Molecular Therapy: Methods & Clinical Development (Jun 2019)

Advancements and Obstacles of CRISPR-Cas9 Technology in Translational Research

  • Liting You,
  • Ruizhan Tong,
  • Mengqian Li,
  • Yuncong Liu,
  • Jianxin Xue,
  • You Lu

Journal volume & issue
Vol. 13
pp. 359 – 370

Abstract

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The expanding CRISPR-Cas9 technology is an easily accessible, programmable, and precise gene-editing tool with numerous applications, most notably in biomedical research. Together with advancements in genome and transcriptome sequencing in the era of metadata, genomic engineering with CRISPR-Cas9 meets the developmental requirements of precision medicine, and clinical tests using CRISPR-Cas9 are now possible. This review summarizes developments and established preclinical applications of CRISPR-Cas9 technology, along with its current challenges, and highlights future applications in translational research. Keywords: CRISPR-Cas9, gene editing, off-target, gene therapy, tumor immunotherapy