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Retroviral-mediated transfer of the iduronate-2-sulfatase gene into lymphocytes for treatment of mild Hunter syndrome (mucopolysaccharidosis type II).

作者信息

Whitley C B, McIvor R S, Aronovich E L, Berry S A, Blazar B R, Burger S R, Kersey J H, King R A, Faras A J, Latchaw R E, McCullough J, Pan D, Ramsay N K, Stroncek D F

机构信息

University of Minnesota Medical School, Minneapolis, USA.

出版信息

Hum Gene Ther. 1996 Mar 1;7(4):537-49. doi: 10.1089/hum.1996.7.4-537.

DOI:10.1089/hum.1996.7.4-537
PMID:8800749
Abstract
摘要

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1
Retroviral-mediated transfer of the iduronate-2-sulfatase gene into lymphocytes for treatment of mild Hunter syndrome (mucopolysaccharidosis type II).逆转录病毒介导的艾杜糖醛酸-2-硫酸酯酶基因转移至淋巴细胞用于治疗轻度亨特综合征（II型黏多糖贮积症）。
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2
Preclinical studies of lymphocyte gene therapy for mild Hunter syndrome (mucopolysaccharidosis type II).轻度亨特综合征（II型黏多糖贮积症）淋巴细胞基因治疗的临床前研究。
Hum Gene Ther. 1996 Feb 10;7(3):283-90. doi: 10.1089/hum.1996.7.3-283.
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Metabolic correction and cross-correction of mucopolysaccharidosis type II (Hunter syndrome) by retroviral-mediated gene transfer and expression of human iduronate-2-sulfatase.通过逆转录病毒介导的基因转移和人艾杜糖醛酸-2-硫酸酯酶的表达对II型粘多糖贮积症（亨特综合征）进行代谢校正和交叉校正
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Combined ultrafiltration-transduction in a hollow-fiber bioreactor facilitates retrovirus-mediated gene transfer into peripheral blood lymphocytes from patients with mucopolysaccharidosis type II.中空纤维生物反应器中的联合超滤转导有助于逆转录病毒介导的基因转移到黏多糖贮积症II型患者的外周血淋巴细胞中。
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Prevention of Neurocognitive Deficiency in Mucopolysaccharidosis Type II Mice by Central Nervous System-Directed, AAV9-Mediated Iduronate Sulfatase Gene Transfer.通过中枢神经系统靶向、AAV9 介导的艾杜糖硫酸酯酶基因转移预防黏多糖贮积症 II 型小鼠的神经认知缺陷。
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Genetics and Gene Therapy in Hunter Disease.亨特病的遗传学和基因治疗。
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In vitro correction of iduronate-2-sulfatase deficiency by adenovirus-mediated gene transfer.通过腺病毒介导的基因转移对艾杜糖醛酸-2-硫酸酯酶缺乏症进行体外校正。
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Idursulfase enzyme replacement therapy in an adult patient with severe Hunter syndrome having a novel mutation of iduronate-2-sulfatase gene.伊杜硫酸酯酶酶替代疗法治疗患有新型艾杜糖-2-硫酸酯酶基因突变的亨特综合征成年患者。
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Pathol Oncol Res. 1999;5(1):3-15. doi: 10.1053/paor.1999.0003.
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