A novel mixing device for the reproducible generation of nonviral gene therapy formulations

BioTechniques

卷:49

Graciela A. Nunez-Alonso¹  Lee A. Davies¹  Deborah R. Gill¹  Stephen C. Hyde¹  Henry L. Hebel²  Ron K. Scheule³  Seng H. Cheng³ 

[1] 1Gene Medicine Group, Nuffield Department of Clinical Laboratory Sciences, University of Oxford, John Radcliffe Hospital, Oxford, UK;

[2] 2VGXI, Houston, TX, USA;

[3] 3Genzyme Corporation, Framingham, MA, USA;

关键词: Gene therapy;    nonviral formulations;    lipoplexes;    polyplexes;   

DOI  :  10.2144/000113498

来源: DOAJ

【 摘 要 】

Nonviral gene therapy utilizing plasmid DNA (pDNA) complexed with cationic lipids (lipoplexes) or cationic polymers (polyplexes) has demonstrated considerable potential for the treatment of a variety of diseases. However, progress toward clinical application is often delayed by the lack of reliable and scalable mixing of components sufficient to guarantee consistent performance in vivo. Attempts to improve and standardize mixing have been limited by the sensitivity of pDNA to shear-related degradation. Here we describe a simple pneumatic mixing device that enables the rapid and reproducible production of large volumes of nonviral gene therapy formulations and demonstrate its suitability for use with shearsensitive pDNA.

【 授权许可】

Unknown