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羊膜组织移植作为某些溶酶体贮积病的一种治疗试验。

Amniotic tissue transplantation as a trial of treatment in some lysosomal storage diseases.

作者信息

Tylki-Szymańska A, Maciejko D, Kidawa M, Jabłońska-Budaj U, Czartoryska B

出版信息

J Inherit Metab Dis. 1985;8(3):101-4. doi: 10.1007/BF01819289.

DOI:10.1007/BF01819289
PMID:3939584
Abstract

This communication reports the clinical and biochemical results in six patients: four with mucopolysaccharidosis, one with GM1 gangliosidosis (Morquio B) and one with I-cell disease, who were treated by amniotic tissue transplantation. The sole evident clinical result was the diminishing of corneal clouding in three cases. A slight increase of beta-galactosidase activity in one patient's plasma was observed. The time of improvement was about 2 months after the transplantation and was transitory.

摘要

本通讯报道了6例接受羊膜组织移植治疗的患者的临床和生化结果:4例黏多糖贮积症患者、1例GM1神经节苷脂贮积症(Morquio B型)患者和1例I细胞病患者。唯一明显的临床结果是3例患者的角膜混浊减轻。观察到1例患者血浆中的β-半乳糖苷酶活性略有增加。改善时间约在移植后2个月,且是暂时的。

相似文献

1
Amniotic tissue transplantation as a trial of treatment in some lysosomal storage diseases.羊膜组织移植作为某些溶酶体贮积病的一种治疗试验。
J Inherit Metab Dis. 1985;8(3):101-4. doi: 10.1007/BF01819289.
2
A therapeutic trial of amniotic epithelial cell implantation in patients with lysosomal storage diseases.溶酶体贮积症患者羊膜上皮细胞植入的治疗试验。
Am J Med Genet. 1985 Oct;22(2):347-55. doi: 10.1002/ajmg.1320220219.
3
Mucopolysaccharidoses and mucolipidoses.黏多糖贮积症和黏脂贮积症。
J Clin Pathol Suppl (R Coll Pathol). 1974;8:64-93.
4
Ganglioside GM1 metabolism in living human fibroblasts with beta-galactosidase deficiency.β-半乳糖苷酶缺乏的人活体成纤维细胞中神经节苷脂GM1的代谢
Hum Genet. 1986 May;73(1):35-8. doi: 10.1007/BF00292661.
5
Genetic mucopolysaccharidoses, mannosidosis, sialidosis, galactosialidosis, and I-cell disease. Ultrastructural analysis of cultured fibroblasts.遗传性黏多糖贮积症、甘露糖苷贮积症、唾液酸贮积症、半乳糖唾液酸贮积症和I细胞病。培养成纤维细胞的超微结构分析。
Acta Pathol Jpn. 1987 Mar;37(3):385-400. doi: 10.1111/j.1440-1827.1987.tb00373.x.
6
Transplantation of amniotic epithelial membranes in patients with mucopolysaccharidoses.黏多糖贮积症患者羊膜上皮膜移植
Exp Clin Immunogenet. 1985;2(1):43-8.
7
[Storage diseases. Gangliosidosis and mucopolysaccharidosis].[贮积病。神经节苷脂沉积症和黏多糖贮积症]
Minerva Med. 1980 May 19;71(20):1469-72.
8
Corneal clouding in GM1-generalized gangliosidosis.GM1型全身性神经节苷脂贮积症中的角膜混浊
Br J Ophthalmol. 1976 Aug;60(8):565-7. doi: 10.1136/bjo.60.8.565.
9
The mucopolysaccharidoses and mucolipidoses.黏多糖贮积症和黏脂贮积症。
Clin Orthop Relat Res. 1976 Jan-Feb(114):116-33.
10
[Mucopolysaccharidoses. New therapeutic possibilities increase the need of early diagnosis].[黏多糖贮积症。新的治疗可能性增加了早期诊断的必要性]
Lakartidningen. 2002 Apr 18;99(16):1804-9.

引用本文的文献

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Human Amniotic Epithelial Stem Cells: A Promising Seed Cell for Clinical Applications.人羊膜上皮干细胞:一种有前途的临床应用种子细胞。
Int J Mol Sci. 2020 Oct 19;21(20):7730. doi: 10.3390/ijms21207730.
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Placenta-derived stem cells: new hope for cell therapy?胎盘源干细胞:细胞治疗的新希望?
Cytotechnology. 2008 Sep;58(1):33-42. doi: 10.1007/s10616-008-9162-z. Epub 2008 Sep 28.
3
Beta-galactosidase deficiency: an approach to chaperone therapy.β-半乳糖苷酶缺乏症:伴侣蛋白疗法的一种方法。

本文引用的文献

1
Enzyme replacement therapy by transplantation of HLA-compatible fibroblasts in Sanfilippo A syndrome.通过移植 HLA 相容的成纤维细胞对 Sanfilippo A 综合征进行酶替代疗法。
Pediatr Res. 1981 Jun;15(6):959-63. doi: 10.1203/00006450-198106000-00016.
2
The treatment of lysosomal storage diseases by fibroblast transplantation: some preliminary observations.成纤维细胞移植治疗溶酶体贮积症:一些初步观察结果。
Birth Defects Orig Artic Ser. 1980;16(1):457-74.
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A clinical trial of fibroblast transplantation for the treatment of mucopolysaccharidoses.
J Inherit Metab Dis. 2006 Apr-Jun;29(2-3):471-6. doi: 10.1007/s10545-006-0287-y.
4
Chemical chaperone therapy for brain pathology in G(M1)-gangliosidosis.用于GM1神经节苷脂贮积症脑部病变的化学伴侣疗法
Proc Natl Acad Sci U S A. 2003 Dec 23;100(26):15912-7. doi: 10.1073/pnas.2536657100. Epub 2003 Dec 15.
5
Attempted enzyme replacement using human amnion membrane implantations in mucopolysaccharidoses.尝试在黏多糖贮积症中使用人羊膜植入进行酶替代。
J Inherit Metab Dis. 1992;15(1):25-37. doi: 10.1007/BF01800340.
成纤维细胞移植治疗黏多糖贮积症的临床试验。
J Inherit Metab Dis. 1983;6(2):62-81. doi: 10.1007/BF02338973.
4
Expression of HLA antigens, beta 2-microglobulin and enzymes by human amniotic epithelial cells.人羊膜上皮细胞HLA抗原、β2-微球蛋白及酶的表达
Nature. 1982 Jan 28;295(5847):325-7. doi: 10.1038/295325a0.
5
Immunogenicity of human amniotic epithelial cells after transplantation into volunteers.人羊膜上皮细胞移植到志愿者体内后的免疫原性。
Lancet. 1981 Nov 7;2(8254):1003-5. doi: 10.1016/s0140-6736(81)91212-5.
6
A modified screening test for glycosaminoglycan excretion.一种改良的糖胺聚糖排泄筛查试验。
J Clin Pathol. 1969 May;22(3):379-80. doi: 10.1136/jcp.22.3.379-b.
7
A review and selection of simple laboratory methods used for the study of glycosaminoglycan excretion and the diagnosis of the mucopolysaccharidoses.用于研究糖胺聚糖排泄及黏多糖贮积症诊断的简单实验室方法综述与筛选。
J Clin Pathol. 1976 Feb;29(2):111-23. doi: 10.1136/jcp.29.2.111.
8
Arylsulfatases isoenzymes in metachromatic leucodystrophy/detection of a new variant by electrophoresis improvement of quantitative assay.异染性脑白质营养不良中的芳基硫酸酯酶同工酶/通过电泳改进定量测定法检测一种新变体
Biomedicine. 1975 Apr 10;23(3):116-9.