Relieving bottlenecks in RNA drug discovery for retinal diseases.
Relieving bottlenecks in RNA drug discovery for retinal diseases.
复制标题
缓解视网膜疾病 RNA 药物发现的瓶颈。
DOI：
10.1007/978-1-4614-0631-0_20
复制
发表时间：
2012
影响因子：
--
通讯作者：
Kolniak,TiffanyA
中科院分区：
文献类型：
--
作者：
Sullivan,JackM;Yau,EdwinH;Taggart,RThomas;Butler,MarkC;Kolniak,TiffanyA
开发有效和安全的转录后基因沉默（PTGS）试剂是一项具有挑战性的科学奋进，在许多层面上包含“生物复杂性”。靶mRNA表现出一定水平的结构复杂性，这极大地限制了PTGS试剂的退火。PTGS试剂是大分子RNA，其必须被设计成折叠成能够切割靶mRNA的催化活性结构。推进和超越生物复杂性需要高通量筛选的新技术，以有效和快速地评估参与RNA药物发现的一组生物和实验变量。
The development of efficacious and safe post transcriptional gene silencing (PTGS) agents is a challenging scientific endeavor that embraces “biocomplexity” at many levels. The target mRNA exhibits a level of structural complexity that profoundly limits annealing of PTGS agents. PTGS agents are macromolecular RNAs that must be designed to fold into catalytically active structures able to cleave the target mRNA. Pushing into and beyond the biological complexity requires new technologies for high throughput screening to efficiently and rapidly assess a set of biological and experimental variables engaged in RNA drug discovery.