Molecular remission of infant B-ALL after infusion of universal TALEN gene-edited CAR T cells

Waseem Qasim; Hong Zhan; Sujith Samarasinghe; Stuart Adams; Persis Amrolia; Sian Stafford; Katie Butler; Christine Rivat; Gary Wright; Kathy Somana; Sara Ghorashian; Danielle Pinner; Gul Ahsan; Kimberly Gilmour; Giovanna Lucchini; Sarah Inglott; William Mifsud; Robert Chiesa; Karl S. Peggs; Lucas Chan; Farzin Farzaneh; Adrian J. Thrasher; Ajay Vora; Martin Pule; Paul Veys

doi:10.1126/scitranslmed.aaj2013

ReportsCancer

Molecular remission of infant B-ALL after infusion of universal TALEN gene-edited CAR T cells

Waseem Qasim¹,²,*,
Hong Zhan¹,
Sujith Samarasinghe²,
Stuart Adams²,
Persis Amrolia¹,²,
Sian Stafford¹,
Katie Butler¹,
Christine Rivat¹,
Gary Wright²,
Kathy Somana²,
Sara Ghorashian¹,
Danielle Pinner²,
Gul Ahsan²,
Kimberly Gilmour²,
Giovanna Lucchini²,
Sarah Inglott²,
William Mifsud²,
Robert Chiesa²,
Karl S. Peggs³,
Lucas Chan⁴,
Farzin Farzaneh⁴,
Adrian J. Thrasher¹,
Ajay Vora⁵,
Martin Pule³ and
Paul Veys¹

¹Great Ormond Street Institute of Child Health, University College London, London WC1N 1EH, UK.
²Great Ormond Street Hospital National Health Service Trust, London WC1N 1LE, UK.
³Cancer Institute, University College London, London WC1E 6DD, UK.
⁴Division of Cancer Studies, Department of Haematological Medicine, King’s College London, London SE5 9NU, UK.
⁵Sheffield Children’s Hospital, Sheffield S10 2TH, UK.

↵*Corresponding author. Email: w.qasim{at}ucl.ac.uk

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Science Translational Medicine 25 Jan 2017:
Vol. 9, Issue 374, eaaj2013
DOI: 10.1126/scitranslmed.aaj2013

Waseem Qasim

1Great Ormond Street Institute of Child Health, University College London, London WC1N 1EH, UK.

2Great Ormond Street Hospital National Health Service Trust, London WC1N 1LE, UK.

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For correspondence: w.qasim@ucl.ac.uk

Hong Zhan

1Great Ormond Street Institute of Child Health, University College London, London WC1N 1EH, UK.

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Sujith Samarasinghe

2Great Ormond Street Hospital National Health Service Trust, London WC1N 1LE, UK.

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Stuart Adams

2Great Ormond Street Hospital National Health Service Trust, London WC1N 1LE, UK.

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Persis Amrolia

1Great Ormond Street Institute of Child Health, University College London, London WC1N 1EH, UK.

2Great Ormond Street Hospital National Health Service Trust, London WC1N 1LE, UK.

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Sian Stafford

1Great Ormond Street Institute of Child Health, University College London, London WC1N 1EH, UK.

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Katie Butler

1Great Ormond Street Institute of Child Health, University College London, London WC1N 1EH, UK.

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Christine Rivat

1Great Ormond Street Institute of Child Health, University College London, London WC1N 1EH, UK.

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Gary Wright

2Great Ormond Street Hospital National Health Service Trust, London WC1N 1LE, UK.

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Kathy Somana

2Great Ormond Street Hospital National Health Service Trust, London WC1N 1LE, UK.

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Sara Ghorashian

1Great Ormond Street Institute of Child Health, University College London, London WC1N 1EH, UK.

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Danielle Pinner

2Great Ormond Street Hospital National Health Service Trust, London WC1N 1LE, UK.

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Gul Ahsan

2Great Ormond Street Hospital National Health Service Trust, London WC1N 1LE, UK.

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Kimberly Gilmour

2Great Ormond Street Hospital National Health Service Trust, London WC1N 1LE, UK.

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Giovanna Lucchini

2Great Ormond Street Hospital National Health Service Trust, London WC1N 1LE, UK.

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Sarah Inglott

2Great Ormond Street Hospital National Health Service Trust, London WC1N 1LE, UK.

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William Mifsud

2Great Ormond Street Hospital National Health Service Trust, London WC1N 1LE, UK.

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Robert Chiesa

2Great Ormond Street Hospital National Health Service Trust, London WC1N 1LE, UK.

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Karl S. Peggs

3Cancer Institute, University College London, London WC1E 6DD, UK.

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Lucas Chan

4Division of Cancer Studies, Department of Haematological Medicine, King’s College London, London SE5 9NU, UK.

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Farzin Farzaneh

4Division of Cancer Studies, Department of Haematological Medicine, King’s College London, London SE5 9NU, UK.

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Adrian J. Thrasher

1Great Ormond Street Institute of Child Health, University College London, London WC1N 1EH, UK.

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Ajay Vora

5Sheffield Children’s Hospital, Sheffield S10 2TH, UK.

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Martin Pule

3Cancer Institute, University College London, London WC1E 6DD, UK.

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Paul Veys

1Great Ormond Street Institute of Child Health, University College London, London WC1N 1EH, UK.

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Molecular remission of infant B-ALL after infusion of universal TALEN gene-edited CAR T cells

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