New methods for quantifying antisense drug delivery to target cells and tissues

February 1, 2013
©2013, Mary Ann Liebert, Inc., publishers

Powerful antisense drugs that target disease-associated genes to block their expression can be used to treat a broad range of diseases. Though antisense therapy has been proven effective, challenges remain in ensuring that the drugs reach their intended targets. Two new methods for detecting and measuring the levels of antisense drugs in cells that could accelerate the development of improved antisense drugs are described in an article in BioResearch Open Access.

In the article "Development of Novel Bioanalytical Methods to Determine the Effective Concentrations of Phosphorodiamidate Morpholino Oligomers in Tissues and Cells," Frederick Schnell, Stacy Crumley, Dan Mourich, and P.L. Iversen, from Sarepta Therapeutics and Oregon State University (Corvallis, OR), describe two fast and sensitive methods for detecting a promising type of antisense therapeutic called a phosphorodiamidate morpholine oligomer, or PMO. Using these novel methods they were able to detect PMO delivery to individual cells and quantify how much PMO resides in a particular tissue in the body, such as the . For example, the authors describe the measurement of intranasally delivered PMO in lung tissue and, more specifically, in different cell types in the lung. They were able to measure the clearance kinetics of the PMO and determine whether it stayed in the .

"The development of novel, rapid PMO detection techniques such as these will advance the field of PMO research in a significant way, providing valid alternatives to the current time-consuming and labor-intensive methods," says Editor-in-Chief Jane Taylor, PhD, MRC Centre for , University of Edinburgh, Scotland.

Explore further: Antisense approach promising for treatment of parasitic infections

More information: www.liebertpub.com/biores

Related Stories

Antisense oligonucleotides make sense in myotonic dystrophy

February 27, 2012

Antisense oligonucleotides – short segments of genetic material designed to target specific areas of a gene or chromosome – that activated an enzyme to "chew up" toxic RNA (ribonucleic acid) could point the way ...

US researchers identify first human lung stem cell

May 11, 2011

For the first time, researchers at Brigham and Women's Hospital (BWH) have identified a human lung stem cell that is self-renewing and capable of forming and integrating multiple biological structures of the lung including ...

Recommended for you

Artificial beta cells

December 8, 2016

Researchers led by ETH Professor Martin Fussenegger at the Department of Biosystems Science and Engineering (D-BSSE) in Basel have produced artificial beta cells using a straightforward engineering approach.

Key regulator of bone development identified

December 8, 2016

Loss of a key protein leads to defects in skeletal development including reduced bone density and a shortening of the fingers and toes—a condition known as brachydactyly. The discovery was made by researchers at Penn State ...

Researchers question lifelong immunity to toxoplasmosis

December 8, 2016

Medical students are taught that once infected with Toxoplasma gondii—the "cat parasite"—then you're protected from reinfection for the rest of your life. This dogma should be questioned, argue researchers in an Opinion ...

TET proteins drive early neurogenesis

December 7, 2016

The fate of stem cells is determined by series of choices that sequentially narrow their available options until stem cells' offspring have found their station and purpose in the body. Their decisions are guided in part by ...

0 comments

Please sign in to add a comment. Registration is free, and takes less than a minute. Read more

Click here to reset your password.
Sign in to get notified via email when new comments are made.