Novel therapeutic strategy for single gene disorders delivers RNA that encodes the missing protein

(Mary Ann Liebert, Inc./Genetic Engineering News) Researchers have demonstrated the feasibility of delivering an RNA that encodes for the protein alpha-1-antitrypsin -- which is missing or nonfunctional in the genetic disorder AAT deficiency -- into cells in the laboratory, enabling the cells to produce highly functional AAT. This innovative approach to treating single gene disorders such as AAT deficiency offers and safe, simpler, and more cost-effective alternative to gene therapy or protein replacement, according to the authors of the study published in Nucleic Acid Therapeutics.

http://www.eurekalert.org/pub_releases/2015-07/mali-nts072715.php

Source: EurekAlert! - Medicine and Health - July 27, 2015 Category: Global & Universal Source Type: news