a gene-silencing platform, which may enable the development and discovery of a new class of drugs to treat a wide array of diseases.

The researchers have revealed that critical to the technology is the approach they specifically took to target RNA biosynthesis.

Research leader Samuel Gunderson, an associate professor in the Department of Molecular Biology and Biochemistry at Rutgers, claims that his team have developed highly efficient gene silencing agents that function via a novel mechanism of action.

According to him, the agents are single-stranded oligonucleotides, called U1 Adaptors, which have dual and independent functions.

The researcher says that first is a target-gene binding domain that can be tailored to any gene, while the second domain inhibits mRNA maturation by binding U1 snRNP, a component of the cellular splicing apparatus.

Gunderson suggests that combining both capabilities in the same molecule can enable the U1 Adaptor to inhibit the pre-mRNA maturation step of polyA tail addition in a gene specific manner.

Further, the domains of the oligonucleotide are independent so transcript binding and U1 snRNP binding can be independently optimized and adapted to a wide array of genes associated with disease.

"The U1 Adaptor platform expands on early technologies that used 5'-end-mutated U1 snRNA. The U1 Adaptor is an oligonucleotide version of this older method and instead targets the 3' end processing step. U1 Adaptors have high activity when used alone and are synergistic when used in combination with RNAi," Nature magazine quoted Gunderson as saying.

The researcher further said that the range of possible targets was very broad due to the mechanism of action in which inhibition occurs during the biosynthesis of mRNA at the near universal 3' end processing step.

He even suggests that U1 Adaptors can possibly inhibit genes that do not respond to current RNAi methods.

A research paper on this project has been published in the journal Nature Biotechnology. (ANI)