检索结果-南通市图书馆

Delivering on the promise of gene editing for cystic fibrosis

Genes & Diseases 2019年第2期6卷 97-108页

作者： craig a.hodges Ronald A.Conlon Department of Pediatrics Case Western Reserve UniversityClevelandOHUSA Department of Genetics and Genome Sciences Case Western Reserve UniversityClevelandOHUSA

In this review,we describe a path for translation of gene editing into therapy for cystic fibrosis(CF).Cystic fibrosis results from mutations in the CFTR gene,with one allele predominant in patient *** simple,genetic etiology makes gene editing appealing for treatment of this *** already have been success in applying this approach to cystic fibrosis in cell and animal models,although these advances have been modest in comparison to advances for other *** than six years after its first demonstration in animals,CRISPR/Cas gene editing is in early clinical trials for several *** clinical trials,thus far,attempt to edit genes in cells of the blood *** advantage of the blood is that the stem cells are known,can be isolated,edited,selected,expanded,and returned to the *** likely next trials will be in the liver,which is accessible to many delivery *** cystic fibrosis,the biggest hurdle is to deliver editors to other,less accessible *** outline a path by which delivery can be *** translation of new therapies doesn’t occur in isolation,and the development of gene editors is occurring as advances in gene therapy and small molecule therapeutics are being *** advances made in gene therapy may help develop delivery vehicles for gene editing,although major improvements are ***,the approval of effective small molecule therapies for many patients with cystic fibrosis will raise the bar for translation of gene editing.

关键词： CFTR gene CRISPR/Cas9 Cystic fibrosis Gene editing Gene therapy

来源：

维普期刊数据库评论

在线全文

维普期刊数据库

学校读者我要写书评

暂无评论

欢迎您,

建议与咨询留下您的常用邮箱和电话号码，以便我们向您反馈解决方案和替代方法

时间限定

文献类型

馆藏选择

核心期刊

语言

文献类型

帮助

文字说明：

检索规则说明：

检索范例：

分类表

所选分类

限定检索结果

文献类型

馆藏范围

日期分布

学科分类号

主题

机构

作者

语言

在线全文

请选择保存的检索档案：

请选择收藏分类：

通借通还

欢迎您,

建议与咨询 留下您的常用邮箱和电话号码，以便我们向您反馈解决方案和替代方法

时间限定

文献类型

馆藏选择

核心期刊

语言

文献类型

帮助

文字说明：

检索规则说明：

检索范例：

分类表

所选分类

限定检索结果

文献类型

馆藏范围

日期分布

学科分类号

主题

机构

作者

语言

在线全文

请选择保存的检索档案： 新增检索档案 确定 取消

请选择收藏分类： 新增自定义分类 确定 取消

通借通还

建议与咨询留下您的常用邮箱和电话号码，以便我们向您反馈解决方案和替代方法

请选择保存的检索档案：

请选择收藏分类：