NYU Researchers Developing Molecular Delivery Vehicles For Genetic Therapies

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NYU Researchers Developing Molecular Delivery Vehicles For Genetic

Therapies

http://www.medicalnewstoday.com/medicalnews.php?newsid=56609

Researchers at New York University are working to develop molecular

delivery vehicles that can be used to transport nucleic acids into

diverse cell types, which may lead to eventual applications in

genetic therapies. Their work is described as part of the cover

story in the Nov. 13 issue of the American Chemical Society

publication Chemical and Engineering News.

Scientists have been exploring RNA interference (RNAi) as a gene

therapy technique to silence genes that are improperly produced.

The " RNAi " approach requires the delivery into the cell of short

pieces of the genetic material Ribonucleic Acid (RNA). These

synthetic short RNA " oligos " can then pair with specific sites in

the cell's own RNA, targeting the genetic messages for destruction

and turning off expression of the corresponding genes. However, the

widespread clinical use of this genetic therapy relies upon

technical improvements, including new delivery vehicles such as the

one Kent Kirshenbaum, an assistant professor in NYU's Department of

Chemistry, and colleagues present in their work.

The NYU researchers use a modular linear molecule to deliver

therapeutic RNA into cells. The molecule has a positively charged

site that forms favorable stabilizing interactions with the

negatively charged RNA, and a fatty component that interacts with

cell membranes. The molecules and RNA form complexes, which protect

the RNA from being degraded and deliver it to cells. As a result,

the targeted deleterious genes are silenced.

Their research concentrates on making the transition from the lab

into real-life smoother. Their linear molecule can be used to

deliver small therapeutic RNAs into cell types that are much more

representative of cellular targets that investigators are likely to

encounter in clinical situations.

Kirshenbaum and his coworkers are now focused on understanding the

physical chemical characteristics that give enhanced activity to

their molecule, and then use the knowledge to generate a set of more

sophisticated delivery reagents for siRNA.

" Our goal is to develop a platform that would allow us to create a

library that could be used in different settings or for delivery to

different cell types, " he told Chemical and Engineering News.