新規治療法：mRNAネブライザー治療

hyperbranched poly(beta amino esters) (hPBAEs) を用い In vitro transcribed (IVT) mRNA をネブライザー投与

治療用蛋白産生メッセンジャーRNAをターゲット細胞へ運搬目的の吸入治療

Asha Kumari Patel, James C. Kaczmarek, Suman Bose, Kevin J. Kauffman, Faryal Mir, Michael W. Heartlein, Frank DeRosa, Robert Langer, Daniel G. Anderson. Inhaled Nanoformulated mRNA Polyplexes for Protein Production in Lung Epithelium.
Advanced Materials, 2019; 1805116 
DOI: 10.1002/adma.201805116


nebulized delivery of IVT‐mRNA facilitated by hPBAE vectors


非侵襲的な投与法で、核酸ベースの治療

のう胞性線維症 phase 1/2臨床トライアルの検証開始

他疾患への適応にも期待

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解説：


Engineers create an inhalable form of messenger RNA
Patients with lung disease could find relief by breathing in messenger RNA molecules
Date:　January 4, 2019
Source:
Massachusetts Institute of Technology
Summary:

In an advance that could lead to new treatments for lung disease, researchers have now designed an inhalable form of mRNA. This aerosol could be administered directly to the lungs to help treat diseases such as cystic fibrosis, the researchers say.

https://www.sciencedaily.com/releases/2019/01/190104104032.htm