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Office
300 Longwood Ave
Karp 8
Boston, MA 02115Phone+1 617-919-2697Fax+1 617-730-0934- Is this information wrong?
Summary
- Chief Scientific Officer and SVP for Research; Chief of Hematology/Oncology, Boston Children's Hospital
Education & Training
- Boston Children’s Hospital/Boston Medical CenterFellowship, Pediatric Hematology/Oncology, 1982 - 1987
- Cincinnati Children's Hospital Medical CenterResidency, Pediatrics, 1979 - 1982
- Indiana University School of MedicineClass of 1979
Certifications & Licensure
- MA State Medical License 1982 - 2025
- OH State Medical License 1980 - 2009
- IN State Medical License 1979 - 2003
- American Board of Pediatrics Pediatrics
Awards, Honors, & Recognition
- Elected Member Institute of Medicine, 2002
- Elected Member The American Society for Clinical Investigation, 1993
- Fellow (FACC) American College of Cardiology
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Clinical Trials
- Multicenter Phase II Trial-Hematopoietic Stem Cell Transplantation for Treatment of Patients With FA Lacking a Genotypically Identical Donor, Using a Chemotherapy Only Cytoreduction With Busulfan, Cyclophosphamide and Fludarabine Start of enrollment: 2009 Apr 01
- Gene Transfer for Severe Combined Immunodeficiency, X-linked (SCID-X1) Using a Self-inactivating (SIN) Gammaretroviral Vector Start of enrollment: 2010 Apr 01
- Pilot and Feasibility Study of Hematopoietic Stem Cell Gene Transfer for the Wiskott-Aldrich Syndrome Start of enrollment: 2011 Jul 01
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Publications & Presentations
PubMed
- 74 citationsMechanisms underlying genetic susceptibility to multisystem inflammatory syndrome in children (MIS-C).Janet Chou, Craig D. Platt, Saddiq Habiballah, Alan A. Nguyen, Megan Elkins
The Journal of Allergy and Clinical Immunology. 2021-09-01 - 183 citationsPost-Transcriptional Genetic Silencing of BCL11A to Treat Sickle Cell DiseaseErica B. Esrick, Leslie Lehmann, Alessandra Biffi, Maureen Achebe, Christian Brendel
The New England Journal of Medicine. 2021-01-21 - 8 citationsDevelopment of a double shmiR lentivirus effectively targeting both BCL11A and ZNF410 for enhanced induction of fetal hemoglobin to treat β-hemoglobinopathies.Boya Liu, Christian Brendel, Divya S Vinjamur, Yu Zhou, Chad Harris
Molecular Therapy. 2022-08-03
Abstracts/Posters
- Investigation of Cell-Contact Dependent Alterations in Immune Synapse Formation between T Cells and BCP-ALL CellsDavid A Williams, 61st Annual American Society of Hematology Meeting, Orlando, FL, 12/8/2019
- Outcome of Hematopoietic Stem Cell Gene Therapy for Wiskott-Aldrich SyndromeDavid A Williams, 61st Annual American Society of Hematology Meeting, Orlando, FL, 12/9/2019
Press Mentions
- Gene Therapy Offers Huge Potential for Combating Disease, Doctors SayMay 31st, 2024
Hospital Affiliations
- Dana-Farber Cancer InstituteBoston, Massachusetts
- Boston Children's HospitalBoston, Massachusetts
- Brigham and Women's HospitalBoston, Massachusetts
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