Progress Towards Liver and Lung-Directed Gene Therapy with Helper- Dependent Adenoviral Vectors

Nicola Brunetti-Pierri Philip Ng

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Progress Towards Liver and Lung-Directed Gene Therapy with Helper- Dependent Adenoviral Vectors

机译：辅助性腺病毒载体在肝和肺相关基因治疗方面的进展

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Helper-dependent adenoviral vectors (HDAd) have several characteristics making them very attractive for human gene therapy. These vectors are completely devoid of viral coding sequences and are able to mediate high efficiency transduction in vivo to direct high level transgene expression with negligible chronic toxicity. Progress towards liver and lung directed gene therapy with HDAd as well as the current obstacles facing human applications and possible strategies to overcome these obstacles are discussed.

机译：依赖于助手的腺病毒载体（HDAd）具有几个特征，使其对人类基因治疗非常有吸引力。这些载体完全没有病毒编码序列，并且能够介导体内高效转导以指导高水平转基因表达，而慢性毒性可忽略不计。讨论了用HDAd向肝和肺定向基因治疗的进展以及人类应用面临的当前障碍以及克服这些障碍的可能策略。

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来源
《Current Gene Therapy》 |2009年第5期|p.329-340|共12页
作者
Nicola Brunetti-Pierri Philip Ng;
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作者单位

Department of Molecular and Human Genetics, Baylor College of Medicine, One Baylor Plaza, 630E, Houston, TX 77030, USA.;

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原文格式 PDF
正文语种 eng
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关键词
Gene therapy, adenoviral vectors, helper-dependent adenoviral vectors, gutless, cystic fibrosis;

机译：基因治疗;腺病毒载体;辅助性腺病毒载体;无肠;囊性纤维化;

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1. Progress towards liver and lung-directed gene therapy with helper-dependent adenoviral vectors. [J] . Brunetti Pierri N, Ng P Current gene therapy . 2009,第5期

机译：辅助性腺病毒载体在肝和肺相关基因治疗方面的进展。
2. Progress towards the clinical application of helper-dependent adenoviral vectors for liver and lung gene therapy. [J] . Brunetti-Pierri N, Ng P Current Opinion in Molecular Therapeutics . 2006,第5期

机译：辅助依赖型腺病毒载体在肝和肺基因治疗中的临床应用进展。
3. Progress and prospects: gene therapy for genetic diseases with helper-dependent adenoviral vectors. [J] . Brunetti-Pierri N, Ng P Gene therapy . 2008,第8期

机译：进展和前景：遗传性疾病的基因治疗与辅助依赖的腺病毒载体。
4. An Experimental Comparing Analysis Research on the Nano-Vectors for Drug Delivery and for Gene Therapy [C] . Tang Shao-Pu Advances in biomedical research . 2010

机译：纳米载体用于药物传递和基因治疗的实验比较分析研究
5. Engineering polypeptide-coated adenoviral gene delivery vectors for systemic cancer therapy. [D] . Koh, Sok Boon Shuwen. 2009

机译：工程性多肽包被的腺病毒基因递送载体，用于系统性癌症治疗。
6. Progress and prospects: gene therapy for genetic diseases with helper-dependent adenoviral vectors [O] . N Brunetti-Pierri, P Ng -1

机译：进展和前景：辅助性腺病毒载体对遗传性疾病的基因治疗
7. Helper-dependent adenoviral vectors for liver-directed gene therapy of primary hyperoxaluria type 1 [O] . Castello, R, Borzone, R, D'Aria, S, 2015

机译：辅助依赖性腺病毒载体，用于原发性高草酸尿1型的肝脏定向基因治疗
8. Transcription Activator Reprogramming Gene Therapy (TARGET) of Breast Cancer Cells with Adenoviral Vectors for Interferon Regulatory Factors; Final rept. 15 Jul 2005-14 Jul 2006 [R] . Yim, J. H. 2006

机译：乳腺癌细胞的转录激活子重编程基因治疗（TaRGET）与腺病毒载体的干扰素调节因子;最终的评论。 2005年7月15日至2006年7月14日

Progress Towards Liver and Lung-Directed Gene Therapy with Helper- Dependent Adenoviral Vectors

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