Preclinical modeling highlights the therapeutic potential of hematopoietic stem cell gene editing for correction of SCID-X1. (Q45873054)

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6 April 2020

title

Preclinical modeling highlights the therapeutic potential of hematopoietic stem cell gene editing for correction of SCID-X1 (English)

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Angelo Leone Lombardo

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Giulia Schiroli

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Samuele Ferrari

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Aurelien Jacob

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Valentina Capo

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Tiziana Plati

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Maria Carmina Castiello

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Rahul Palchaudhuri

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David T Scadden

13

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Giovanni Sitia

16

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Pietro Genovese

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Luigi Naldini

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author name string

Anthony Conway

3

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Luisa Albano

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Francesca Sanvito

9

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Andrew R Gennery

10

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Chiara Bovolenta

11

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Michael C Holmes

14

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language of work or name

English

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publication date

1 October 2017

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Science Translational Medicine

6 April 2020

published in

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volume

9

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issue

411

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Genome editing. The new frontier of genome engineering with CRISPR-Cas9

6 April 2020

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21 January 2018

Identifiers

DOI

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6 April 2020

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