Every year, right after New Year holidays, we are picking the most significant and interesting methods of the year. The winner of 2011 – targeted genome editing by zinc finger nucleases.
Few major advances in using zinc finger nucleases (ZNF) technology were made in 2011.
First of all, the usability of this technology in clinic was demonstrated in HIV cell therapy trial. Tthe first preliminary results of Sangamo‘s HIV clinical trial were reported. They use ZNF technology (targenig CCR5) in order to make human T-cells resistant to HIV. John Zaia from the City of Hope Medical Center proposed to used the same approach, but on hematopoietic progenitor/ stem cells (HSPC). They will isolate HSPC from the patients with AIDS, use CCR5-specific ZFNs and re-infuse “edited” HIV-resistant stem cells.
Second, demonstration of in vivo genome editing using ZNF technology. Researchers were able to restore haemostasis in a mouse model of haemophilia by applying specific ZNFs.
Third, few research groups have demonstrated the possibility of using ZNFs for modeling and fixing disease in human iPS cells:
- X-linked chronic granulomatous disease
- Generation of isogenic pluripotent stem cells from Parkinson’s disease patients
- Targeted gene correction of laminopathy-associated LMNA mutations
I’m happy to tell you that ZNFs-based genome editing was chosen by Nature Methods as “Method of the Year 2011“.
Please watch this wonderful video to learn more about this magic technology:
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