Zobrazeno 1 - 7
of 7
pro vyhledávání: '"N = 1 Collaborative"'
Publikováno v:
Communications Medicine, Vol 4, Iss 1, Pp 1-11 (2024)
Abstract Antisense oligonucleotides (ASOs) are incredibly versatile molecules that can be designed to specifically target and modify RNA transcripts to slow down or halt rare genetic disease progression. They offer the potential to target groups of p
Externí odkaz:
https://doaj.org/article/5921451646084845bc63da2e688a2b76
Akademický článek
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Autor:
Aartsma-Rus, A., Garanto, A., Roon-Mom, W. van, McConnell, E.M., Suslovitch, V., Yan, W.X., Watts, J.K., Yu, T.W., N 1 Collaborative
Publikováno v:
Nucleic Acid Therapeutics, 33, 17-25
Nucleic Acid Therapeutics, 33(1). MARY ANN LIEBERT, INC
Nucleic Acid Therapeutics, 33, 1, pp. 17-25
Nucleic Acid Therapeutics, 33(1). MARY ANN LIEBERT, INC
Nucleic Acid Therapeutics, 33, 1, pp. 17-25
Contains fulltext : 291301.pdf (Publisher’s version ) (Open Access) Antisense oligonucleotides (ASOs) can modulate pre-mRNA splicing. This offers therapeutic opportunities for numerous genetic diseases, often in a mutation-specific and sometimes ev
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::d05c342d5e6158a996b81a2ffd6dab90
https://hdl.handle.net/1887/3563780
https://hdl.handle.net/1887/3563780
Autor:
Kim-McManus O; Rady Children's Institute for Genomic Medicine, San Diego, CA, USA. okimmcmanus@health.ucsd.edu.; Department of Neurosciences, University of California, La Jolla, CA, USA. okimmcmanus@health.ucsd.edu., Gleeson JG; Rady Children's Institute for Genomic Medicine, San Diego, CA, USA.; Department of Neurosciences, University of California, La Jolla, CA, USA.; n-Lorem Foundation, Carlsbad, CA, USA., Mignon L; n-Lorem Foundation, Carlsbad, CA, USA., Smith Fine A; Department of Neurology, Kennedy Krieger Institute, Baltimore, MD, USA.; Johns Hopkins School of Medicine, Baltimore, MD, USA., Yan W; N=1 Collaborative, Somerville, MA, USA., Nolen N; N=1 Collaborative, Somerville, MA, USA., Demarest S; Department of Pediatrics, Children's Hospital Colorado, CO, Baltimore, USA., Berry-Kravis E; Department of Pediatrics, Rush University Medical Center, Chicago, IL, USA., Finkel R; Department of Pediatric Medicine, St. Jude Children's Research Hospital, Memphis, TN, USA., Leonard S; N=1 Collaborative, Somerville, MA, USA., Finlayson S; Department of Pediatrics, Seattle Children's Hospital, Seattle, WA, USA., Augustine E; Department of Neurology, Kennedy Krieger Institute, Baltimore, MD, USA., Lyon GJ; Department of Human Genetics, New York State Institute for Basic Research in Developmental Disabilities, Staten Island, NY, USA.; Graduate Center, The City University of New York, New York, NY, USA., Schule R; Department of Neurology, Heidelberg University Hospital, Heidelberg, Germany., Yu T; N=1 Collaborative, Somerville, MA, USA.; Division of Genetics and Genomics, Boston Children's Hospital, Boston, MA, USA.; Department of Pediatrics, Harvard Medical School, Boston, MA, USA.
Publikováno v:
Nature communications [Nat Commun] 2024 Nov 12; Vol. 15 (1), pp. 9802. Date of Electronic Publication: 2024 Nov 12.
Autor:
Pian JMY; Boston Children's Hospital, Boston, MA, USA. julia.pian@childrens.harvard.edu.; N=1 Collaborative, Boston, MA, USA. julia.pian@childrens.harvard.edu., Owusu N; Columbia University Vagelos College of Physicians & Surgeons, New York, NY, USA., Vitarello J; N=1 Collaborative, Boston, MA, USA.; Mila's Miracle Foundation, Boulder, CO, USA., Yan WX; N=1 Collaborative, Boston, MA, USA., Lo AW; MIT Sloan School of Management, MIT Laboratory for Financial Engineering, MIT Computer Science and Artificial Intelligence Laboratory, Cambridge, MA, USA.; Santa Fe Institute, Santa Fe, NM, USA., Yu TW; Boston Children's Hospital, Boston, MA, USA. timothy.yu@childrens.harvard.edu.; N=1 Collaborative, Boston, MA, USA. timothy.yu@childrens.harvard.edu.; Harvard Medical School, Boston, MA, USA. timothy.yu@childrens.harvard.edu.
Publikováno v:
Nature medicine [Nat Med] 2024 Jul; Vol. 30 (7), pp. 1816-1818.
Autor:
Pacanowski M; U.S. Food and Drug Administration, Center for Drug Evaluation and Research, Office of Translational Sciences, Office of Clinical Pharmacology, Silver Spring, Maryland, USA., Vitarello J; Mila's Miracle Foundation, Boulder, Colorado, USA.; N=1 Collaborative, Somerville, Massachusetts, USA.; EveryONE Medicines, Boston, Massachusetts, USA., Hyun I; Harvard Medical School, Center for Bioethics, Boston, Massachusetts, USA., Yu T; N=1 Collaborative, Somerville, Massachusetts, USA.; Division of Genetics and Genomics, Boston Children's Hospital, Boston, Massachusetts, USA.; Broad Institute of MIT and Harvard, Cambridge, Massachusetts, USA.; Harvard Medical School, Boston, Massachusetts, USA., Zineh I; U.S. Food and Drug Administration, Center for Drug Evaluation and Research, Office of Translational Sciences, Office of Clinical Pharmacology, Silver Spring, Maryland, USA.
Publikováno v:
Clinical pharmacology and therapeutics [Clin Pharmacol Ther] 2023 Nov; Vol. 114 (5), pp. 994-1001. Date of Electronic Publication: 2023 Sep 13.
Autor:
Aartsma-Rus A; Dutch Center for RNA Therapeutics, the Netherlands.; Department of Human Genetics, Leiden University Medical Center, Leiden, the Netherlands.; N = 1 Collaborative., Garanto A; Dutch Center for RNA Therapeutics, the Netherlands.; Department of Pediatrics and Department of Human Genetics, Radboud Institute for Molecular Life Sciences, Radboud University Medical Center, Nijmegen, the Netherlands., van Roon-Mom W; Dutch Center for RNA Therapeutics, the Netherlands.; Department of Human Genetics, Leiden University Medical Center, Leiden, the Netherlands., McConnell EM; N = 1 Collaborative., Suslovitch V; N = 1 Collaborative.; Department of Genetics and Genomics, Boston Children's Hospital, Boston, Massachusetts, USA., Yan WX; N = 1 Collaborative., Watts JK; RNA Therapeutics Institute, UMass Chan Medical School, Worcester, Massachusetts, USA., Yu TW; N = 1 Collaborative.; Department of Genetics and Genomics, Boston Children's Hospital, Boston, Massachusetts, USA.; Harvard Medical School, Division of Genetics and Genomics, Boston, Massachusetts, USA.
Publikováno v:
Nucleic acid therapeutics [Nucleic Acid Ther] 2023 Jan; Vol. 33 (1), pp. 17-25. Date of Electronic Publication: 2022 Dec 13.