With the recent advances in gene editing with systems such as CRISPR-Cas9, precise genome editing in utero is on the horizon. Sickle cell disease is an excellent candidate for in utero fetal gene therapy, because the disease is monogenic, causes irreversible harm, and has life-limiting morbidity. Gene therapy has recently been proven to be effective in an adolescent patient. Several hurdles still impede the progress for fetal gene therapy in humans, including an incomplete understanding of the fetal immune system, unclear maternal immune responses to in utero gene therapy, risks of off-target effects from gene editing, gestational age constraints, and ethical questions surrounding fetal genetic intervention. However, none of these barriers appears insurmountable, and the journey to in utero gene therapy for sickle cell disease and other conditions should be well underway.

中文翻译：

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母胎医学会特别声明：超越手术刀：子宫内胎儿基因治疗和治疗医学

随着 CRISPR-Cas9 等系统在基因编辑方面的最新进展，子宫内精确的基因组编辑即将到来。镰状细胞病是子宫内胎儿基因治疗的绝佳候选者，因为该疾病是单基因的，会造成不可逆的伤害，并且具有限制生命的发病率。最近被证明基因疗法对青少年患者有效。人类胎儿基因治疗的进展仍然面临一些障碍，包括对胎儿免疫系统的不完全了解、母体对子宫内基因治疗的免疫反应不明确、基因编辑脱靶效应的风险、胎龄限制和伦理问题围绕胎儿遗传干预。然而，这些障碍似乎都不是不可克服的，镰状细胞病和其他疾病的子宫内基因治疗之旅应该会顺利进行。