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

作者信息

Mahiny Azita J, Dewerth Alexander, Mays Lauren E, Alkhaled Mohammed, Mothes Benedikt, Malaeksefat Emad, Loretz Brigitta, Rottenberger Jennifer, Brosch Darina M, Reautschnig Philipp, Surapolchai Pacharapan, Zeyer Franziska, Schams Andrea, Carevic Melanie, Bakele Martina, Griese Matthias, Schwab Matthias, Nürnberg Bernd, Beer-Hammer Sandra, Handgretinger Rupert, Hartl Dominik, Lehr Claus-Michael, Kormann Michael S D

机构信息

Department of Pediatrics I-Pediatric Infectiology and Immunology, Translational Genomics and Gene Therapy, University of Tübingen, Tübingen, Germany.

1] Department of Pharmacology and Experimental Therapy, University of Tübingen, Tübingen, Germany. [2] ICePha, University of Tübingen, Tübingen, Germany.

出版信息

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

DOI:10.1038/nbt.3241
PMID:25985262
Abstract
摘要

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