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연구자들이 자신의 연구와 전문성을 널리 알리고, 새로운 협력의 기회를 만들 수 있는 네트워킹 공간이에요.
논문 기본 정보
- 자료유형
- 학술저널
- 저자정보
- 발행연도
- 2005.12
- 수록면
- 397 - 405 (9page)
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Gene therapy has offered potential promises for treatment of the diseases that have not been curable using the currently available therapeutic approaches. To develop a successful gene therapeutic medicine, two essential components of gene therapy should be considered thoroughly. One is to choose a therapeutic gene and the other is to select a safe and efficient gene delivery vehicle to introduce a therapeutic gene to the target tissue. The latter has been the major challenge to develop a more efficient gene therapeutic approach. Here we evaluated preclinical safety of adeno-associated virus serotype 2(AAV2)-mediated gene delivery vector containing the green fluorescent protein (GFP), recombinant AAV2-GFP (rAAV2-GFP) and also examined in vitro transduction efficiency of rAAV2-GFP. The rAAV vectors were infected in MKN74 cancer cells much higher (＞ 90%) than in HUVECs. The results of the single dose toxicity and the immunotoxicity evaluations of rAAV2-GFP showed no toxic effects as a therapeutic vector. However, the genotoxicity study revealed that the rAAV2-GFP vectors were inserted into the genome of testis, but not inserted into the genomes of other organs such as liver and ovary. The data of rAAV2-GFP vector distribution monitored by a confocal microscopy demonstrated that the vectors transduced most of the tested organs although the GFP expression directed by rAAV was quite weak. The GFP expression in the liver and kidney was slightly higher than that in the rest of the organs. The results from RT-PCR to quantitate the expressed GFP transcripts in the tested organs were largely consistent with those of GFP protein expression monitored by a confocal microscopy. Taken altogether, our present study demonstrated that no preclinical safety evaluations that we carried out revealed any adverse effects as a gene delivery vector and suggests that AAV2-mediated gene delivery system can be one of the safest gene therapy vectors.
목차
Materials and Methods
Results and Discussion
Acknowledgments
References
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UCI(KEPA) : I410-ECN-0101-2009-510-016362616