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Updated: Aug 15, 2026

Intrafemoral Injection of Human Hematopoietic Stem and Progenitor Cells into Immunocompromised Mice
Published on: December 8, 2023
Successful hematopoietic stem cell transplantation for Niemann-Pick disease type B
Ami J Shah1, Neena Kapoor, Gay M Crooks
1Division of Research Immunology/Bone Marrow Transplantation, Childrens Hospital Los Angeles, CA 90027, USA. ashah@chla.usc.edu
Abstract:
Histocompatible hematopoietic stem cell transplantation (HSCT) was conducted on a 4.5-year-old girl with Niemann-Pick disease type B. The donor was her unaffected brother. At the time of transplantation, she had severe pulmonary disease. After her first HSCT, she developed graft failure. Five years after her second HSCT, her sphingomyelinase levels are within normal levels, she has no pulmonary symptoms, and aside from persistent graft versus host disease, she is doing well.
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