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Correction of DMD exon 45 deletion by homology-independent targeted integration (HITI)

Correction of DMD exon 45 deletion by homology-independent targeted integration (HITI)
通过同源独立靶向整合 (HITI) 校正 DMD 外显子 45 缺失
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

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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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