Math1 gene transfer based on the delivery system of quaternized chitosan/Na-carboxymethyl-beta-cyclodextrin nanoparticles.
Mammalian cochlear hair cells don't regenerate naturally after injury, which usually leave permanent hearing loss. Math1 gene is a positive regulator of hair cell differentiation during cochlear development and was proved to be very critical in hair cell regeneration in deaf animals. Generating new cochlear hair cells by forced Math1 expression may be a cure for hearing loss. However, satisfying gene delivering vectors in gene therapy are not available. We combined quaternized chitosan (QCS) with Na-carboxymethyl-beta-cyclodextrin (CM-beta-CD) as novel non-viral vector, which adsorbs pRK5-Math1-EGFP perfectly at the mass ratio of 4:1. In vitro cell transfection can reach a 40% transfect efficiency and relatively low cytotoxity than liposomes. These results suggest that QCS/CM-beta-CD nanoparticle complexes could be a novel non-viral gene carrier in further clinical application.
Department of Otolaryngology Head and Neck Surgery, Institute of Otolaryngology, Chinese PLA General Hospital, Beijing 100853, China.
Li-Li
LL
Ren
Yan
Y
Wu
Dong
D
Han
Li-Dong
LD
Zhao
Quan-Mei
QM
Sun
Wei-Wei
WW
Guo
Jian-He
JH
Sun
Nan
N
Wu
Xing-Qi
XQ
Li
Suo-Qiang
SQ
Zhai
Dong-Yi
DY
Han
Wie-Yen
WY
Young
Shi-Ming
SM
Yang
D051792:Basic Helix-Loop-Helix Proteins / Q000235:genetics*; D048271:Chitosan / Q000737:chemistry*; D018014:Gene Transfer Techniques; D053758:Nanoparticles; D020133:Reverse Transcriptase Polymerase Chain Reaction; D013213:Starch / Q000031:analogs & derivatives* / Q000737:chemistry; D047392:beta-Cyclodextrins / Q000737:chemistry*
D051792:Basic Helix-Loop-Helix Proteins; D048271:Chitosan; D013213:Starch; D047392:beta-Cyclodextrins; C500943:Atoh1 protein, mouse; C034848:carboxymethyl starch; C031215:betadex