2022
Prenatal Somatic Cell Gene Therapies: Charting a Path Toward Clinical Applications (Proceedings of the CERSI‐FDA Meeting)
Herzeg A, Almeida‐Porada G, Charo RA, David AL, Gonzalez‐Velez J, Gupta N, Lapteva L, Lianoglou B, Peranteau W, Porada C, Sanders SJ, Sparks TN, Stitelman DH, Struble E, Sumner CJ, MacKenzie TC. Prenatal Somatic Cell Gene Therapies: Charting a Path Toward Clinical Applications (Proceedings of the CERSI‐FDA Meeting). The Journal Of Clinical Pharmacology 2022, 62: s36-s52. PMID: 36106778, PMCID: PMC9547535, DOI: 10.1002/jcph.2127.Peer-Reviewed Original Research
2021
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