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Multi-armed cationic cyclodextrin:poly(ethylene glycol) polyrotaxanes as efficient gene silencing vectors.


ABSTRACT: A family of branched polyrotaxanes (bPRTx(+)), threaded with multiple cationic ?-cyclodextrins (?-CDs) onto a multi-armed poly(ethylene glycol) (PEG) core, were synthesized and studied as gene silencing vectors. These bPRTx(+) formed stable, positively charged complexes with diameters of 150-250 nm at N/P ratios as low as 2.5. The bPRTx(+) materials were shown to have gene-silencing efficiencies comparable to those of Lipofectamine 2000 (L2k) and bPEI, while displaying similar toxicity profiles. The unique structure of these polyrotaxanes allows them to effectively condense and complex siRNA into nanoparticles at much lower N/P ratios than L2k or bPEI. These findings suggest that bPRTx(+) may be useful materials for gene therapy applications.

SUBMITTER: Kulkarni A 

PROVIDER: S-EPMC3524380 | biostudies-other | 2013 Jan

REPOSITORIES: biostudies-other

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Multi-armed cationic cyclodextrin:poly(ethylene glycol) polyrotaxanes as efficient gene silencing vectors.

Kulkarni Aditya A   DeFrees Kyle K   Schuldt Ryan A RA   Vlahu Alexander A   VerHeul Ross R   Hyun Seok-Hee SH   Deng Wei W   Thompson David H DH  

Integrative biology : quantitative biosciences from nano to macro 20130101 1


A family of branched polyrotaxanes (bPRTx(+)), threaded with multiple cationic α-cyclodextrins (α-CDs) onto a multi-armed poly(ethylene glycol) (PEG) core, were synthesized and studied as gene silencing vectors. These bPRTx(+) formed stable, positively charged complexes with diameters of 150-250 nm at N/P ratios as low as 2.5. The bPRTx(+) materials were shown to have gene-silencing efficiencies comparable to those of Lipofectamine 2000 (L2k) and bPEI, while displaying similar toxicity profiles.  ...[more]

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