In Utero Fetal Therapy: Stem Cells, Cell Transplantation, Gene Therapy, and CRISPR-Cas9
In Utero Fetal Therapy: Stem Cells, Cell Transplantation, Gene Therapy, and CRISPR-Cas9
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DOI：
10.1097/grf.0000000000000663
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发表时间：
2021-12-01
影响因子：
1.5
通讯作者：
Massa, Andrew
中科院分区：
文献类型：
--
作者：
Shear, Matthew A.;Massa, Andrew
子宫内胎儿治疗提供了预防和治疗细胞或遗传基础疾病的机会。成功的胎儿治疗的组成部分包括替代细胞群的分离、子宫内干细胞移植、具有胎儿免疫耐受的细胞植入和持续的细胞功能。使用CRISPR-Cas9的胎儿基因疗法代表了一种令人兴奋的潜在疗法，用于治疗不适合通过腺病毒载体转导进行基因补充的遗传疾病。这些胎儿疗法具有独特的伦理和安全考虑。子宫内细胞治疗的临床试验正在进行中，因为干细胞生物学和基因治疗的其他发现更接近临床转化。
In utero fetal therapy offers the opportunity to prevent and treat diseases with a cellular or genetic basis. Components of successful fetal treatment include isolation of a replacement cell population, in utero stem cell transplantation, cell engraftment with fetal immune tolerance, and ongoing cell function. Fetal gene therapy with CRISPR-Cas9 represents an exciting potential therapy for genetic diseases not amenable to gene supplementation via adenoviral vector transduction. These fetal therapies have unique ethical and safety considerations. Clinical trials for in utero cell therapy are underway, as additional discoveries in stem cell biology and gene therapy move closer to clinical translation.