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In Utero Enzyme-Replacement Therapy for Infantile-Onset Pompe's Disease.
Cohen JL, Chakraborty P, Fung-Kee-Fung K, Schwab ME, Bali D, Young SP, Gelb MH, Khaledi H, DiBattista A, Smallshaw S, Moretti F, Wong D, Lacroix C, El Demellawy D, Strickland KC, Lougheed J, Moon-Grady A, Lianoglou BR, Harmatz P, Kishnani PS, MacKenzie TC. Cohen JL, et al. N Engl J Med. 2022 Dec 8;387(23):2150-2158. doi: 10.1056/NEJMoa2200587. Epub 2022 Nov 9. N Engl J Med. 2022. PMID: 36351280 Free PMC article.
Dual α-globin-truncated erythropoietin receptor knockin restores hemoglobin production in α-thalassemia-derived erythroid cells.
Chu SN, Soupene E, Sharma D, Sinha R, McCreary T, Hernandez B, Shen H, Wienert B, Bowman C, Yin H, Lesch BJ, Jia K, Romero KA, Kostamo Z, Zhang Y, Tran T, Cordero M, Homma S, Hampton JP, Gardner JM, Conklin BR, MacKenzie TC, Sheehan VA, Porteus MH, Cromer MK. Chu SN, et al. Cell Rep. 2025 Jan 28;44(1):115141. doi: 10.1016/j.celrep.2024.115141. Epub 2025 Jan 3. Cell Rep. 2025. PMID: 39754719 Free PMC article.
In Utero Gene Therapy Consensus Statement from the IFeTIS.
Almeida-Porada G, Waddington SN, Chan JKY, Peranteau WH, MacKenzie T, Porada CD. Almeida-Porada G, et al. Mol Ther. 2019 Apr 10;27(4):705-707. doi: 10.1016/j.ymthe.2019.02.015. Epub 2019 Mar 2. Mol Ther. 2019. PMID: 30837116 Free PMC article. No abstract available.
Growth Failure Prevalence in Neonates with Gastroschisis : A Statewide Cohort Study.
Strobel KM, Romero T, Kramer K, Fernandez E, Rottkamp C, Uy C, Keller R, Moyer L, Poulain F, Kim JH, DeUgarte DA, Calkins KL; University of California Fetal Consortium. Strobel KM, et al. J Pediatr. 2021 Jun;233:112-118.e3. doi: 10.1016/j.jpeds.2021.02.013. Epub 2021 Feb 26. J Pediatr. 2021. PMID: 33647253 Free PMC article.
764 results