Saraswat P, Soni R R, Bhandari A, Nagori B P
Mahatma Gandhi Medical College and Hospital, RIICO Institutional Area, Sitapura, Jaipur-302 022, India.
Indian J Pharm Sci. 2009 Sep;71(5):488-98. doi: 10.4103/0250-474X.58169.
Human gene therapy is the introduction of new genetic material into the cells of an individual with the intention of producing a therapeutic benefit for the patient. Deoxyribonucleic acid and ribonucleic acid are used in gene therapy. Over time and with proper oversight, human gene therapy might become an effective weapon in modern medicine's arsenal to help fight diseases such as cancer, acquired immunodeficiency syndrome, diabetes, high blood pressure, coronary heart disease, peripheral vascular disease, neurodegenerative diseases, cystic fibrosis, hemophilia and other genetic disorders. Gene therapy trials in humans are of two types, somatic and germ line gene therapy. There are many ethical, social, and commercial issues raised by the prospects of treating patients whose consent is impossible to obtain. This review summarizes deoxyribonucleic acid-based therapeutics and gene transfer technologies for the diseases that are known to be genetic in origin. Deoxyribonucleic acid-based therapeutics includes plasmids, oligonucleotides for antisense and antigene applications, deoxyribonucleic acid aptamers and deoxyribonucleic acidzymes. This review also includes current status of gene therapy and recent developments in gene therapy research.
人类基因治疗是将新的遗传物质导入个体细胞,旨在为患者带来治疗益处。基因治疗中使用脱氧核糖核酸(DNA)和核糖核酸(RNA)。随着时间的推移并在适当监督下,人类基因治疗可能会成为现代医学武器库中的一种有效手段,以帮助对抗诸如癌症、获得性免疫缺陷综合征、糖尿病、高血压、冠心病、外周血管疾病、神经退行性疾病、囊性纤维化、血友病及其他遗传疾病等。人类基因治疗试验有两种类型,即体细胞基因治疗和生殖系基因治疗。对于那些无法获得其同意的患者进行治疗,这一前景引发了许多伦理、社会和商业问题。本综述总结了针对已知起源于遗传的疾病的基于脱氧核糖核酸的治疗方法和基因转移技术。基于脱氧核糖核酸的治疗方法包括质粒、用于反义及反基因应用的寡核苷酸、脱氧核糖核酸适配体和脱氧核酶。本综述还包括基因治疗的现状以及基因治疗研究的最新进展。