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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
Prenatal delivery of a therapeutic antisense oligonucleotide achieves broad biodistribution in the brain and ameliorates Angelman syndrome phenotype in mice.
Molecular therapy : the journal of the American Society of Gene Therapy, 2024-02-06.
Herzeg A, Borges B, Lianoglou BR, Gonzalez-Velez J, Canepa E, Munar D, Young SP, Bali D, Gelb MH, Chakraborty P, Kishnani PS, Harmatz P, Cohen JL, MacKenzie TC
Intrauterine enzyme replacement therapies for lysosomal storage disorders: Current developments and promising future prospects.
Prenatal diagnosis, 2023-11-13.
Lowther C, Valkanas E, Giordano JL, Wang HZ, Currall BB, O'Keefe K, Pierce-Hoffman E, Kurtas NE, Whelan CW, Hao SP, Weisburd B, Jalili V, Fu J, Wong I, Collins RL, Zhao X, Austin-Tse CA, Evangelista E, Lemire G, Aggarwal VS, Lucente D, Gauthier LD, Tolonen C, Sahakian N, Stevens C, An JY, Dong S, Norton ME, MacKenzie TC, Devlin B, Gilmore K, Powell BC, Brandt A, Vetrini F, DiVito M, Sanders SJ, MacArthur DG, Hodge JC, O'Donnell-Luria A, Rehm HL, Vora NL, Levy B, Brand H, Wapner RJ, Talkowski ME
Systematic evaluation of genome sequencing for the diagnostic assessment of autism spectrum disorder and fetal structural anomalies.
American journal of human genetics, 2023-08-17.
Yu TW, Kingsmore SF, Green RC, MacKenzie T, Wasserstein M, Caggana M, Gold NB, Kennedy A, Kishnani PS, Might M, Brooks PJ, Morris JA, Parisi MA, Urv TK
Are we prepared to deliver gene-targeted therapies for rare diseases?
American journal of medical genetics. Part C, Seminars in medical genetics, 2023-01-24.
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
In Utero Enzyme-Replacement Therapy for Infantile-Onset Pompe's Disease.
The New England journal of medicine, 2022-11-09.
MacKenzie TC, Schwab ME, Lianoglou BR, Gano D, Gonzalez J, Arvon RL, Baschat AA, Bianchi DW, Bitanga M, Bourguignon A, Brown RN, Chen B, Chien M, Davis-Nelson S, de Laat MWM, Ekwattanakit S, Gollin Y, Hirata G, Jelin A, Jolley J, Meyer P, Miller J, Norton ME, Ogasawara KK, Panchalee T, Schindewolf E, Shaw SW, Stumbaugh TL, Thompson AA, Towner D, Tsai PS, Viprakasit V, Volanakis E, Vichinsky E, Allen IE, Zhang L
The impact of in utero transfusions on perinatal outcomes in patients with alpha thalassemia major: the UCSF registry.
Blood advances, 2022-10-28.
Schwab ME, Shao S, Zhang L, Lianoglou B, Belter L, Jarecki J, Schroth M, Sumner CJ, MacKenzie T
Investigating attitudes toward prenatal diagnosis and fetal therapy for spinal muscular atrophy.
Prenatal diagnosis, 2022-09-03.
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).
Journal of clinical pharmacology, 2022-09-01.
Sun IH, Gillis-Buck E, Mackenzie TC, Gardner JM
Thymic and extrathymic Aire-expressing cells in maternal-fetal tolerance.
Immunological reviews, 2022-05-10.
Schwab ME, Brown JEH, Lianoglou B, Jin C, Conroy PC, Gallagher RC, Harmatz P, MacKenzie TC
Fetal therapies and trials for lysosomal storage diseases: a survey of attitudes of parents and patients.
Orphanet journal of rare diseases, 2022-01-29.
Horvei P, MacKenzie T, Kharbanda S
Advances in the management of α-thalassemia major: reasons to be optimistic.
Hematology. American Society of Hematology. Education Program, 2021-12-10.
Schwab ME, MacKenzie TC
Prenatal Gene Therapy.
Clinical obstetrics and gynecology, 2021-12-01.
MacKenzie TC, Amid A, Angastiniotis M, Butler C, Gilbert S, Gonzalez J, Keller RL, Kharbanda S, Kirby-Allen MA, Koenig BA, Kyono W, Lal A, Lianoglou BR, Norton ME, Ogasawara KK, Panchalee T, Rosner M, Schwab ME, Thompson AA, Waye JS, Vichinsky E
Consensus statement for the perinatal management of patients with alpha thalassemia major.
Blood advances, 2021-11-08.
Le BL, Sper R, Nielsen SCA, Pineda S, Nguyen QH, Lee JY, Boyd SD, MacKenzie TC, Sirota M
Maternal and Infant Immune Repertoire Sequencing Analysis Identifies Distinct Ig and TCR Development in Term and Preterm Infants.
Journal of immunology (Baltimore, Md. : 1950), 2021-10-15.
Schwab ME, Dong S, Lianoglou BR, Aguilar Lucero AF, Schwartz GB, Norton ME, MacKenzie TC, Sanders SJ
Exome sequencing of fetuses with congenital diaphragmatic hernia supports a causal role for NR2F2, PTPN11, and WT1 variants.
American journal of surgery, 2021-07-20.
Gillis-Buck E, Miller H, Sirota M, Sanders SJ, Ntranos V, Anderson MS, Gardner JM, MacKenzie TC
Extrathymic Aire-expressing cells support maternal-fetal tolerance.
Science immunology, 2021-07-16.
Bianchi DW, Khosrotehrani K, Way SS, MacKenzie TC, Bajema I, O'Donoghue K
Forever Connected: The Lifelong Biological Consequences of Fetomaternal and Maternofetal Microchimerism.
Clinical chemistry, 2021-01-08.
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
Exome Sequencing for Prenatal Diagnosis in Nonimmune Hydrops Fetalis.
The New England journal of medicine, 2020-10-07.
Sagar R, Almeida-Porada G, Blakemore K, Chan JKY, Choolani M, Götherström C, Jaulent A, MacKenzie TC, Mattar C, Porada CD, Peranteau WH, Schneider H, Shaw SW, Waddington SN, Westgren M, David AL
Fetal and Maternal Safety Considerations for In Utero Therapy Clinical Trials: iFeTiS Consensus Statement.
Molecular therapy : the journal of the American Society of Gene Therapy, 2020-10-16.
Witt RG, Blair L, Frascoli M, Rosen MJ, Nguyen QH, Bercovici S, Zompi S, Romero R, Mackenzie TC
Detection of microbial cell-free DNA in maternal and umbilical cord plasma in patients with chorioamnionitis using next generation sequencing.
PloS one, 2020-04-15.
Nguyen QH, Witt RG, Wang B, Eikani C, Shea J, Smith LK, Boyle G, Cadaoas J, Sper R, MacKenzie JD, Villeda S, MacKenzie TC
Tolerance induction and microglial engraftment after fetal therapy without conditioning in mice with Mucopolysaccharidosis type VII.
Science translational medicine, 2020-02-26.
Mouzakiti N, Sierra F, Herzeg A, Al Naimi A, Reising C, Bahlmann F, Kyvernitakis I
The impact of a short cervix and funneling on the outcome in singleton pregnancies treated with an Arabin-pessary or a McDonald cerclage.
The journal of maternal-fetal & neonatal medicine : the official journal of the European Association of Perinatal Medicine, the Federation of Asia and Oceania Perinatal Societies, the International Society of Perinatal Obstetricians, 2019-09-25.
Halkias J, Rackaityte E, Hillman SL, Aran D, Mendoza VF, Marshall LR, MacKenzie TC, Burt TD
CD161 contributes to prenatal immune suppression of IFN?-producing PLZF+ T cells.
The Journal of clinical investigation, 2019-05-30.
Almeida-Porada G, Waddington SN, Chan JKY, Peranteau WH, MacKenzie T, Porada CD
In Utero Gene Therapy Consensus Statement from the IFeTIS.
Molecular therapy : the journal of the American Society of Gene Therapy, 2019-03-02.
Wolnicki BG, von Wedel F, Mouzakiti N, Al Naimi A, Herzeg A, Bahlmann F, Kyvernitakis I
Combined treatment of McDonald cerclage and Arabin-pessary: a chance in the prevention of spontaneous preterm birth?
The journal of maternal-fetal & neonatal medicine : the official journal of the European Association of Perinatal Medicine, the Federation of Asia and Oceania Perinatal Societies, the International Society of Perinatal Obstetricians, 2019-01-30.
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