Molecular and Cellular In Utero Therapy.
Molecular and Cellular In Utero Therapy.
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子宫内分子和细胞治疗。
DOI：
10.1016/j.clp.2022.06.005
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发表时间：
2022
影响因子：
2.1
通讯作者：
Peranteau,WilliamH
中科院分区：
文献类型：
--
作者：
Berkowitz,CaraL;Luks,ValerieL;Puc,Marcelina;Peranteau,WilliamH
母体胎儿医学和基因测序技术的重大进步促进了宫内分子和细胞治疗的新前沿，包括基因编辑、酶替代治疗和干细胞移植，以治疗出生后治疗策略有限的单基因疾病。宫内治疗利用胎儿独特的发育特性，在不可逆转的疾病病理发展之前纠正单基因障碍。虽然早期的动物模型临床前研究令人鼓舞，但在广泛临床应用之前，还需要更多的研究来进一步评估其安全性和有效性。
Significant advances in maternal fetal medicine and gene sequencing technology have fostered a new frontier of in utero molecular and cellular therapeutics, including gene editing, enzyme replacement therapy and stem cell transplantation to treat single gene disorders with limited postnatal treatment strategies. In utero therapies take advantage of unique developmental properties of the fetus to allow for correction of monogenic disorders before irreversible disease pathology develops. While early preclinical studies in animal models are encouraging, more studies are needed to further evaluate their safety and efficacy prior to widespread clinical use.