Division of Newborn Medicine, Boston Children's Hospital, Boston, Massachusetts.
Department of Pediatrics, Harvard Medical School, Boston, Massachusetts.
JAMA Pediatr. 2020 Oct 1;174(10):985-991. doi: 10.1001/jamapediatrics.2020.1519.
Gene and stem cell therapies have become viable therapeutic options for many postnatal disorders. For select conditions, prenatal application would provide improved outcomes. The fetal state allows for several theoretical advantages over postnatal therapy, including immune immaturity and cellular niche accessibility.
Advances in prenatal diagnostic accuracy and surgical precision, as well as improvements in stem cell and gene therapy methods, have made prenatal gene and stem cell therapy realistic. Studies in mouse models and early human trials demonstrate the feasibility of these approaches. Additional efforts are under way to streamline fetal applications of stem cell and gene therapy while carefully considering best ethical practice and following established regulatory pathways.
Fetal stem cell and gene therapy bring important therapeutic opportunities for select disorders that present in the fetal and neonatal periods. While this field is in its infancy, these therapies are starting to be available clinically, and clinicians should be aware of their benefits and challenges.
基因和干细胞疗法已成为许多产后疾病的可行治疗选择。对于某些特定情况，产前应用将提供更好的效果。与产后治疗相比，胎儿状态具有几个理论上的优势，包括免疫不成熟和细胞生态位可及性。
产前诊断准确性和手术精度的提高，以及干细胞和基因治疗方法的改进，使得产前基因和干细胞治疗成为现实。在小鼠模型和早期人体试验中的研究证明了这些方法的可行性。正在努力简化干细胞和基因治疗的胎儿应用，同时仔细考虑最佳伦理实践并遵循既定的监管途径。
胎儿干细胞和基因治疗为在胎儿和新生儿期出现的某些疾病带来了重要的治疗机会。虽然这个领域还处于起步阶段，但这些治疗方法已经开始在临床上应用，临床医生应该了解它们的益处和挑战。
