A narrative review of in utero gene therapy: advances, challenges, and future considerations
Yung NK, Maassel NL, Ullrich SJ, Ricciardi AS, Stitelman DH. A narrative review of in utero gene therapy: advances, challenges, and future considerations. Translational Pediatrics 2021, 10: 1486496-1481496. PMID: 34189107, PMCID: PMC8192997, DOI: 10.21037/tp-20-89.Peer-Reviewed Original ResearchHighest effective doseGene therapyPathogenesis increasesLysosomal storage disorderImmune tolerancePostnatal treatmentTreatment optionsClinical trialsAnimal modelsCystic fibrosisFetal diagnosisNarrative reviewTranslational gapEffective doseUtero gene therapyPhysiologic phenomenonStorage disorderDiseaseNext-generation sequencingIUGTTechnologic advancementsCandidate diseaseClinical realmTherapyPotential disease