title: In vivo genome editing using nuclease-encoding mRNA corrects SP-B deficiency.
authors.affiliation: Department of Pediatrics I-Pediatric Infectiology and Immunology, Translational Genomics and Gene Therapy, University of Tübingen, Tübingen, Germany.
authors.forename: Azita J
authors.initials: AJ
authors.lastname: Mahiny
authors.affiliation: Department of Pediatrics I-Pediatric Infectiology and Immunology, Translational Genomics and Gene Therapy, University of Tübingen, Tübingen, Germany.
authors.forename: Alexander
authors.initials: A
authors.lastname: Dewerth
authors.affiliation: Department of Pediatrics I-Pediatric Infectiology and Immunology, Translational Genomics and Gene Therapy, University of Tübingen, Tübingen, Germany.
authors.forename: Lauren E
authors.initials: LE
authors.lastname: Mays
authors.affiliation: Department of Pediatrics I-Pediatric Infectiology and Immunology, Translational Genomics and Gene Therapy, University of Tübingen, Tübingen, Germany.
authors.forename: Mohammed
authors.initials: M
authors.lastname: Alkhaled
authors.affiliation: 1] Department of Pharmacology and Experimental Therapy, University of Tübingen, Tübingen, Germany. [2] ICePha, University of Tübingen, Tübingen, Germany.
authors.forename: Benedikt
authors.initials: B
authors.lastname: Mothes
authors.affiliation: Helmholtz Institute for Pharmaceutical Research Saarland (HIPS), Helmholtz Center for Infection Research (HZI), Saarbrücken, Germany.
authors.forename: Emad
authors.initials: E
authors.lastname: Malaeksefat
authors.affiliation: Helmholtz Institute for Pharmaceutical Research Saarland (HIPS), Helmholtz Center for Infection Research (HZI), Saarbrücken, Germany.
authors.forename: Brigitta
authors.initials: B
authors.lastname: Loretz
authors.affiliation: Department of Pediatrics I-Pediatric Infectiology and Immunology, Translational Genomics and Gene Therapy, University of Tübingen, Tübingen, Germany.
authors.forename: Jennifer
authors.initials: J
authors.lastname: Rottenberger
authors.affiliation: Department of Pediatrics I-Pediatric Infectiology and Immunology, Translational Genomics and Gene Therapy, University of Tübingen, Tübingen, Germany.
authors.forename: Darina M
authors.initials: DM
authors.lastname: Brosch
authors.affiliation: Department of Pediatrics I-Pediatric Infectiology and Immunology, Translational Genomics and Gene Therapy, University of Tübingen, Tübingen, Germany.
authors.forename: Philipp
authors.initials: P
authors.lastname: Reautschnig
authors.affiliation: Department of Pediatrics I-Pediatric Infectiology and Immunology, Translational Genomics and Gene Therapy, University of Tübingen, Tübingen, Germany.
authors.forename: Pacharapan
authors.initials: P
authors.lastname: Surapolchai
authors.affiliation: Department of Pediatrics I-Pediatric Infectiology and Immunology, Translational Genomics and Gene Therapy, University of Tübingen, Tübingen, Germany.
authors.forename: Franziska
authors.initials: F
authors.lastname: Zeyer
authors.affiliation: 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.
authors.forename: Andrea
authors.initials: A
authors.lastname: Schams
authors.affiliation: Department of Pediatrics I-Pediatric Infectiology and Immunology, Translational Genomics and Gene Therapy, University of Tübingen, Tübingen, Germany.
authors.forename: Melanie
authors.initials: M
authors.lastname: Carevic
authors.affiliation: Department of Pediatrics I-Pediatric Infectiology and Immunology, Translational Genomics and Gene Therapy, University of Tübingen, Tübingen, Germany.
authors.forename: Martina
authors.initials: M
authors.lastname: Bakele
authors.affiliation: 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.
authors.forename: Matthias
authors.initials: M
authors.lastname: Griese
authors.affiliation: 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.
authors.forename: Matthias
authors.initials: M
authors.lastname: Schwab
authors.affiliation: Department of Pharmacology and Experimental Therapy, University of Tübingen, Tübingen, Germany.
authors.forename: Bernd
authors.initials: B
authors.lastname: Nürnberg
authors.affiliation: Department of Pharmacology and Experimental Therapy, University of Tübingen, Tübingen, Germany.
authors.forename: Sandra
authors.initials: S
authors.lastname: Beer-Hammer
authors.affiliation: Department of Pediatrics I-Pediatric Infectiology and Immunology, Translational Genomics and Gene Therapy, University of Tübingen, Tübingen, Germany.
authors.forename: Rupert
authors.initials: R
authors.lastname: Handgretinger
authors.affiliation: Department of Pediatrics I-Pediatric Infectiology and Immunology, Translational Genomics and Gene Therapy, University of Tübingen, Tübingen, Germany.
authors.forename: Dominik
authors.initials: D
authors.lastname: Hartl
authors.affiliation: 1] Helmholtz Institute for Pharmaceutical Research Saarland (HIPS), Helmholtz Center for Infection Research (HZI), Saarbrücken, Germany. [2] Department of Pharmacy, Saarland University, Saarbrücken, Germany.
authors.forename: Claus-Michael
authors.initials: CM
authors.lastname: Lehr
authors.affiliation: Department of Pediatrics I-Pediatric Infectiology and Immunology, Translational Genomics and Gene Therapy, University of Tübingen, Tübingen, Germany.
authors.forename: Michael S D
authors.initials: MS
authors.lastname: Kormann
mesh_terms: D000818:Animals; D006801:Humans; D051379:Mice; D001249:Asthma / Q000235:genetics / Q000628:therapy; D048271:Chitosan / Q000627:therapeutic use; D004195:Disease Models, Animal; D008822:Mice, Transgenic; D053758:Nanoparticles / Q000627:therapeutic use; D011649:Pulmonary Alveolar Proteinosis / Q000151:congenital / Q000235:genetics / Q000628:therapy; D037701:Pulmonary Surfactant-Associated Protein B / Q000172:deficiency / Q000235:genetics; D017393:RNA Editing / Q000235:genetics; D012333:RNA, Messenger / Q000235:genetics / Q000627:therapeutic use; D017563:Lung Diseases, Interstitial
chemical_list: D048271:Chitosan; D037701:Pulmonary Surfactant-Associated Protein B; D012333:RNA, Messenger