In utero gene editing for monogenic lung disease

Deepthi Alapati; William J. Zacharias; Heather A. Hartman; Avery C. Rossidis; John D. Stratigis; Nicholas J Ahn; Barbara Coons; Su Zhou; Hiaying Li; Kshitiz Singh; Jeremy Katzen; Yaniv Tomer; Alexandra C. Chadwick; Kiran Musunuru; Michael F. Beers; Edward E. Morrisey; William H. Peranteau

doi:10.1126/scitranslmed.aav8375

Research ArticleGENE EDITING

In utero gene editing for monogenic lung disease

Deepthi Alapati¹,²,³,⁴,⁵,
William J. Zacharias ³,⁴,⁵,⁶,
Heather A. Hartman⁷,
Avery C. Rossidis⁷,
John D. Stratigis⁷,
Nicholas J Ahn⁷,
Barbara Coons ⁷,
Su Zhou³,⁵,
Hiaying Li⁷,
Kshitiz Singh⁷,
Jeremy Katzen ³,⁴,
Yaniv Tomer³,⁴,
Alexandra C. Chadwick ³,⁵,
Kiran Musunuru ³,⁵,
Michael F. Beers ³,⁴,
Edward E. Morrisey ³,⁴,⁵,* and
William H. Peranteau ⁷,*

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

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.

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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.

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Heather A. Hartman

7Center for Fetal Research, Division of General, Thoracic, and Fetal Surgery, Children’s Hospital of Philadelphia, Philadelphia, PA 19104, USA.

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Avery C. Rossidis

7Center for Fetal Research, Division of General, Thoracic, and Fetal Surgery, Children’s Hospital of Philadelphia, Philadelphia, PA 19104, USA.

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John D. Stratigis

7Center for Fetal Research, Division of General, Thoracic, and Fetal Surgery, Children’s Hospital of Philadelphia, Philadelphia, PA 19104, USA.

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Nicholas J Ahn

7Center for Fetal Research, Division of General, Thoracic, and Fetal Surgery, Children’s Hospital of Philadelphia, Philadelphia, PA 19104, USA.

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Barbara Coons

7Center for Fetal Research, Division of General, Thoracic, and Fetal Surgery, Children’s Hospital of Philadelphia, Philadelphia, PA 19104, USA.

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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.

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Hiaying Li

7Center for Fetal Research, Division of General, Thoracic, and Fetal Surgery, Children’s Hospital of Philadelphia, Philadelphia, PA 19104, USA.

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Kshitiz Singh

7Center for Fetal Research, Division of General, Thoracic, and Fetal Surgery, Children’s Hospital of Philadelphia, Philadelphia, PA 19104, USA.

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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.

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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.

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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.

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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.

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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.

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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.

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For correspondence: emorrise@pennmedicine.upenn.edu peranteauw@email.chop.edu

William H. Peranteau

7Center for Fetal Research, Division of General, Thoracic, and Fetal Surgery, Children’s Hospital of Philadelphia, Philadelphia, PA 19104, USA.

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ORCID record for William H. Peranteau
For correspondence: emorrise@pennmedicine.upenn.edu peranteauw@email.chop.edu

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