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.

中文翻译：

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腺相关病毒介导的基因治疗

载体的选择是基因治疗中最关键的一步。腺相关病毒（AAV）；第三代载体因其非致病性、安全性好、免疫反应低、转导非分裂细胞效率高、遗传物质转移能力大、表达时间长等优点而受到科学家的广泛关注。遗传有效负载。AAV 有多种血清型，每种血清型都显示出对特定细胞的趋向性。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营销应用，可以完美地融入宿主目标基因组的预期位点，从而实现长寿转基因的表达。一些治疗产品使用用于治疗脂蛋白脂肪酶缺乏症 (LPLD) 的 AAV 载体，并通过肌肉注射来治疗引入视网膜下腔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症和 rAAV2 -CFTR载体被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精准基因疗法的奇迹，用于治疗最严重甚至有时致命的疾病。