Department of Medical Biotechnology, School of Advanced Technologies in Medicine, Tehran University of Medical Sciences, Tehran, Iran.
Clin Exp Med. 2023 Aug;23(4):1107-1121. doi: 10.1007/s10238-022-00925-x. Epub 2022 Oct 25.
Genetics and molecular genetic techniques have changed many perspectives and paradigms in medicine. Using genetic methods, many diseases have been cured or alleviated. Gene therapy, in its simplest definition, is application of genetic materials and related techniques to treat various human diseases. Evaluation of the trends in the field of medicine and therapeutics clarifies that gene therapy has attracted a lot of attention due to its powerful potential to treat a number of diseases. There are various genetic materials that can be used in gene therapy such as DNA, single- and double-stranded RNA, siRNA and shRNA. The main gene editing techniques used for in vitro and in vivo gene modification are ZNF, TALEN and CRISPR-Cas9. The latter has increased hopes for more precise and efficient gene targeting as it requires two separate recognition sites which makes it more specific and can also cause rapid and sufficient cleavage within the target sequence. There must be carriers for delivering genes to the target tissue. The most commonly used carriers for this purpose are viral vectors such as adenoviruses, adeno-associated viruses and lentiviruses. Non-viral vectors consist of bacterial vectors, liposomes, dendrimers and nanoparticles.
遗传学和分子遗传技术已经改变了医学的许多观点和范例。利用遗传方法，许多疾病已经得到治愈或缓解。基因治疗的最简单定义是应用遗传物质和相关技术来治疗各种人类疾病。评估医学和治疗学领域的趋势表明，由于其治疗多种疾病的强大潜力，基因治疗引起了广泛关注。有多种遗传物质可用于基因治疗，如 DNA、单链和双链 RNA、siRNA 和 shRNA。用于体外和体内基因修饰的主要基因编辑技术有 ZNF、TALEN 和 CRISPR-Cas9。后者增加了对更精确和高效基因靶向的希望，因为它需要两个单独的识别位点，这使其更具特异性，并且还可以在靶序列内迅速且充分地切割。必须有载体将基因递送到靶组织。为此目的最常用的载体是病毒载体，如腺病毒、腺相关病毒和慢病毒。非病毒载体包括细菌载体、脂质体、树枝状聚合物和纳米颗粒。
