Abstract

2014: A Year of Breakthroughs for Translational Science

Highlights

  • 2014 has been a remarkable year of scientific achievements, from advances in our understanding of molecular pathways of human diseases to the development of new interventional tools to tackle them

  • Clustered regularly interspaced short palindromic repeats (CRISPR) are sequences naturally found in bacterial DNA that work with CRISPR-associated (Cas) nucleases and guide RNAs to protect the bacterial genomes from attacks by targeting specific sequences detected in invading bacteriophages

  • It was reported that the CRISPR-Cas system has successfully been applied as a tool for performing efficient and highly specific genome-wide screening in human cells, and vast improvements have been made in 2014 on increasing target specificity, which opens up limitless possibilities for interrogating gene function relevant to human health and diseases

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Summary

Introduction

2014 has been a remarkable year of scientific achievements, from advances in our understanding of molecular pathways of human diseases to the development of new interventional tools to tackle them. Proof-ofprinciple research has emerged recently, which harnesses the high sequence specificity of the system to cure genetic disorders in animal models as well as creating disease-free stem cells from affected patients.

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