Correction of DMD exon 45 deletion by homology-independent targeted integration (HITI)
Correction of DMD exon 45 deletion by homology-independent targeted integration (HITI)
批准号:
454857
负责人:
Nicolau Stefan
金额:
$14.57万
依托单位国家:
加拿大
项目类别:
Fellowship Programs
财政年份:
2021
资助国家:
加拿大
项目状态:
未结题
起止时间:
2021-10-01 至 2025-10-01
中文摘要
杜氏肌营养不良症是一种严重的肌肉疾病,由DMD基因突变引起,该基因产生一种称为肌营养不良蛋白的蛋白质。这种基因位于X染色体上,因此几乎只影响男孩。这是最常见的童年-
英文摘要
Duchenne muscular dystrophy is a severe muscle disease caused by mutations in the DMD gene, which produces a protein called dystrophin. This gene is located on the X chromosome, and thus almost exclusively affects boys. This is the most common childhood-
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