Research ArticleGENE EDITING
In utero gene editing for monogenic lung disease
- View ORCID ProfileDeepthi Alapati1,2,3,4,5,
- View ORCID ProfileWilliam J. Zacharias3,4,5,6,
- View ORCID ProfileHeather A. Hartman7,
- View ORCID ProfileAvery C. Rossidis7,
- View ORCID ProfileJohn D. Stratigis7,
- View ORCID ProfileNicholas J Ahn7,
- View ORCID ProfileBarbara Coons7,
- Su Zhou3,5,
- View ORCID ProfileHiaying Li7,
- View ORCID ProfileKshitiz Singh7,
- Jeremy Katzen3,4,
- Yaniv Tomer3,4,
- View ORCID ProfileAlexandra C. Chadwick3,5,
- Kiran Musunuru3,5,
- View ORCID ProfileMichael F. Beers3,4,
- View ORCID ProfileEdward E. Morrisey3,4,5,* and
- View ORCID ProfileWilliam H. Peranteau7,*
- 1Department of Pediatrics Nemours, Alfred I duPont Hospital for Children, Wilmington, DE 19803, USA.
- 2Department of Pediatrics Sidney Kimmel Medical College, Thomas Jefferson University Philadelphia, Philadelphia, PA 19107, USA.
- 3Department of Medicine, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA 19104, USA.
- 4Penn Center for Pulmonary Biology, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA 19104, USA.
- 5Penn Cardiovascular Institute, Perelman School of Medicine at the University of Pennsylvania Philadelphia, Philadelphia, PA 19104, USA.
- 6Division of Pulmonary Biology Cincinnati Children’s Hospital, Department of Pediatrics University of Cincinnati College of Medicine, Cincinnati, OH 45529, USA.
- 7Center for Fetal Research, Division of General, Thoracic, and Fetal Surgery, Children’s Hospital of Philadelphia, Philadelphia, PA 19104, USA.
- ↵*Corresponding author. Email: emorrise{at}pennmedicine.upenn.edu (E.E.M.); peranteauw{at}email.chop.edu (W.H.P.)
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Science Translational Medicine 17 Apr 2019:
Vol. 11, Issue 488, eaav8375
DOI: 10.1126/scitranslmed.aav8375
Vol. 11, Issue 488, eaav8375
DOI: 10.1126/scitranslmed.aav8375
Deepthi Alapati
1Department of Pediatrics Nemours, Alfred I duPont Hospital for Children, Wilmington, DE 19803, USA.
2Department of Pediatrics Sidney Kimmel Medical College, Thomas Jefferson University Philadelphia, Philadelphia, PA 19107, USA.
3Department of Medicine, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA 19104, USA.
4Penn Center for Pulmonary Biology, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA 19104, USA.
5Penn Cardiovascular Institute, Perelman School of Medicine at the University of Pennsylvania Philadelphia, Philadelphia, PA 19104, USA.
William J. Zacharias
3Department of Medicine, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA 19104, USA.
4Penn Center for Pulmonary Biology, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA 19104, USA.
5Penn Cardiovascular Institute, Perelman School of Medicine at the University of Pennsylvania Philadelphia, Philadelphia, PA 19104, USA.
6Division of Pulmonary Biology Cincinnati Children’s Hospital, Department of Pediatrics University of Cincinnati College of Medicine, Cincinnati, OH 45529, USA.
Heather A. Hartman
7Center for Fetal Research, Division of General, Thoracic, and Fetal Surgery, Children’s Hospital of Philadelphia, Philadelphia, PA 19104, USA.
Avery C. Rossidis
7Center for Fetal Research, Division of General, Thoracic, and Fetal Surgery, Children’s Hospital of Philadelphia, Philadelphia, PA 19104, USA.
John D. Stratigis
7Center for Fetal Research, Division of General, Thoracic, and Fetal Surgery, Children’s Hospital of Philadelphia, Philadelphia, PA 19104, USA.
Nicholas J Ahn
7Center for Fetal Research, Division of General, Thoracic, and Fetal Surgery, Children’s Hospital of Philadelphia, Philadelphia, PA 19104, USA.
Barbara Coons
7Center for Fetal Research, Division of General, Thoracic, and Fetal Surgery, Children’s Hospital of Philadelphia, Philadelphia, PA 19104, USA.
Su Zhou
3Department of Medicine, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA 19104, USA.
5Penn Cardiovascular Institute, Perelman School of Medicine at the University of Pennsylvania Philadelphia, Philadelphia, PA 19104, USA.
Hiaying Li
7Center for Fetal Research, Division of General, Thoracic, and Fetal Surgery, Children’s Hospital of Philadelphia, Philadelphia, PA 19104, USA.
Kshitiz Singh
7Center for Fetal Research, Division of General, Thoracic, and Fetal Surgery, Children’s Hospital of Philadelphia, Philadelphia, PA 19104, USA.
Jeremy Katzen
3Department of Medicine, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA 19104, USA.
4Penn Center for Pulmonary Biology, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA 19104, USA.
Yaniv Tomer
3Department of Medicine, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA 19104, USA.
4Penn Center for Pulmonary Biology, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA 19104, USA.
Alexandra C. Chadwick
3Department of Medicine, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA 19104, USA.
5Penn Cardiovascular Institute, Perelman School of Medicine at the University of Pennsylvania Philadelphia, Philadelphia, PA 19104, USA.
Kiran Musunuru
3Department of Medicine, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA 19104, USA.
5Penn Cardiovascular Institute, Perelman School of Medicine at the University of Pennsylvania Philadelphia, Philadelphia, PA 19104, USA.
Michael F. Beers
3Department of Medicine, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA 19104, USA.
4Penn Center for Pulmonary Biology, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA 19104, USA.
Edward E. Morrisey
3Department of Medicine, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA 19104, USA.
4Penn Center for Pulmonary Biology, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA 19104, USA.
5Penn Cardiovascular Institute, Perelman School of Medicine at the University of Pennsylvania Philadelphia, Philadelphia, PA 19104, USA.
William H. Peranteau
7Center for Fetal Research, Division of General, Thoracic, and Fetal Surgery, Children’s Hospital of Philadelphia, Philadelphia, PA 19104, USA.
