Boyle AK, Rinaldi SF, Norman JE, Stock SJ. Preterm birth: inflammation, fetal injury and treatment strategies. J Reprod Immunol. 2017;119:62–6.
David AL, Ahmadzia H, Ashcroft R, Bucci-Rechtweg C, Spencer RN, Thornton S. Improving development of drug treatments for pregnant women and the Fetus. Ther Innov Regul Sci. 2022;56(6):976–90.
Article PubMed PubMed Central Google Scholar
Papanikolaou E, Bosio A. The promise and the hope of gene therapy. Front Genome Ed. 2021;3:618346.
Article PubMed PubMed Central Google Scholar
Massaro G, Mattar CNZ, Wong AMS, Sirka E, Buckley SMK, Herbert BR, et al. Fetal gene therapy for neurodegenerative disease of infants. Nat Med. 2018;24(9):1317–23.
Article CAS PubMed PubMed Central Google Scholar
Rashnonejad A, Amini Chermahini G, Gunduz C, Onay H, Aykut A, Durmaz B, et al. Fetal gene therapy using a single injection of recombinant AAV9 rescued SMA phenotype in mice. Mol Ther. 2019;27(12):2123–33.
Article CAS PubMed PubMed Central Google Scholar
Ma W, Wei X, Gu H, Liu D, Luo W, An D, et al. Therapeutic potential of adenovirus-encoding brain-derived neurotrophic factor for spina bifida aperta by intra-amniotic delivery in a rat model. Gene Ther. 2020;27(12):567–78.
Article CAS PubMed Google Scholar
Shangaris P, Loukogeorgakis SP, Subramaniam S, Flouri C, Jackson LH, Wang W, et al. In utero gene therapy (IUGT) using GLOBE lentiviral vector phenotypically corrects the heterozygous humanised mouse model and its progress can be monitored using MRI techniques. Sci Rep. 2019;9(1):11592.
Article CAS PubMed PubMed Central Google Scholar
Karda R, Buckley SM, Mattar CN, Ng J, Massaro G, Hughes MP, et al. Perinatal systemic gene delivery using adeno-associated viral vectors. Front Mol Neurosci. 2014;7:89.
Article PubMed PubMed Central Google Scholar
Peranteau WH, Flake AW. The future of in utero gene therapy. Mol Diagn Ther. 2020;24(2):135–42.
Article PubMed PubMed Central Google Scholar
David AL, Peebles D. Gene therapy for the fetus: is there a future? Best Pract Res Clin Obstet Gynaecol. 2008;22(1):203–18.
Buckley SM, Rahim AA, Chan JK, David AL, Peebles DM, Coutelle C, et al. Recent advances in fetal gene therapy. Ther Deliv. 2011;2(4):461–9.
Article CAS PubMed Google Scholar
Coutelle C, Waddington SN. The concept of prenatal gene therapy. Methods Mol Biol (Clifton, NJ). 2012;891:1–7.
Waddington SN, Kramer MG, Hernandez-Alcoceba R, Buckley SM, Themis M, Coutelle C, et al. In utero gene therapy: current challenges and perspectives. Mol Ther. 2005;11(5):661–76.
Article CAS PubMed Google Scholar
Waddington SN, Peranteau WH, Rahim AA, Boyle AK, Kurian MA, Gissen P, et al. Fetal gene therapy. J Inherit Metab Dis. 2024;47(1):192–210.
David AL. Maternal uterine artery VEGF gene therapy for treatment of intrauterine growth restriction. Placenta. 2017;59(Suppl 1):S44–50.
Article CAS PubMed Google Scholar
David AL, Torondel B, Zachary I, Wigley V, Abi-Nader K, Mehta V, et al. Local delivery of VEGF adenovirus to the uterine artery increases vasorelaxation and uterine blood flow in the pregnant sheep. Gene Ther. 2008;15(19):1344–50.
Article CAS PubMed Google Scholar
Mehta V, Abi-Nader KN, Peebles DM, Benjamin E, Wigley V, Torondel B, et al. Long-term increase in uterine blood flow is achieved by local overexpression of VEGF-A(165) in the uterine arteries of pregnant sheep. Gene Ther. 2012;19(9):925–35.
Article CAS PubMed Google Scholar
Carr DJ, Wallace JM, Aitken RP, Milne JS, Mehta V, Martin JF, et al. Uteroplacental adenovirus vascular endothelial growth factor gene therapy increases fetal growth velocity in growth-restricted sheep pregnancies. Hum Gene Ther. 2014;25(4):375–84.
Article CAS PubMed PubMed Central Google Scholar
Carr DJ, Wallace JM, Aitken RP, Milne JS, Martin JF, Zachary IC, et al. Peri- and postnatal effects of prenatal adenoviral VEGF gene therapy in growth-restricted sheep. Biol Reprod. 2016;94(6):142.
Mehta V, Ofir K, Swanson A, Kloczko E, Boyd M, Barker H, et al. Gene targeting to the uteroplacental circulation of pregnant guinea pigs. Reprod Sci. 2016;23(8):1087–95.
Article CAS PubMed Google Scholar
Swanson AM, Rossi CA, Ofir K, Mehta V, Boyd M, Barker H, et al. Maternal therapy with Ad.VEGF-A165 increases fetal weight at term in a guinea-pig model of fetal growth restriction. Hum Gene Ther. 2016;27(12):997–1007.
Article CAS PubMed Google Scholar
Rossi C, Lees M, Mehta V, Heikura T, Martin J, Zachary I, et al. Comparison of efficiency and function of vascular endothelial growth factor adenovirus vectors in endothelial cells for gene therapy of placental insufficiency. Hum Gene Ther. 2020;31(21–22):1190–202.
Article CAS PubMed PubMed Central Google Scholar
Mehta V, Abi-Nader KN, Shangaris P, Shaw SW, Filippi E, Benjamin E, et al. Local over-expression of VEGF-DΔNΔC in the uterine arteries of pregnant sheep results in long-term changes in uterine artery contractility and angiogenesis. PLoS ONE. 2014;9(6):e100021.
Article PubMed PubMed Central Google Scholar
Desforges M, Rogue A, Pearson N, Rossi C, Olearo E, Forster R, et al. In Vitro Human placental studies to support adenovirus-mediated VEGF-DΔNΔC maternal gene therapy for the treatment of severe early-onset fetal growth restriction. Hum Gene Ther Clin Dev. 2018;29(1):10–23.
Article CAS PubMed Google Scholar
Vaughan OR, Rossi CA, Ginsberg Y, White A, Hristova M, Sebire NJ, et al. Perinatal and long-term effects of maternal uterine artery adenoviral VEGF-A165 gene therapy in the growth-restricted guinea pig fetus. Am J Physiol Regul Integr Comp Physiol. 2018;315(2):R344–53.
Article CAS PubMed Google Scholar
Spencer R, Rossi C, Lees M, Peebles D, Brocklehurst P, Martin J, et al. Achieving orphan designation for placental insufficiency: annual incidence estimations in Europe. BJOG. 2019;126(9):1157–67.
Article CAS PubMed Google Scholar
Spencer R, Ambler G, Brodszki J, Diemert A, Figueras F, Gratacos E, et al. EVERREST prospective study: a 6-year prospective study to define the clinical and biological characteristics of pregnancies affected by severe early onset fetal growth restriction. BMC Pregnancy Childbirth. 2017;17(1):43.
Article PubMed PubMed Central Google Scholar
• Spencer R, Maksym K, Hecher K, Maršál K, Figueras F, Ambler G, et al. Maternal PlGF and umbilical Dopplers predict pregnancy outcomes at diagnosis of early-onset fetal growth restriction. J Clin Invest. 2023;133(18):e169199. This study has important implications for the progression of the first in human trial for maternal gene therapy. It highlights the necessity for robust methods of outcome prediction for advancing experimental therapies.
Article PubMed PubMed Central Google Scholar
Babischkin JS, Aberdeen GW, Lindner JR, Bonagura TW, Pepe GJ, Albrecht ED. Vascular endothelial growth factor delivery to placental basal plate promotes uterine artery remodeling in the primate. Endocrinology. 2019;160(6):1492–505.
Article CAS PubMed PubMed Central Google Scholar
Swingle KL, Safford HC, Geisler HC, Hamilton AG, Thatte AS, Billingsley MM, et al. Ionizable lipid nanoparticles for in vivo mRNA delivery to the placenta during pregnancy. J Am Chem Soc. 2023;145(8):4691–706.
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