Wyniki wyszukiwania

1

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.

2

Synthetic amphiphiles as non-viral vectors in gene therapy. Development and mechanisms

80%

Hoekstra D., Oberle V., Audouy S., Zuhorn I., Engberts J.

Cellular and Molecular Biology Letters

|

1999

|

tom 04

|

nr 2

3

The intracellular fate of non-viral DNA carriers

80%

Langner M.

Cellular and Molecular Biology Letters

|

2000

|

tom 05

|

nr 3

Rapid progress in molecular biology and genetic engineering techniques has made the induction of cell behavior modifications by altering genetic material possible. This allows us to think about the medical application of gene therapy as an alternative to classical pharmacology and as a means of introducing a new approach to treating hereditary diseases. The major obstacle that prevents the application of this method in clinical practice is the difficulty of delivering genetic material to its destination. The fragile DNA molecule (very unstable in in vivo) requires the assistance of supramolecular structures in order to ensure its extended lifetime in circulation. A number of DNA delivery methods have been considered. One of them, a viral carrier, has been extensively studied as a potential candidate for this purpose. Unfortunately, a number of serious problems exist associated with this approach. The risk of accidental infection has not been eliminated and virus presentation causes the immune system to activate. Transfection efficiency achieved with viral vectors is superior to that of other methods, but safety concerns and financial considerations have stimulated studies aimed at the development of non-viral DNA carriers. At present, the transfection efficiency of such carriers is very low. This paper discusses problems encountered when developing a stable and efficient lipid-DNA aggregate, capable of delivering genetic material into the targeted cell nucleus. Discussion is limited to aggregate interaction with the cellular plasma membrane and its fate in the cytosol.

4

Use of HIV as a gene transfer vector

61%

Pluta K., Kacprzak M. M.

Acta Biochimica Polonica

|

2009

|

tom 56

|

nr 4

531-595

Despite the extensive research efforts over the past 25 years that have focused on HIV, there is still no cure for AIDS. However, tremendous progress in the understanding of the structure and biology of the HIV virus led to the development of safe and potent HIV-based transgene delivery vectors. These genetic vehicles are referred to as lentiviral vectors. They appear to be better suited for particular applications, such as transgene delivery into stem cells, compared to other viral- and non-viral vectors. This is because Lentivirus-based vectors can efficiently infect nondividing and slowly dividing cells. In the present review article, the current state of understanding of HIV-1 is discussed and the main characteristics that had an impact on vector design are outlined. A historical view on the vector concept is presented to facilitate discussion of recent results in vector engineering in a broader context. Subsequently, a state of the art overview concerning vector construction and vector production is given. This review also touches upon the subject of lentiviral vector safety and related topics that can be helpful in addressing this issue are discussed. Finally, examples of Lentivirus-based gene delivery systems and their applications are presented, with emphasis on animal transgenesis and human gene therapy.

Ograniczanie wyników

2 Acta Biochimica Polonica

2 Cellular and Molecular Biology Letters

1 Audouy S.

1 Cichon T.

1 Engberts J.

1 Hoekstra D.

1 Jazowiecka-Rakus J.

1 Kacprzak M. M.

1 Langner M.

1 Makselon M.

1 Oberle V.

1 Pluta K.

1 Smolarczyk R.

1 Sochanik A.

1 Strozyk M.

1 Szala S.

1 Zuhorn I.

1 2009

1 2004

1 2000

1 1999

In vivo gene transfer using cetylated polyethylenimine

Synthetic amphiphiles as non-viral vectors in gene therapy. Development and mechanisms

The intracellular fate of non-viral DNA carriers

Use of HIV as a gene transfer vector