Delivery technologies for genome editing.
With the recent development of CRISPR technology, it is becoming increasingly easy to engineer the genome. Genome-editing systems based on CRISPR, as well as transcription activator-like effector nucleases (TALENs) and zinc-finger nucleases (ZFNs), are becoming valuable tools for biomedical research, drug discovery and development, and even gene therapy. However, for each of these systems to effectively enter cells of interest and perform their function, efficient and safe delivery technologies are needed. This Review discusses the principles of biomacromolecule delivery and gene editing, examines recent advances and challenges in non-viral and viral delivery methods, and highlights the status of related clinical trials.
David H. Koch Institute for Integrative Cancer Research, Massachusetts Institute of Technology, Cambridge, Massachusetts 02139, USA.
Hao
H
Yin
David H. Koch Institute for Integrative Cancer Research, Massachusetts Institute of Technology, Cambridge, Massachusetts 02139, USA.|Department of Chemical Engineering, Massachusetts Institute of Technology, Cambridge, Massachusetts 02139, USA.
Kevin J
KJ
Kauffman
David H. Koch Institute for Integrative Cancer Research, Massachusetts Institute of Technology, Cambridge, Massachusetts 02139, USA.|Department of Chemical Engineering, Massachusetts Institute of Technology, Cambridge, Massachusetts 02139, USA.|Harvard-Massachusetts Institute of Technology Division of Health Sciences and Technology, Cambridge, Massachusetts 02139, USA.|Institute of Medical Engineering and Science, Massachusetts Institute of Technology, Cambridge, Massachusetts 02139, USA.
Daniel G
DG
Anderson
D000818:Animals; D035843:Biomedical Research / Q000379:methods; D064112:Clustered Regularly Interspaced Short Palindromic Repeats / Q000235:genetics; D055808:Drug Discovery / Q000379:methods; D000072669:Gene Editing / Q000379:methods*; D018014:Gene Transfer Techniques; D005818:Genetic Engineering / Q000379:methods*; D015316:Genetic Therapy / Q000379:methods*; D015894:Genome, Human / Q000235:genetics*; D006801:Humans; D000069896:Transcription Activator-Like Effector Nucleases / Q000235:genetics; D016335:Zinc Fingers / Q000235:genetics
D000069896:Transcription Activator-Like Effector Nucleases