Poly(beta-amino esters): procedures for synthesis and gene delivery.
Non-viral gene delivery systems are promising as they avoid many problems of viral gene therapy by having increased design flexibility, high safety, large DNA cargo capacity, and ease of manufacture. Here, we describe the use of polymeric vectors, in particular biodegradable poly(beta-amino esters) (PBAEs), for non-viral gene delivery. These polymers are able to self-assemble with DNA and form positively charged gene delivery nanoparticles. Methods for synthesis of these polymers, particle self-assembly, and transfection using these particles are delineated. A standard protocol is presented as well as a high-throughput screening technique that can be used to more quickly optimize transfection parameters for efficient delivery.
Green
Jordan J
JJ
Department of Biological Engineering, Department of Chemical Engineering, and Center for Cancer Research, Massachusetts Institute of Technology, Cambridge, MA 02139, USA.
Zugates
Gregory T
GT
Langer
Robert
R
Anderson
Daniel G
DG
D000818:Animals; D002478:Cells, Cultured; D018014:Gene Transfer Techniques; D006801:Humans; D011091:Polyesters / Q000138:chemical synthesis / Q000737:chemistry*
D011091:Polyesters