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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. Among authors: mackenzie t. 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.
Prenatal delivery of a therapeutic antisense oligonucleotide achieves broad biodistribution in the brain and ameliorates Angelman syndrome phenotype in mice.
Clarke MT, Remesal L, Lentz L, Tan DJ, Young D, Thapa S, Namuduri SR, Borges B, Kirn G, Valencia J, Lopez ME, Lui JH, Shiow LR, Dindot S, Villeda S, Sanders SJ, MacKenzie TC. Clarke MT, et al. Among authors: mackenzie tc. Mol Ther. 2024 Apr 3;32(4):935-951. doi: 10.1016/j.ymthe.2024.02.004. Epub 2024 Feb 6. Mol Ther. 2024. PMID: 38327047 Free PMC article.
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.
Non-Immune Hydrops Fetalis: Do Placentomegaly and Polyhydramnios Matter?
Berger VK, Sparks TN, Jelin AC, Derderian C, Jeanty C, Gosnell K, Mackenzie T, Gonzalez JM. Berger VK, et al. Among authors: mackenzie t. J Ultrasound Med. 2018 May;37(5):1185-1191. doi: 10.1002/jum.14462. Epub 2017 Oct 27. J Ultrasound Med. 2018. PMID: 29076544 Free PMC article.
Exome Sequencing for Prenatal Diagnosis in Nonimmune Hydrops Fetalis.
Sparks TN, Lianoglou BR, Adami RR, Pluym ID, Holliman K, Duffy J, Downum SL, Patel S, Faubel A, Boe NM, Field NT, Murphy A, Laurent LC, Jolley J, Uy C, Slavotinek AM, Devine P, Hodoglugil U, Van Ziffle J, Sanders SJ, MacKenzie TC, Norton ME; University of California Fetal–Maternal Consortium; University of California, San Francisco Center for Maternal–Fetal Precision Medicine. Sparks TN, et al. Among authors: mackenzie tc. N Engl J Med. 2020 Oct 29;383(18):1746-1756. doi: 10.1056/NEJMoa2023643. Epub 2020 Oct 7. N Engl J Med. 2020. PMID: 33027564 Free PMC article.
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.
Advances in the management of α-thalassemia major: reasons to be optimistic.
Horvei P, MacKenzie T, Kharbanda S. Horvei P, et al. Among authors: mackenzie t. Hematology Am Soc Hematol Educ Program. 2021 Dec 10;2021(1):592-599. doi: 10.1182/hematology.2021000295. Hematology Am Soc Hematol Educ Program. 2021. PMID: 34889445 Free PMC article. Review.
694 results