Despite Challenges RNAi-based Therapies Have Made Huge Progress, says New Report
News Nov 13, 2009
Piribo, the online destination for business intelligence for the biotech and pharmaceutical industry, has added a new report analyzing partnering agreements in diagnostics.
“Delivering RNAi-Based Therapeutics & Diagnostics: Challenges & Opportunities”, which is available at bit.ly/4hSRfd, reports that over the last decade, tremendous advances have been made in the field of RNAinterference (RNAi), a naturally occurring mechanism for gene regulation. Researchers have begun to unravel the underlying mechanisms of gene silencing and along the way a number of new gene silencing agents have been uncovered such as microRNAs.
The 225 page report assesses the commercial potential of RNAi-based therapies in all of the key therapeutic areas: cancer, CNS disorders, viral infectious diseases, ocular diseases, cardiovascular, metabolic and other conditions.
Furthermore it shows that several novel RNAi-based therapeutics are expected to be launched in the next ten years as companies have made significant advances in identifying, characterizing, designing and delivering gene silencing agents to a variety of target tissues.
Whilst RNAi has become a useful tool for understanding the function of specific genes and a means of identifying new targets for small molecule intervention, many scientists have harnessed its power to develop RNAi-based therapeutics, which can treat and in some cases prevent disease and have expanded the repertoire of targets previously deemed 'undruggable'.
In addition, a second generation of RNAi therapeutics, dicer substrate RNA (disRNA), have been discovered that act higher up the RNAi pathway than siRNAs and use endogenous enzymes to process them into siRNAs. Interestingly, this new class of RNA-based therapeutics appear to be more potent and have a longer duration of action than their siRNA counterparts.
However, this field is still in its infancy and the full potential of RNA-based therapeutics has yet to be realized. To date, no RNAi-based therapeutic has been approved, although several products are in clinical development.
In a new study in cells, University of Illinois researchers have adapted CRISPR gene-editing technology to cause the cell’s internal machinery to skip over a small portion of a gene when transcribing it into a template for protein building. This gives researchers a way not only to eliminate a mutated gene sequence, but to influence how the gene is expressed and regulated.
Researchers published today a detailed description of the complete genome of bread wheat, the world's most widely-cultivated crop. This work will pave the way for the production of wheat varieties better adapted to climate challenges, with higher yields, enhanced nutritional quality and improved sustainability.