BONE-MARROW TRANSPLANTATION IN BATTEN-DISEASE (NEURONAL CEROID-LIPOFUSCINOSIS) - WILL IT WORK - PRELIMINARY STUDIES ON COCULTURE EXPERIMENTS AND ON BONE-MARROW TRANSPLANT IN LATE INFANTILE BATTEN-DISEASE
BONE-MARROW TRANSPLANTATION IN BATTEN-DISEASE (NEURONAL CEROID-LIPOFUSCINOSIS) - WILL IT WORK - PRELIMINARY STUDIES ON COCULTURE EXPERIMENTS AND ON BONE-MARROW TRANSPLANT IN LATE INFANTILE BATTEN-DISEASE
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DOI：
10.1002/ajmg.1320570253
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发表时间：
1995-06-05
期刊：
影响因子：
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通讯作者：
VELLODI, A
中科院分区：
文献类型：
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作者：
LAKE, BD;HENDERSON, DC;VELLODI, A
淋巴细胞从临床前晚期婴儿巴滕氏病患者单独培养，并与淋巴细胞从捐助者，和命运的曲线夹杂物的疾病特征进行了监测，通过电子显微镜。没有证据表明可能引起储存物质去除的缺陷酶或因子的转移，并且曲线轮廓保留在培养的细胞中而没有降解的迹象。用植物血凝素刺激分裂的细胞在培养物中没有表现出储存，这表明储存是细胞年龄的函数，患者在27/12岁时接受了骨髓移植，但临床上仍未受影响，在9个月的时间内，通过电子显微镜监测对皮肤和直肠活检中淋巴细胞和细胞的影响，直到供体骨髓被他有一次癫痫发作，现在有晚期婴儿巴滕氏病的神经生理学证据，骨髓移植可能对已经储存的物质没有影响，但可能防止进一步积累并阻止临床症状的发作，尽管最近对患有巴滕氏病的绵羊进行早期（胎儿）移植的研究没有显示出任何益处。(C)1995 Wiley-Liss，Inc.
Lymphocytes from a patient with preclinical late infantile Batten disease were cultured alone and;with lymphocytes from donors, and the fate of the curvilinear inclusions characteristic of the disease was monitored by electron microscopy. There was no evidence of transfer of deficient enzyme or factor that might have caused removal of the stored material, and the curvilinear profiles remained in the cultured cells without signs of degradation, Cells stimulated to divide with phytohaemaglutinin did not exhibit storage in culture suggesting that storage is a function of the age of the cell, The patient received a bone marrow transplant at 2 7/12 years while still clinically unaffected, and the effect on lymphocytes and cells in skin and rectal biopsies was monitored by electron microscopy over a period of 9 months until the donor marrow became displaced by the host cells, He has had one seizure and now has neurophysiological evidence of late infantile Batten's disease, Bone marrow transplant may have no effect on material already stored but might prevent further build-up and halt the onset of the clinical symptoms although very recent studies on early (fetal) transplants in sheep with a form of Batten disease have shown no benefit. (C) 1995 Wiley-Liss,Inc.