Barranger J A, Rice E O, Swaney W P
Human Genetics Department at the University of Pittsburgh, PA 15261, USA.
Neurochem Res. 1999 Apr;24(4):601-15. doi: 10.1023/a:1022548232735.
The work summarized in this paper used animal and cell culture models systems to develop gene therapy approaches for the lysosomal storage disorders. The results have provided the scientific basis for a clinical trial of gene transfer to hematopoietic stem cells (HSC) in Gaucher disease which is now in progress. The clinical experiment is providing evidence of HSC transduction, competitive engraftment of genetically corrected HSC, expression of the GC transgene, and the suggestion of a clinical response. In this paper we will review the progress made in Gaucher disease and include how gene transfer might be studied in other lysosomal storage disorders.
本文总结的工作使用动物和细胞培养模型系统来开发针对溶酶体贮积症的基因治疗方法。研究结果为目前正在进行的戈谢病造血干细胞（HSC）基因转移临床试验提供了科学依据。该临床实验正在提供造血干细胞转导、基因校正造血干细胞的竞争性植入、GC转基因表达的证据，以及临床反应的迹象。在本文中，我们将回顾戈谢病所取得的进展，并探讨如何在其他溶酶体贮积症中研究基因转移。
