Gene editing to cure sickle cell disease

基因编辑治疗镰状细胞病

• 批准号: nhmrc : 2001296
• 金额: $ 82.59万
• 依托单位国家: 澳大利亚
• 项目类别: Ideas Grants
• 财政年份: 2021
• 资助国家: 澳大利亚
• 起止时间: 2021-01-01 至 2024-12-31
• 项目状态: 已结题
• 来源: https://researchdata.edu.au/gene-editing-cure-cell-disease/1779756
• 关键词: Gene; editing; cure; sickle; cell

Sickle Cell Disease was the first molecular disease described in man, and is the most prevalent. In some African countries, India and the Middle East, up to 20% of the population carry the sickle gene mutation. In developing countries, 90% of children die before 5 years of age. In developed countries, patients suffer a lifetime of chronic pain and die ~20 years early. We will employ new gene editing approaches to repair the mutation or recruit fetal hemoglobin to cure SCD in human samples.

镰状细胞病是人类第一个被描述的分子疾病，也是最流行的。在一些非洲国家、印度和中东，多达20%的人口携带镰刀基因突变。在发展中国家，90%的儿童在5岁之前死亡。在发达国家，患者遭受终生慢性疼痛，并早死约20年。我们将使用新的基因编辑方法来修复突变或招募胎儿血红蛋白来治愈人类样本中的SCD。