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Genetic Engineering Publications - GEG Tech top picks
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BigField GEG Tech's insight:
In this review, Matthew H, Porteus discusses about the progress and challenges of genome editing of blood. 
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BigField GEG Tech's insight:
In this study, the scientists report CRISPR/Cas9 targeting of two clinically relevant genes, B2M and CCR5, in primary human CD4+ T cells and CD34+ hematopoietic stem and progenitor cells (HSPCs). Use of single RNA guides led not to efficient mutagenesis in T cells. However, their results results demonstrate that CRISPR/Cas9 can efficiently ablate genes in HSPCs with minimal off-target mutagenesis, which could have broad applicability for hematopoietic cell-based therapy.
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BigField GEG Tech's insight:
In this review the authors summarize the potential of bone marrow gene therapy for the treatment of HIV/AIDS. A broad range of antiviral strategies are discussed, with a particular focus on RNA-based therapies.
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BigField GEG Tech's insight:
The authors used phiC31 integrase to insert a copy of a β-globin / puromycin (purr) vector into specific genomic locations of a human hematopoietic cell line. After genomic integration, they used Cre recombinase to remove the bacterial backbone and purr.Following that, the stable β-chain expression was continued for several months in the absence of selective pressure.
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![Long-term and efficient expression of human [beta]-globin gene in a hematopoietic cell line using a new site-specific integrating non-viral system | Genetic Engineering Publications - GEG Tech top picks | Scoop.it](https://img.scoop.it/McIgc7_5fF4eSerUlFxk4jl72eJkfbmt4t8yenImKBVvK0kTmF0xjctABnaLJIm9)
This article describes the protocol for the delivery of reagents for targeted genome editing to CD34(+) hematopoietic stem/progenitor cells (HSPCs). While optimization steps might be needed for each specific application with respect tonuclease and donor template amount, adherence to this protocol will serve as an excellent starting point for this further work.