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gene-edited immune cells used in human patient with leukaemia – a case study

A team at Great Ormond Street Hospital (GOSH) and the UCL Institute of Child Health has used the TALEN gene editing technique to modify immune T-cells, in attempt to treat a patient with relapsed acute lymphoblastic leukaemia. TALENs (Transcription Activator-Like Effector Nucleases) are molecular tools that act like scissors, cutting specific gene sequences. In this case, scientists used TALENs to engineer immune T-cells to target leukaemia cells.

This is the first time this technique has been attempted in a person with leukaemia.

This is not published or peer-reviewed work (there is no journal paper), but the case will be discussed at the American Society of Haematology annual meeting in December.

The scientists involved in this technique and the clinician involved in the patient’s care came to the SMC to discuss issues such as:

  • how is this patient now, and when and how will we know if this treatment has worked?
  • what is the science behind the technique?
  • might this technique offer hope for other patients or other cancers?
  • what are the limitations, uncertainties and unknowns?
  • how can we interpret a single case study like this?

Roundup comments accompanied this briefing.



Prof. Waseem Qasim, Professor of Cell and Gene Therapy, UCL Institute of Child Health, and Consultant Immunologist, Great Ormond Street Hospital (GOSH)

Prof. Adrian Thrasher, Professor in Paediatric Immunology, UCL Institute of Child Health

Dr Paul Veys, director of the Bone Marrow Transplant Unit, Great Ormond Street Hospital (GOSH)

Dr Sujith Samarasinghe has been a Consultant Paediatric Haematologist at Great Ormond Street Hospital

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