• 主题
高级搜索  >
历史搜索 清空历史
首页> 外文期刊>Molecular therapy: the journal of the American Society of Gene Therapy >Stem cell gene therapy for fanconi anemia: report from the 1st international Fanconi anemia gene therapy working group meeting.
【24h】

Stem cell gene therapy for fanconi anemia: report from the 1st international Fanconi anemia gene therapy working group meeting.

机译：范可尼贫血的干细胞基因治疗：第一次国际范可尼贫血基因治疗工作组会议的报告。

获取原文
获取原文并翻译 | 示例
           
页面导航
  • 摘要
  • 著录项
  • 相似文献
  • 相关主题

摘要

Survival rates after allogeneic hematopoietic cell transplantation (HCT) for Fanconi anemia (FA) have increased dramatically since 2000. However, the use of autologous stem cell gene therapy, whereby the patient's own blood stem cells are modified to express the wild-type gene product, could potentially avoid the early and late complications of allogeneic HCT. Over the last decades, gene therapy has experienced a high degree of optimism interrupted by periods of diminished expectation. Optimism stems from recent examples of successful gene correction in several congenital immunodeficiencies, whereas diminished expectations come from the realization that gene therapy will not be free of side effects. The goal of the 1st International Fanconi Anemia Gene Therapy Working Group Meeting was to determine the optimal strategy for moving stem cell gene therapy into clinical trials for individuals with FA. To this end, key investigators examined vector design, transduction method, criteria for large-scale clinical-grade vector manufacture, hematopoietic cell preparation, and eligibility criteria for FA patients most likely to benefit. The report summarizes the roadmap for the development of gene therapy for FA.
机译：自2000年以来，异基因造血细胞移植（HCT）后的范可尼贫血（FA）的存活率显着提高。但是，自体干细胞基因疗法的应用使患者自身的血液干细胞得以修饰以表达野生型基因产物，有可能避免同种异体HCT的早期和晚期并发症。在过去的几十年中，基因治疗经历了高度的乐观情绪，并因期望期缩短而中断。乐观来自最近在一些先天性免疫缺陷中成功进行基因校正的例子，而对基因治疗将不会产生副作用的认识却降低了期望。第一届国际范可尼贫血基因治疗工作组会议的目标是确定将干细胞基因治疗应用于FA患者的临床试验的最佳策略。为此，主要研究人员检查了载体设计，转导方法，大规模临床级载体生产的标准，造血细胞的制备以及最有可能受益的FA患者的资格标准。该报告总结了发展FA基因治疗的路线图。

著录项

相似文献

  • 外文文献
  • 中文文献
  • 专利
获取原文

客服邮箱：kefu@zhangqiaokeyan.com

京公网安备：11010802029741号 ICP备案号：京ICP备15016152号-6 六维联合信息科技 (北京) 有限公司©版权所有

  • 客服微信

  • 服务号