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In vivo gene transfer using cetylated polyethylenimine

100%

Sochanik A., Cichon T., Makselon M., Strozyk M., Smolarczyk R., Jazowiecka-Rakus J., Szala S.

Acta Biochimica Polonica

2004

tom 51

nr 3

This report describes gene transfer in vitro as well as in vivo using cetylated low-molecular mass (600 Da) polyethylenimine (28% of amine groups substituted with cetyl moieties), termed CT-PEI. This compound is hydrophobic and has to be incorporated into liposomes in order to be suitable for gene transfer studies. Serum-induced plasmid DNA degradation assay demonstrated that CT-PEI-containing liposomal carriers could protect complexed DNA (probably via condensation). In vitro lu- ciferase gene expression achieved using medium supplemented with 10% serum was comparable to that achieved in serum-reduced medium and was highest for CT-PEI/cholesterol liposomes, followed by CT-PEI/dioleoylphosphatidylcholine liposomes and PEI 600 Da (uncetylated) carrier. In vivo systemic transfer into mice was most efficient when liposome formulations contained CT-PEI and cholesterol. Higher luciferase expression was then observed in lungs than in liver. In conclusion: liposomes containing cetylated polyethylenimine and cholesterol are a suitable vehicle for investigating systemic plasmid DNA transfer into lungs.

Assessment of viral and non-viral gene transfer into adult rat brains using HSV-1, calcium phosphate and PEI-based methods

80%

Corso T. D., Torres G., Goulah C., Roy I., Gambino A. S., Nayda J., Buckley T., Stachowiak E. K., Bergey E. J., Pudavar H., Dutta P., Bloom D. C., Bowers W. J., Stachowiak M. K.

Folia Morphologica

2005

tom 64

nr 3

130-144

CNS gene transfer could provide new approaches to the modelling of neurodegenerative diseases and devising potential therapies. One such disorder is Parkinson’s disease (PD), in which dysfunction of several different metabolic processes has been implicated. Here we review the literature on gene transfer systems based on herpes simplex virus type 1 (HSV-1) and non-viral polyethyleneimine (PEI) and calcium phosphate nanoparticle methods. We also assess the usefulness of various CNS gene delivery methods and present some of our own data to exemplify such usefulness. Our data result from vectors stereotaxically introduced to the substantia nigra (SN) of adult rats and evaluated 1 week and/or 1 month post injection using histochemical methods to assess recombinant β-galactosidase enzyme activity. Gene transfer using PEI or calcium phosphate-mediated transfections was observed for both methods and PEI was comparable to that of HSV-1 amplicon. Our data show that the amplicon delivery was markedly increased when packaged with a helper virus and was similar to the expression profile achieved with a full-size replication-defective HSV-1 recombinant (8117/43). We also examine whether PEI or HSV-1 amplicon-mediated gene transfer could facilitate assessment of the biological effects induced by a dominant negative FGF receptor-1 mutant to model the reduced FGF signalling thought to occur in Parkinson’s disease.

Ograniczanie wyników

1 Acta Biochimica Polonica

1 Folia Morphologica

1 Bergey E. J.

1 Bloom D. C.

1 Bowers W. J.

1 Buckley T.

1 Cichon T.

1 Corso T. D.

1 Dutta P.

1 Gambino A. S.

1 Goulah C.

1 Jazowiecka-Rakus J.

1 Makselon M.

1 Nayda J.

1 Pudavar H.

1 Roy I.

1 Smolarczyk R.

1 Sochanik A.

1 Stachowiak E. K.

1 Stachowiak M. K.

1 Strozyk M.

1 Szala S.

1 Torres G.

1 2005

1 2004

Wyniki wyszukiwania

In vivo gene transfer using cetylated polyethylenimine

Assessment of viral and non-viral gene transfer into adult rat brains using HSV-1, calcium phosphate and PEI-based methods