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Adeno-associated virus vectors in gene therapy

100%

Kamieniarz K., Gozdzicka-Jozefiak A.

Biotechnologia

2005

issue 1

79-94

The development of targeted vectors, capable of tissue-specific transduction, remains one of the most important aims of vector modification for gene therapy applications. The gaining popularity of recombinant vectors based on adeno-associated viruses (rAAV) in gene therapy can be attributed to their lack of pathogenicity, added safety due to their replication defectiveness, relatively low immunogenicity and their ability to mediate long-term expression in a variety of tissues. The major shortcoming of these vectors is their small packaging capacity. AAV vectors have already broad utility in the therapy of many diseases, including neurological disorders and various types of cancer. Moreover, they can also serve as transfer vehicles for DNA vaccines.

Biotechnology in regenerative and reproductive medicine

81%

Kamieniarz K., Nawrot R., Grajek K., Gozdzicka-Jozefiak A.

Biotechnologia

2006

issue 2

31-48

Rapid progress in molecular biology, genetics, and mammalian biotechnology has revolutionized diagnostic, therapeutic and reproductive cloning in mammals. Recently, several human gene products have been able to be pharmaceutically explored in transgenic organisms and employed for medical applications. When organs or tissues are irreparably damaged, they may be also replaced with an artificial device or donor organ. Promising and also controversial application for therapeutic and regenerative medicine is stem cells engineering.

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2 Biotechnologia

1 2006

1 2005

2 Gozdzicka-Jozefiak A.

2 Kamieniarz K.

1 Grajek K.

1 Nawrot R.

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Adeno-associated virus vectors in gene therapy

Biotechnology in regenerative and reproductive medicine