Researchers at Great Ormond Street Hospital for Children (GOSH) and UCL Great Ormond Street Institute of Child Health (UCL GOS ICH) have used CRISPR/Cas9 technology to engineer donor T cells to try to treat seriously ill children with resistant leukaemia, who had otherwise exhausted all available therapies.
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Researchers have used CRISPR/Cas9 technology to engineer donor T cells to treat critically ill children with resistant leukemia who had otherwise exhausted all available therapies. The Phase I trial, published in Science Translational Medicine , is the first use of CRISPR-edited "universal" cells in humans and represents a significant advance in the use of genetically engineered cells for cancer treatment. In the trial, the research team constructed and applied a new generation of genome-edited "universal" T cells, which builds on previous work that used older, less precise technology. The T cells were modified using CRISPR. In addition, the piece of genetic code allows the T cells to express a CAR receptor that can recognize a marker on the surface of cancerous B cells and then destroy them. The T cells were then genetically modified with CRISPR so that they could be used "off the shelf" without the need for a donor match. In specialist clean rooms, researchers manufactured their banks of donor CAR T-cells using a single disabled virus to transfer both the CAR and a CRISPR guidance system, and then applied cutting-edge mRNA technology to activate the gene editing steps.
Researchers have used CRISPR/Cas9 technology to engineer donor T cells to treat critically ill children with resistant leukemia who had otherwise exhausted all available therapies. The Phase I trial, published in Science Translational Medicine , is the first use of CRISPR-edited "universal" cells in humans and represents a significant advance in the use of genetically engineered cells for cancer treatment. In the trial, the research team constructed and applied a new generation of genome-edited "universal" T cells, which builds on previous work that used older, less precise technology. The T cells were modified using CRISPR. In addition, the piece of genetic code allows the T cells to express a CAR receptor that can recognize a marker on the surface of cancerous B cells and then destroy them. The T cells were then genetically modified with CRISPR so that they could be used "off the shelf" without the need for a donor match. In specialist clean rooms, researchers manufactured their banks of donor CAR T-cells using a single disabled virus to transfer both the CAR and a CRISPR guidance system, and then applied cutting-edge mRNA technology to activate the gene editing steps.
Une thérapie génique et universelle. La technologie CRISPR/Cas9 est un espoir dans la thérapie de la leucémie résistante. Une attention toute particulière doit toutefois se porter sur l'insertion génétique qui doit être finement régulée afin d'éviter les effets délétères.