Eman M. Elsabbagh: Pediatric Nonmalignant HCT Enters the Era of Precision Conditioning and Gene Therapy
Eman M. Elsabbagh, Physician Scientist, Director at CITADEL Lab, shared a post on LinkedIn about a recent article she and her colleagues co-authored, published in Slack Journals, adding:
“Pediatric nonmalignant HCT is moving into a precision era. The goal is no longer simply to achieve engraftment, but to maximize cure while minimizing toxicity and long term burden.
In our review, ‘Pediatric Nonmalignant Hematopoietic Cell Transplantation in the Era of Precision Conditioning and Gene Therapy,’ we discuss how the field is evolving through:
- disease adapted and reduced toxicity conditioning
- pharmacokinetic guided approaches
- broader donor options
- earlier referral and improved patient selection
- the expanding role of gene therapy as an alternative curative strategy
The central question is increasingly not just whether to transplant, but which curative platform is best, for which child, and at what time.
Published in the September 2026 special issue of Pediatric Annals.”
Title: Pediatric Nonmalignant Hematopoietic Cell Transplantation in the Era of Precision Conditioning and Gene Therapy
Authors: Eman M. Elsabbagh, Ahmed A. Shawky, Christopher C. Dvorak
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