Department of Pharmaceutical Sciences, University of Nebraska Medical Center (UNMC), 986025 Nebraska Medical Center, Omaha, NE, 68198-6025, USA.
Drug Deliv Transl Res. 2018 Feb;8(1):266-272. doi: 10.1007/s13346-017-0457-5.
Precision medicine is promising for treating human diseases, as it focuses on tailoring drugs to a patient's genes, environment, and lifestyle. The need for personalized medicines has opened the doors for turning nucleic acids into therapeutics. Although gene therapy has the potential to treat and cure genetic and acquired diseases, it needs to overcome certain obstacles before creating the overall prescription drugs. Recent advancement in the life science has helped to understand the effective manipulation and delivery of genome-engineering tools better. The use of sequence-specific nucleases allows genetic changes in human cells to be easily made with higher efficiency and precision than before. Nanotechnology has made rapid advancement in the field of drug delivery, but the delivery of nucleic acids presents unique challenges. Also, designing efficient and short time-consuming genome-editing tools with negligible off-target effects are in high demand for precision medicine. In the fourth annual Biopharmaceutical Research and Development Symposium (BRDS) held at the University of Nebraska Medical Center (UNMC) on September 7-8, 2017, we covered different facets of developing tools for precision medicine for therapeutic and diagnosis of genetic disorders.
精准医疗在治疗人类疾病方面很有前景，因为它专注于根据患者的基因、环境和生活方式来定制药物。对个性化药物的需求为将核酸转化为治疗药物开辟了道路。尽管基因疗法有可能治疗和治愈遗传和获得性疾病，但在创造整体处方药物之前，它需要克服某些障碍。生命科学的最新进展有助于更好地理解对基因组工程工具的有效操作和传递。使用序列特异性核酸酶可以比以前更高的效率和精度轻松地对人类细胞中的遗传变化进行修饰。纳米技术在药物输送领域取得了快速进展，但核酸的输送带来了独特的挑战。此外，设计高效且耗时短、脱靶效应小的基因组编辑工具对于精准医疗也有很高的需求。在 2017 年 9 月 7 日至 8 日于内布拉斯加大学医学中心（UNMC）举行的第四届年度生物制药研发研讨会（BRDS）上，我们涵盖了开发用于治疗和诊断遗传疾病的精准医学工具的不同方面。
