Successful correction of hemophilia by CRISPR/Cas9 genome editing in vivo : delivery vector and immune responses are the key to success

Tuan Nguyen; Ignacio Anegon

doi:10.15252/emmm.201606325

Journal articles

Successful correction of hemophilia by CRISPR/Cas9 genome editing in vivo : delivery vector and immune responses are the key to success

Tuan Nguyen ^{1, 2, 3} Ignacio Anegon ^{1, 2, 3, *}

* Corresponding author

1 U1064 Inserm - CRTI - Centre de Recherche en Transplantation et Immunologie

2 ITUN - Institut de transplantation urologie-néphrologie

3 Faculté de Médecine - Université de Nantes

Abstract : Comment on CRISPR/Cas9-mediated somatic correction of a novel coagulator factor IX gene mutation ameliorates hemophilia in mouse. [EMBO Mol Med. 2016]

Domain :

Life Sciences [q-bio] / Human health and pathology

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https://www.hal.inserm.fr/inserm-02150381
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Submitted on : Friday, June 7, 2019 - 11:30:53 AM
Last modification on : Saturday, June 8, 2019 - 1:25:37 AM

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Successful correction of hemophilia by CRISPR/Cas9 genome editing in vivo : delivery vector and immune responses are the key to success

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