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Nat Biotechnol. 2015 Jun;33(6):584-6. doi: 10.1038/nbt.3241. Epub 2015 May 18.

In vivo genome editing using nuclease-encoding mRNA corrects SP-B deficiency.

Author information

1
Department of Pediatrics I-Pediatric Infectiology and Immunology, Translational Genomics and Gene Therapy, University of Tübingen, Tübingen, Germany.
2
1] Department of Pharmacology and Experimental Therapy, University of Tübingen, Tübingen, Germany. [2] ICePha, University of Tübingen, Tübingen, Germany.
3
Helmholtz Institute for Pharmaceutical Research Saarland (HIPS), Helmholtz Center for Infection Research (HZI), Saarbrücken, Germany.
4
1] Department of Pediatric Pulmonology, Dr. von Hauner Children's Hospital, Ludwig Maximilians University, Munich, Germany. [2] Member of the German Center for Lung Research (DZL), Munich, Germany.
5
1] ICePha, University of Tübingen, Tübingen, Germany. [2] Department of Clinical Pharmacology, University of Tübingen, Tübingen, Germany. [3] Dr. Margarete Fischer-Bosch Institute of Clinical Pharmacology, Stuttgart, Germany.
6
Department of Pharmacology and Experimental Therapy, University of Tübingen, Tübingen, Germany.
7
1] Helmholtz Institute for Pharmaceutical Research Saarland (HIPS), Helmholtz Center for Infection Research (HZI), Saarbrücken, Germany. [2] Department of Pharmacy, Saarland University, Saarbrücken, Germany.
PMID:
25985262
DOI:
10.1038/nbt.3241
[Indexed for MEDLINE]

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