【摘 要】
：
Oligonucleotides such as siRNAs, antimiRs, miRNA mimics, IncRNAs and antisense oligonucleotides offer unique opportunities for developing novel therapeutic
【机 构】
：
R&DAllianceManagement,Sanofi,GermanyScientificManagingDirectorofCOMPACTConsortium,InnovativeMedicine
【出 处】
：
The 2nd Canton Nucleic Acids Forum(第二届广州核酸国际论坛)
论文部分内容阅读
Oligonucleotides such as siRNAs, antimiRs, miRNA mimics, IncRNAs and antisense oligonucleotides offer unique opportunities for developing novel therapeutic modalities against disease-relevant targets by rational drug design instead of random screening for small molecule modulators.Moreover, miRNAs open a novel target space by modulation of disease relevant miRNA-regulated biochemical pathways and networks of pathways instead of modulation of single targets.However,a key hurdle for translating oligonucleotide-based therapeutics into clinical applications remains their effective delivery across biological barriers and cellular membranes in order to reach their disease-relevant targets in the patient.This presentation will summarize the opportunities of oligonucleotide-based therapeutics for innovation in the pharmaceutical industry,illustrate the major hurdles for translating these therapeutic modalities into the clinic, and describe examples of concepts that pharmaceutical companies are employing to overcome these hurdles.
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