Amor D
Genetic Health Services Victoria, Royal Children's Hospital, Melbourne, Victoria.
Aust Fam Physician. 2001 Oct;30(10):953-8.
Gene therapy represents an exciting new possibility for the treatment of rare genetic disorders and common multifactorial diseases.
To provide an overview of the principals of gene therapy and to outline the current progress of human gene therapy trials.
Gene therapy is a novel approach to treat, cure or prevent disease by changing the expression of a person's genes. Typically gene therapy involves using a vector such as a virus to deliver a therapeutic gene to the appropriate target cells. Gene therapy is still in its infancy and is not yet available outside clinical trials. Gene therapy was originally envisaged as a treatment of monogenic disorders, but the majority of gene therapy trials now involve the treatment of cancer, infectious diseases and vascular disease. Human gene therapy raises several important ethical issues, in particular the potential use of genetic therapies for genetic enhancement and the potential impact of germline gene therapy on future generations.
基因治疗为罕见遗传疾病和常见多因素疾病的治疗带来了令人兴奋的新可能性。
概述基因治疗的原理，并简述人类基因治疗试验的当前进展。
基因治疗是一种通过改变人的基因表达来治疗、治愈或预防疾病的新方法。通常，基因治疗涉及使用病毒等载体将治疗性基因传递到适当的靶细胞。基因治疗仍处于起步阶段，尚未在临床试验之外应用。基因治疗最初被设想用于治疗单基因疾病，但现在大多数基因治疗试验都涉及癌症、传染病和血管疾病的治疗。人类基因治疗引发了几个重要的伦理问题，特别是基因疗法用于基因增强的潜在用途以及生殖系基因治疗对后代的潜在影响。
