Kaye E M
Massachusetts General Hospital, Boston, Massachusetts, 02111, USA.
Curr Opin Pediatr. 1995 Dec;7(6):650-4. doi: 10.1097/00008480-199512000-00004.
Nascent therapies for the lysosomal storage diseases have begun. The replacement enzyme therapy for Gaucher's disease now includes a recombinant form, and effective dosing schedules are being developed. Bone marrow transplantation appears to be a very successful treatment for nonneuronopathic Gaucher's disease and halts the progression of other lysosomal storage disorders. Following the success of bone marrow transplantation, gene therapy trials using transduced human hematopoietic cells are beginning in Gaucher's disease, which should lead to autologous bone marrow transplantation using genetically engineered cells. Experimental studies hold promise for neurologic treatment in the lysosomal storage diseases using transplanted recombinant cells and neural progenitor cells.
针对溶酶体贮积病的新型疗法已经展开。戈谢病的替代酶疗法如今包括一种重组形式，并且正在制定有效的给药方案。骨髓移植似乎是治疗非神经病变型戈谢病的一种非常成功的方法，并且能阻止其他溶酶体贮积症的进展。继骨髓移植取得成功之后，利用转导的人类造血细胞进行的基因治疗试验已在戈谢病中展开，这有望实现使用基因工程细胞进行自体骨髓移植。实验研究有望利用移植的重组细胞和神经祖细胞对溶酶体贮积病进行神经学治疗。
