Innovative approach to treating AAT deficiency

Researchers have demonstrated the feasibility of delivering an RNA that encodes for the protein alpha-1-antitrypsin (AAT)--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, a peer-reviewed journal from Mary Ann Liebert, Inc., publishers. The article is available free on the Nucleic Acid Therapeutics website until August 27, 2015.

In the article "In vitro Evaluation of a Novel mRNA-Based Therapeutic Strategy for the Treatment of Patients Suffering from Alpha-1-Antitrypsin Deficiency", Tatjana Michel, Stefanie Krajewski, and coauthors, University Medical Center, Tuebingen, Germany, produced a messenger RNA sequence that cells can translate to generate the AAT protein. The researchers assessed the stability and utility of the encapsulated RNA over time and evaluated the amount of AAT protein produced by the cells and how well the protein functioned. The data show no negative effects of the transfected RNA on the viability of the cells and no immune activation.

"The field is looking for advances in modified mRNA as a therapeutic strategy," says Executive Editor Graham C. Parker, PhD, The Carman and Ann Adams Department of Pediatrics, Wayne State University School of Medicine, Children's Hospital of Michigan, Detroit, MI. "Demonstrations such as this from the University Medical Center, Tuebingen, Germany, show real progress."

Comments

The opinions expressed here are the views of the writer and do not necessarily reflect the views and opinions of News Medical.
Post a new comment
Post

While we only use edited and approved content for Azthena answers, it may on occasions provide incorrect responses. Please confirm any data provided with the related suppliers or authors. We do not provide medical advice, if you search for medical information you must always consult a medical professional before acting on any information provided.

Your questions, but not your email details will be shared with OpenAI and retained for 30 days in accordance with their privacy principles.

Please do not ask questions that use sensitive or confidential information.

Read the full Terms & Conditions.

You might also like...
Brazilian vegans meet protein needs but rely on ultra-processed products, study shows