William H. Peranteau

faculty photo
Associate Professor of Surgery at the Children's Hospital of Philadelphia
Active Surgeon, Pennsylvania Hospital, Philadelphia, PA
Active Surgeon, Children's Hospital of Philadelphia, Philadelphia, PA
Attending Surgeon, Hospital of the University of Pennsylvania, Philadelphia, PA
Member, Institute for Translational Medicine and Therapeutics (ITMAT), Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA
Active Physician, Virtua Hospital, Voorhees, NJ
Adzick-McCausland Distinguished Chair in Fetal and Pediatric Surgery, Children's Hospital of Philadelphia, Philadelphia, PA (Inaugural chair holder)
Co-Director, The Center for Fetal Research, Children's Hospital of Philadelphia, Philadelphia, PA
Department: Surgery
Graduate Group Affiliations

Contact information
Division of Pediatric General, Thoracic & Fetal Surgery
The Children's Hospital of Philadelphia
Center for Fetal Research
3615 Civic Center Blvd
Abramson Research Center, Rm 1116E
Philadelphia, PA 19104
Office: 215-590-2730
Fax: 215-590-4875
Lab: 215-590-4810
Education:
BA (molecular biology)
Princeton University, Princeton, NJ, 1997.
MD
University of Pennsylvania School of Medicine, Philadelphia, PA, 2002.
Post-Graduate Training
Intern in General Surgery, Brigham & Women's Hospital, Boston, MA, 2002-2003.
Resident in General Surgery, Brigham & Women's Hospital, Boston, MA, 2003-2010.
Research Fellow in Surgery, Children's Institute for Surgical Science, The Children's Hospital of Philadelphia, Philadelphia, PA, 2004-2007.
Fellow in Pediatric Surgery, The Children's Hospital of Philadelphia, Philadelphia, PA, 2010-2012.
Apprentice in Fetal Surgery, Children's Hospital of Philadelphia, Philadelphia, PA, 2012-2013.
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Description of Itmat Expertise

My interest primarily involves the study of in utero hematopoietic cell transplantation and in utero gene therapy/editing as potential therapeutic avenues for many congenital hematologic and genetic disorders such as Sickle cell disease, Tyrosinemia, and Friedreich's ataxia. We are currently investigating mechanisms to optimize the success and safety of these techniques in the murine and preclinical canine model prior to translation into clinical utility.

Description of Research Expertise

I have an interest in fetal development as it relates to the potential understanding of and fetal intervention for congenital anomalies. Specifically, I have had a deep interest and significant experience evaluating in utero hematopoietic stem cell transplantation in the murine and canine model. These studies provide interesting information and address basic questions on the development of the fetal hematopoietic niche. One of the main goals of my research is a better understanding of the barriers to successful allogeneic in utero bone marrow transplantation. Allogeneic in utero bone marrow transplantation would allow for nonmyeloablative unmatched transplants without the need for immunosuppression and thus holds significant clinical promise for the treatment of hemoglobinopathies, such as Sickle Cell disease.

Selected Publications

Baeza J, Coons BE, Lin Z, Riley J, Mendoza M, Peranteau WH, Garcia BA: In utero pulse injection of isotopic amino acids quantifies protein turnover rates during murine fetal development. bioRxiv May 21 2023 (Preprint) Notes: doi: 10.1101/2023.05.18.541242.

Waddington SN, Peranteau WH, Rahim AA, Boyle AK, Kurian MA, Gissen P, Chan JKY, David AL: Fetal gene therapy. Journal of Inherited Metabolic Disease July 20 2023 (Epub) Notes: doi: 10.1002/jimd.12659.

Riley JS, Schomberg J, Mantha A, Guner Y, Cuevas A, Stephenson CD, Peranteau WH, Yu PT: A novel VACTERL assessment tool to facilitate counseling for expectant families. Fetal Diagnosis & Therapy June 20 2023 (Epub) Notes: doi: 10.1159/000531611.

Palanki R, Bose SK, Dave A, White BM, Berkowitz C, Luks V, Yaqoob F, Han E, Swingle KL, Menon P, Hodgson E, Biswas A, Billingsley MM, Li L, Yiping F, Carpenter M, Trokhan A, Yeo J, Johana N, Wan TY, Alameh MG, Bennett FC, Storm PB, Jain R, Chan J, Weissman D, Mitchell MJ, Peranteau WH: Ionizable lipid nanoparticles for therapeutic base editing of congenital brain disease. ACS Nano 17(14): 13594-13610, July 25 2023 Notes: Epub July 17, 2023.

Brooks DL, Carrasco MJ, Qu P, Peranteau WH, Ahrens-Nicklas RC, Musunuru K, Alameh MG, Wang X: Rapid and definitive treatment of phenylketonuria in variant-humanized mice with corrective editing. Nature Communications 14(1): 3451, June 10 2023.

Dumitru AM, Berkowitz C, Whittaker M, Wang X, Kus N, Luks V, Riley J, Carpenter M, Archer NF, Brooks D, Johnston W, Alameh MG, Musunuru K, Peranteau W: Optimizing therapeutic base editing for hereditary tyrosinemia type 1. Molecular Therapy 31(4 Suppl 1): 83-84, May 1 2023 Notes: Presented at the 26th Annual Meeting of the American Society of Gene & Cell Therapy, Los Angeles, CA, May 2023.

Luks VL, Dumitru AM, White B, Palanki R, Dave A, Alameh MG, Beers MF, Jeremy K, Peranteau WH: Cytosine base editing of surfactant protein C mutation restores genotype and phenotype in human and murine models. Molecular Therapy 31(4 Suppl 1): 266-267, May 1 2023 Notes: Presented at the 26th Annual Meeting of the American Society of Gene & Cell Therapy, Los Angeles, CA, May 2023.

Riley JS, Luks VL, Berkowitz CL, Dave A, Zoltick PW, Peranteau WH: Pre-existing maternal immunity to adeno-associated virus causes fetal mortality after in utero gene editing mediated by maternal interleukin 6 and CXCL1. Molecular Therapy 31(4 Suppl 1): 76-77, May 1 2023 Notes: Presented at the 26th Annual Meeting of the American Society of Gene & Cell Therapy, Los Angeles, CA, May 2023.

Luks VL, Dave A, Berkowitz CL, Riley JS, Carpenter M, Zoltick PW, Peranteau W: In utero pulmonary gene editing persists after postnatal lung injury. Molecular Therapy 31(4 Suppl 1): 652-653, May 1 2023 Notes: Presented at the 26th Annual Meeting of the American Society of Gene & Cell Therapy, Los Angeles, CA, May 2023.

Palanki R, Bose S, Dave A, Luks VL, Mitchell M, Peranteau W: Ionizable lipid nanoparticles for therapeutic base editing of congenital brain disease. Molecular Therapy 31(4 Suppl 1): 20-21, May 1 2023 Notes: Presented at the 26th Annual Meeting of the American Society of Gene & Cell Therapy, Los Angeles, CA, May 2023.

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Last updated: 08/18/2023
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