In Vivo delivery systems for therapeutic genome editing

Luyao Wang, Fangfei Li, Lei Dang, Chao Liang, Chao Wang, Bing He, Jin Liu, Defang Li, Xiaohao Wu, Xuegong Xu*, Aiping Lyu*, Ge Zhang*

*Corresponding author for this work

Research output: Contribution to journalJournal articlepeer-review

76 Citations (Scopus)
26 Downloads (Pure)


Therapeutic genome editing technology has been widely used as a powerful tool for directly correcting genetic mutations in target pathological tissues and cells to cure of diseases. The modification of specific genomic sequences can be achieved by utilizing programmable nucleases, such as Meganucleases, zinc finger nucleases (ZFNs), transcription activator-like effector nucleases (TALENs), and the clustered regularly-interspaced short palindromic repeat-associated nuclease Cas9 (CRISPR/Cas9). However, given the properties, such as large size, negative charge, low membrane penetrating ability, as well as weak tolerance for serum, and low endosomal escape, of these nucleases genome editing cannot be successfully applied unless in vivo delivery of related programmable nucleases into target organisms or cells is achieved. Here, we look back at delivery strategies having been used in the in vivo delivery of three main genome editing nucleases, followed by methodologies currently undergoing testing in clinical trials, and potential delivery strategies provided by analyzing characteristics of nucleases and commonly used vectors.

Original languageEnglish
Article number626
Number of pages19
JournalInternational Journal of Molecular Sciences
Issue number5
Publication statusPublished - 27 Apr 2016

Scopus Subject Areas

  • Catalysis
  • Molecular Biology
  • Spectroscopy
  • Computer Science Applications
  • Physical and Theoretical Chemistry
  • Organic Chemistry
  • Inorganic Chemistry

User-Defined Keywords

  • Genome editing
  • In vivo delivery systems
  • Programmable nucleases
  • Vectors


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