Sullenger Bruce A, Gilboa Eli
Department of Surgery, Duke University Medical Center, Durham, NC 27710, USA.
Nature. 2002 Jul 11;418(6894):252-8. doi: 10.1038/418252a.
RNA is a versatile biological macromolecule that is crucial in mobilizing and interpreting our genetic information. It is not surprising then that researchers have sought to exploit the inherent properties of RNAs so as to interfere with or repair dysfunctional nucleic acids or proteins and to stimulate the production of therapeutic gene products in a variety of pathological situations. The first generation of the resulting RNA therapeutics are now being evaluated in clinical trials, raising significant interest in this emerging area of medical research.
RNA是一种多功能的生物大分子,在调动和解读我们的遗传信息方面至关重要。因此,研究人员试图利用RNA的固有特性来干扰或修复功能失调的核酸或蛋白质,并在各种病理情况下刺激治疗性基因产物的产生,这并不奇怪。由此产生的第一代RNA疗法目前正在临床试验中进行评估,引发了人们对这一新兴医学研究领域的极大兴趣。