腺相关病毒介导的基因治疗。

IF 1.5 4区 医学 Q4 BIOTECHNOLOGY & APPLIED MICROBIOLOGY
Hassan Zaman, Aakif Khan, Khalid Khan, Shazma Toheed, Muhammad Abdullah, Hafiz Muhammad Zeeshan, Abdul Hameed, Muhammad Umar, Muhammad Shahid, Kausar Malik, Samia Afzal
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引用次数: 0

摘要

载体的选择是基因治疗中最关键的一步。腺相关病毒;第三代载体,由于其无致病性、良好的安全性、低免疫反应、高效转导非分裂细胞、大容量转移遗传物质和长期表达遗传有效载荷等优点,作为载体受到了科学家的广泛关注。AAV具有多种血清型,并且每种血清型都显示出对特定细胞的嗜性。不同的血清型用于靶向肝脏、肺、肌肉、视网膜、心脏、中枢神经系统、肾脏等。此外,基于AAV的基因疗法具有巨大的市场应用,可以完美地结合到宿主靶基因组的预期位点,从而实现转基因的终身表达。一些治疗产品使用用于治疗脂蛋白脂酶缺乏症(LPLD)的AAV载体,并将其肌肉注射,以治疗引入视网膜下间隙的突变视网膜色素上皮RPE65(RPE65),静脉输注以治疗脊髓性肌萎缩,将rAAV2-CFTR载体引入鼻上皮细胞以治疗囊性纤维化。AAV疗法和其他此类跨学科方法可以创造奇迹,为治疗最严重、有时甚至致命的疾病创造精确的基因疗法。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
Adeno-Associated Virus-Mediated Gene Therapy.

Choice of vector is the most critical step in gene therapy. Adeno-associated viruses (AAV); third generation vectors, are getting much attention of scientists to be used as vehicles due to their non-pathogenicity, excellent safety profile, low immune responses, great efficiency to transduce non-dividing cells, large capacity to transfer genetic material and long-term expression of genetic payload. AAVs have multiple serotypes and each serotype shows tropism for a specific cell. Different serotypes are used to target liver, lungs, muscles, retina, heart, CNS, kidneys, etc. Furthermore, AAV based gene therapies have tremendous marketing applications that can be perfectly incorporated in the anticipated sites of the host target genome resulting in life long expression of transgenes. Some therapeutic products use AAV vectors that are used to treat lipoprotein lipase deficiency (LPLD) and it is injected intramuscularly, to treat mutated retinal pigment epithelium RPE65 (RPE65) that is introduced to subretinal space, an intravenous infusion to treat spinal muscular atrophy and rAAV2-CFTR vector is introduced into nasal epithelial cells to treat cystic fibrosis. AAV therapies and other such interdisciplinary methodologies can create the miracles for the generation of precision gene therapies for the treatment of most serious and sometimes fatal disorders.

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来源期刊
Critical Reviews in Eukaryotic Gene Expression
Critical Reviews in Eukaryotic Gene Expression 生物-生物工程与应用微生物
CiteScore
2.70
自引率
0.00%
发文量
67
审稿时长
1 months
期刊介绍: Critical ReviewsTM in Eukaryotic Gene Expression presents timely concepts and experimental approaches that are contributing to rapid advances in our mechanistic understanding of gene regulation, organization, and structure within the contexts of biological control and the diagnosis/treatment of disease. The journal provides in-depth critical reviews, on well-defined topics of immediate interest, written by recognized specialists in the field. Extensive literature citations provide a comprehensive information resource. Reviews are developed from an historical perspective and suggest directions that can be anticipated. Strengths as well as limitations of methodologies and experimental strategies are considered.
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