1.5 小结
核糖核酸(RNA)领域经过几十年的发展,由早期的重点关注编码蛋白基因的RNA,转变到目前重点关注大量的非编码RNA在生命调控及其在医学与农业等领域的应用,而且由最初的基础研究向应用研究、技术开发、成果转化与产业化不断拓展和延伸。从创新链角度看,目前RNA生物学已经发展成相对完善的体系,由前端的基础研究(RNA生成加工与降解、新型RNA识别与鉴定、RNA结构鉴定与功能解析),中端的应用研究(应用于医学、农业等),后端的产业转化(新药、新型诊断产品开发、动植物新品种、新农药与兽药等),以及影响整个领域发展的相关新技术开发(包括研究技术与产业化技术)和RNA信息学(相关数据库与平台建设、算法开发)五大部分组成。未来,随着RNA介导的基因组编辑技术等新技术的发展,以及海量RNA信息被充分挖掘利用,各类RNA在生命中的调控作用将被更清楚地揭示,并与医学与农业交叉融合,推进相关研究成果在这些领域中的应用与产业化,RNA生物学将在人类健康与国民经济发展中发挥重要作用(见图1.13)。
图1.13 RNA领域学科框架
为此,美国、欧盟、日本将RNA生物学作为重点研究领域给予持续资助。该领域新的研究前沿与热点不断涌现;RNA干扰、RNA适配体和RNA介导的基因编辑技术三大核心技术被越来越广泛地应用于疾病治疗、农业生产中,相关研究用技术也发展迅速。
在药物研发与产业化方面,Cortellis数据库收录的目前有效的在研核糖核酸药物共692个,这些药物的重要适应证包括肿瘤、罕见病、传染病、胃肠疾病、呼吸系统疾病、神经系统疾病等。未来以RNA药物为代表的核酸药物市场将进入发展快车道。
我国长期资助各类RNA基础研究项目,并在近年来开始资助RNA医学与农业领域的研究。经检索,我国非编码RNA领域2016—2020年SCI论文62997篇,超过美国,排名全球第一,占全球的54.68%,ESI高水平论文量也已经超过美国,但发表在Nature、Science、Cell三大期刊上的论文量与美国有较大差距;2016—2020年的专利量为6589件,排名第二,仅次于美国,占全球的33.11%;目前在研新药50个,占全球的7.23%,远低于美国的454个(占全球的65.61%),表明我国RNA领域基础研究实力强,但成果转化效率低。比较国际、国内布局重点可以看出,与美国、欧盟相比,我国近年来开始重视RNA医学等应用研究,但资助重点仍在基础研究、应用基础研究方面。因此,我国在保持基础研究优势的同时,需要加强RNA应用研究资助,促进基础研究与应用研究的成果转化,重视技术开发与平台建设,出台相关政策促进研究成果转化。
致谢 中山大学生物工程研究中心主任、中国生物化学与分子生物学会核糖核酸专业委员会主任屈良鹄教授,对本章提出了宝贵的意见和建议,谨致谢忱。
执笔人:中国科学院上海营养与健康研究所/中国科学院上海生命科学信息中心
阮梅花、袁天蔚、于建荣、熊燕
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