Biffi Alessandra
Gene Therapy Program, Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Harvard Medical School, Smith 1158, 450 Brookline Avenue, Boston, MA 02115, USA.
Hematol Oncol Clin North Am. 2017 Oct;31(5):869-881. doi: 10.1016/j.hoc.2017.06.004. Epub 2017 Jul 23.
Increasingly, patients affected by metabolic diseases affecting the central nervous system and neuroinflammatory disorders receive hematopoietic cell transplantation (HCT) in the attempt to slow the course of their disease, delay or attenuate symptoms, and improve pathologic findings. The possible replacement of brain-resident myeloid cells by the transplanted cell progeny contributes to clinical benefit. Genetic engineering of the cells to be transplanted (hematopoietic stem cell) may endow the brain myeloid progeny of these cells with enhanced or novel functions, contributing to therapeutic effects.
越来越多患有影响中枢神经系统的代谢性疾病和神经炎症性疾病的患者接受造血细胞移植（HCT），以期减缓疾病进程、延迟或减轻症状并改善病理表现。移植细胞后代可能替代脑内常驻髓样细胞，这有助于临床获益。对拟移植细胞（造血干细胞）进行基因工程改造，可能使这些细胞的脑髓样后代具备增强的或新的功能，从而产生治疗效果。
