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Rapid Optimization of Gene Delivery by Parallel End-modification of Poly(β-amino ester)s.
| Content Provider | Semantic Scholar |
|---|---|
| Author | Zugates, Gregory T. Peng, Weidan Zumbuehl, Andreas Jhunjhunwala, Siddharth Huang, Yu-Hung Langer, Robert S. Sawicki, Janet A. Anderson, Daniel G. |
| Copyright Year | 2007 |
| Abstract | Poly(β-amino ester)s are cationic degradable polymers that have significant potential as gene delivery vectors. Here we present a generalized method to modify poly(β-amino ester)s at the chain ends to improve their delivery performance. End-chain coupling reactions were developed so that polymers could be synthesized and tested in a high-throughput manner, without the need for purification. In this way, many structural variations at the polymer terminus could be rapidly evaluated. End-modification of the terminal amine structure of a previously optimized poly(β-amino ester), C32, significantly enhanced its in vitro transfection efficiency. In vivo, intraperitoneal (IP) gene delivery using end-modified C32 polymers resulted in expression levels over one order of magnitude higher than unmodified C32 and jet-polyethylenimine (jet-PEI) levels in several abdominal organs. The rapid end-modification strategy presented here has led to the discovery of many effective polymers for gene delivery and may be a useful method to develop and optimize cationic polymers for gene therapy. |
| Starting Page | 1306 |
| Ending Page | 1312 |
| Page Count | 7 |
| File Format | PDF HTM / HTML |
| DOI | 10.1038/sj.mt.6300132 |
| Alternate Webpage(s) | http://www.chem.unifr.ch/zumbuehl/assets/files/2007-MolTher.pdf |
| PubMed reference number | 28182918 |
| Alternate Webpage(s) | https://doi.org/10.1038/sj.mt.6300132 |
| Journal | Medline |
| Volume Number | 15 |
| Issue Number | 7 |
| Journal | Molecular therapy : the journal of the American Society of Gene Therapy |
| Language | English |
| Access Restriction | Open |
| Content Type | Text |
| Resource Type | Article |
