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Tolerance induction and microglial engraftment after fetal therapy without conditioning in mice with Mucopolysaccharidosis type VII.
Quoc-Hung Nguyen, Russell G. Witt, Bowen Wang, Carlo Eikani, Jeremy Shea, Lucas K. Smith, Gabrielle Boyle, Jaclyn Cadaoas, Renan Sper, John D. MacKenzie, et al.
Sci Transl Med. 2020 Feb 26; 12(532) doi: 10.1126/scitranslmed.aay8980

Is Cited by the Following 17 Articles in this Archive:

Prenatal delivery of a therapeutic antisense oligonucleotide achieves broad biodistribution in the brain and ameliorates Angelman syndrome phenotype in mice
Maria T. Clarke, Laura Remesal, Lea Lentz, Danielle J. Tan, David Young, Slesha Thapa, Shalini R. Namuduri, Beltran Borges, Georgia Kirn, Jasmine Valencia, Manuel E. Lopez, Jan H. Lui, Lawrence R. Shiow, Scott Dindot, Saul Villeda, Stephan J. Sanders, Tippi C. MacKenzie
Mol Ther. 2024 Apr 3; 32(4): 935–951. Published online 2024 Feb 6. doi: 10.1016/j.ymthe.2024.02.004
PMCID:
PMC11163203
Abstract Article PDF–9.4M
Intrauterine enzyme replacement therapies for lysosomal storage disorders: Current developments and promising future prospects
Akos Herzeg, Beltran Borges, Billie R. Lianoglou, Juan Gonzalez-Velez, Emma Canepa, Dane Munar, Sarah P. Young, Deeksha Bali, Michel H. Gelb, Pranesh Chakraborty, Priya S. Kishnani, Paul Harmatz, Jennifer L. Cohen, Tippi C. MacKenzie
Prenat Diagn. Author manuscript; available in PMC 2024 Dec 1.
Published in final edited form as: Prenat Diagn. 2023 Dec; 43(13): 1638–1649. Published online 2023 Nov 13. doi: 10.1002/pd.6460
PMCID:
PMC11155627
Currently embargoed: Free in PMC on Dec 1, 2024; PubMed
Transplanting FVIII/ET3-secreting cells in fetal sheep increases FVIII levels long-term without inducing immunity or toxicity
Martin Rodriguez, Brady Trevisan, Ritu M. Ramamurthy, Sunil K. George, Jonathan Diaz, Jordan Alexander, Diane Meares, Denise J. Schwahn, David R. Quilici, Jorge Figueroa, Michael Gautreaux, Andrew Farland, Anthony Atala, Christopher B. Doering, H. Trent Spencer, Christopher D. Porada, Graça Almeida-Porada
Nat Commun. 2023; 14: 4206. Published online 2023 Jul 14. doi: 10.1038/s41467-023-39986-1
PMCID:
PMC10349136
Abstract Article PDF–6.8M
Fetal Genome Editing
Sourav K. Bose, Kara Kennedy, William H. Peranteau
Curr Opin Obstet Gynecol. Author manuscript; available in PMC 2024 Apr 1.
Published in final edited form as: Curr Opin Obstet Gynecol. 2023 Apr 1; 35(2): 134–139. doi: 10.1097/GCO.0000000000000854
PMCID:
PMC10027366
Abstract Article PDF–312K
When is the Best Time to Screen and Evaluate for Treatable Genetic Disorders?: A Lifespan Perspective
Melissa A. Parisi, Michele Caggana, Jennifer L. Cohen, Nina B. Gold, Jill A. Morris, Joseph J. Orsini, Tiina K. Urv, Melissa P. Wasserstein
Am J Med Genet C Semin Med Genet. Author manuscript; available in PMC 2024 Mar 6.
Published in final edited form as: Am J Med Genet C Semin Med Genet. 2023 Mar; 193(1): 44–55. Published online 2023 Mar 6. doi: 10.1002/ajmg.c.32036
PMCID:
PMC10475244
Abstract Article PDF–1.6M
In utero delivery of mRNA to the heart, diaphragm and muscle with lipid nanoparticles
Kewa Gao, Jie Li, Hengyue Song, Hesong Han, Yongheng Wang, Boyan Yin, Diana L. Farmer, Niren Murthy, Aijun Wang
Bioact Mater. 2023 Jul; 25: 387–398. Published online 2023 Feb 17. doi: 10.1016/j.bioactmat.2023.02.011
PMCID:
PMC9950423
Abstract Article PDF–12M
Diagnosis and Emerging Treatment Strategies for Mucopolysaccharidosis VII (Sly Syndrome)
Fabiano de Oliveira Poswar, Johanna Henriques Nehm, Francyne Kubaski, Edina Poletto, Roberto Giugliani
Ther Clin Risk Manag. 2022; 18: 1143–1155. Published online 2022 Dec 22. doi: 10.2147/TCRM.S351300
PMCID:
PMC9791935
Abstract Article PDF–1.3M
Targeting the central nervous system in lysosomal storage diseases: Strategies to deliver therapeutics across the blood-brain barrier
Bethan J. Critchley, H. Bobby Gaspar, Sara Benedetti
Mol Ther. 2023 Mar 1; 31(3): 657–675. Published online 2022 Nov 30. doi: 10.1016/j.ymthe.2022.11.015
PMCID:
PMC10014236
Abstract Article PDF–1.5M
In Utero Enzyme-Replacement Therapy for Infantile-Onset Pompe’s Disease
Jennifer L. Cohen, Pranesh Chakraborty, Karen Fung-Kee-Fung, Marisa E. Schwab, Deeksha Bali, Sarah P. Young, Michael H. Gelb, Hamid Khaledi, Alicia DiBattista, Stacey Smallshaw, Felipe Moretti, Derek Wong, Catherine Lacroix, Dina El Demellawy, Kyle C. Strickland, Jane Lougheed, Anita Moon-Grady, Billie R. Lianoglou, Paul Harmatz, Priya S. Kishnani, Tippi C. MacKenzie
N Engl J Med. Author manuscript; available in PMC 2024 Jan 17.
Published in final edited form as: N Engl J Med. 2022 Dec 8; 387(23): 2150–2158. Published online 2022 Nov 9. doi: 10.1056/NEJMoa2200587
PMCID:
PMC10794051
Abstract Article PDF–1.0M
Allogeneic and xenogeneic lymphoid reconstitution in a RAG2−/−IL2RGy/− severe combined immunodeficient pig: A preclinical model for intrauterine hematopoietic transplantation
Renan B. Sper, Jessica Proctor, Odessa Lascina, Ling Guo, Kathryn Polkoff, Tobias Kaeser, Sean Simpson, Luke Borst, Katherine Gleason, Xia Zhang, Bruce Collins, Yanet Murphy, Jeffrey L. Platt, Jorge A. Piedrahita
Front Vet Sci. 2022; 9: 965316. Published online 2022 Oct 14. doi: 10.3389/fvets.2022.965316
PMCID:
PMC9614384
Abstract Article PDF–8.3M
Experimental and clinical progress of in utero hematopoietic cell transplantation therapy for congenital disorders
Chunyu Shi, Lu Pan, Zheng Hu
Front Pharmacol. 2022; 13: 851375. Published online 2022 Sep 2. doi: 10.3389/fphar.2022.851375
PMCID:
PMC9478511
Abstract Article PDF–1.1M
Prenatal Somatic Cell Gene Therapies: Charting a Path Toward Clinical Applications (Proceedings of the CERSI-FDA Meeting)
Akos Herzeg, Graca Almeida-Porada, R. Alta Charo, Anna L. David, Juan Gonzalez-Velez, Nalin Gupta, Larissa Lapteva, Billie Lianoglou, William Peranteau, Christopher Porada, Stephan J. Sanders, Teresa N. Sparks, David H. Stitelman, Evi Struble, Charlotte J. Sumner, Tippi C. MacKenzie
J Clin Pharmacol. Author manuscript; available in PMC 2023 Sep 1.
Published in final edited form as: J Clin Pharmacol. 2022 Sep; 62(Suppl 1): S36–S52. doi: 10.1002/jcph.2127
PMCID:
PMC9547535
Abstract Article PDF–385K
Fetal therapies and trials for lysosomal storage diseases: a survey of attitudes of parents and patients
Marisa E. Schwab, Julia E. H. Brown, Billie Lianoglou, Chengshi Jin, Patricia C. Conroy, Renata C. Gallagher, Paul Harmatz, Tippi C. MacKenzie
Orphanet J Rare Dis. 2022; 17: 25. Published online 2022 Jan 29. doi: 10.1186/s13023-022-02178-z
PMCID:
PMC8800365
Abstract Article PDF–1020K
Current and emerging pharmacotherapy for Gaucher disease in pediatric populations
Richard Sam, Emory Ryan, Emily Daykin, Ellen Sidransky
Expert Opin Pharmacother. Author manuscript; available in PMC 2022 Aug 1.
Published in final edited form as: Expert Opin Pharmacother. 2021 Aug; 22(11): 1489–1503. Published online 2021 Mar 25. doi: 10.1080/14656566.2021.1902989
PMCID:
PMC8373623
Abstract Article PDF–606K
Ionizable lipid nanoparticles for in utero mRNA delivery
Rachel S. Riley, Meghana V. Kashyap, Margaret M. Billingsley, Brandon White, Mohamad-Gabriel Alameh, Sourav K. Bose, Philip W. Zoltick, Hiaying Li, Rui Zhang, Andrew Y. Cheng, Drew Weissman, William H. Peranteau, Michael J. Mitchell
Sci Adv. 2021 Jan; 7(3): eaba1028. Published online 2021 Jan 13. doi: 10.1126/sciadv.aba1028
PMCID:
PMC7806221
Abstract Article PDF–1.3M
Diagnosing neuronopathic Gaucher disease: New considerations and challenges in assigning Gaucher phenotypes
Emily Daykin, Emory Ryan, Ellen Sidransky
Mol Genet Metab. Author manuscript; available in PMC 2022 Feb 1.
Published in final edited form as: Mol Genet Metab. 2021 Feb; 132(2): 49–58. Published online 2021 Jan 9. doi: 10.1016/j.ymgme.2021.01.002
PMCID:
PMC7884077
Abstract Article PDF–369K
Ostm1 from Mouse to Human: Insights into Osteoclast Maturation
Jean Vacher, Michael Bruccoleri, Monica Pata
Int J Mol Sci. 2020 Aug; 21(16): 5600. Published online 2020 Aug 5. doi: 10.3390/ijms21165600
PMCID:
PMC7460669
Abstract Article PDF–1.9M