Proven and novel strategies for efficient editing of the human genome.
Targeted gene editing with designer nucleases has become increasingly popular. The most commonly used designer nuclease platforms are engineered meganucleases, zinc-finger nucleases, transcription activator-like effector nucleases and the clustered regularly interspaced short palindromic repeat/Cas9 system. These powerful tools have greatly facilitated the generation of plant and animal models for basic research, and harbor an enormous potential for applications in biotechnology and gene therapy. This review recapitulates proven concepts of targeted genome engineering in primary human cells and elaborates on novel concepts that became possible with the dawn of RNA-guided nucleases and RNA-guided transcription factors.
Institute for Cell and Gene Therapy, University Medical Center Freiburg, Freiburg, Germany; Center for Chronic Immunodeficiency, University Medical Center Freiburg, Freiburg, Germany.
Claudio
C
Mussolino
Institute for Cell and Gene Therapy, University Medical Center Freiburg, Freiburg, Germany; Center for Chronic Immunodeficiency, University Medical Center Freiburg, Freiburg, Germany; Spemann Graduate School of Biology and Medicine (SGBM), University of Freiburg, Freiburg, Germany.
Tafadzwa
T
Mlambo
Institute for Cell and Gene Therapy, University Medical Center Freiburg, Freiburg, Germany; Center for Chronic Immunodeficiency, University Medical Center Freiburg, Freiburg, Germany. Electronic address: toni.cathomen@uniklinik-freiburg.de.
Toni
T
Cathomen
D005818:Genetic Engineering; D015894:Genome, Human; D006801:Humans