Gene-editing research update

25 Jan 2017, 3:37 p.m.

Healthy human T Cell

On Wednesday 25 January, details of two patients successfully treated for their leukaemia at Great Ormond Street Hospital using gene-editing technology were published in the journal, Science Translational Medicine. 

One of the patients was Layla Richards who was the first person in the world to receive gene-edited immune cells to treat drug resistant leukaemia. Her story received widespread national and international media coverage.

Commenting on the publication, Professor Waseem Qasim, Professor of Cell and Gene Therapy at UCL Great Ormond Street Institute of Child Health (ICH), Consultant Immunologist at Great Ormond Street Hospital and who carried out the work, said:

“We are delighted to have shown that gene-edited cells can successfully treat leukaemia that has not responded to standard treatments.

“While both patients are now at home and are doing well, we must treat these results with some caution as we don’t yet know if the technique will be successful in treating a larger number of patients. Clinical trials are currently underway to assess this.”

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