The fourth annual BRDS on genome editing and silencing for precision medicines.
The fourth annual BRDS on genome editing and silencing for precision medicines.
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DOI：
10.1007/s13346-017-0457-5
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发表时间：
2018-03
影响因子：
5.4
通讯作者：
Mahato RI
中科院分区：
文献类型：
--
作者：
Chaudhary AK;Bhattarai RS;Mahato RI
精确的医学是治疗人类疾病，因为它专注于对患者的基因，环境和生活方式的量身定制，但对个性化药物的需求已为将核酸变成治疗而开放。具有有效的基因组工具工具的操纵和序列的核变化可以使人类细胞中的遗传变化更容易，而纳米技术的效率和精度更高。在2017年9月7日至8日，在内布拉斯加州大学医学中心（UNMC）举行的第四届年度生物制药研发研讨会（BRDS）中，我们涵盖了开发用于遗传疾病的治疗和诊断的工具的不同方面。
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.
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影响因子：
12.3
作者：
Quadros RM;Miura H;Harms DW;Akatsuka H;Sato T;Aida T;Redder R;Richardson GP;Inagaki Y;Sakai D;Buckley SM;Seshacharyulu P;Batra SK;Behlke MA;Zeiner SA;Jacobi AM;Izu Y;Thoreson WB;Urness LD;Mansour SL;Ohtsuka M;Gurumurthy CB
通讯作者：
Gurumurthy CB
影响因子：
64.8
作者：
Eyquem J;Mansilla-Soto J;Giavridis T;van der Stegen SJ;Hamieh M;Cunanan KM;Odak A;Gönen M;Sadelain M
通讯作者：
Sadelain M
影响因子：
5.5
作者：
André, F;Scharz, NEC;Zitvogel, L
通讯作者：
Zitvogel, L
影响因子：
7
作者：
Ramakrishna S;Kwaku Dad AB;Beloor J;Gopalappa R;Lee SK;Kim H
通讯作者：
Kim H
影响因子：
11.1
作者：
Kaufmann, Kerstin B.;Buening, Hildegard;Galy, Anne;Schambach, Axel;Grez, Manuel
通讯作者：
Grez, Manuel