Allele-Selective Thiomorpholino Antisense Oligonucleotides as a Therapeutic Approach for Fused-in-Sarcoma Amyotrophic Lateral Sclerosis.

Humans Oligonucleotides, Antisense / therapeutic use Amyotrophic Lateral Sclerosis / genetics RNA-Binding Protein FUS / genetics Alleles Fibroblasts / metabolism Gene Knockdown Techniques Morpholinos / therapeutic use
FUS allele-selective amyotrophic lateral sclerosis antisense oligonucleotides thiomorpholino

Journal

International journal of molecular sciences
ISSN: 1422-0067
Titre abrégé: Int J Mol Sci
Pays: Switzerland
ID NLM: 101092791

Informations de publication

Date de publication:
03 Aug 2024
Historique:
received: 28 06 2024
revised: 31 07 2024
accepted: 31 07 2024
medline: 10 8 2024
pubmed: 10 8 2024
entrez: 10 8 2024
Statut: epublish

Résumé

Pathogenic variations in the fused in sarcoma (

Identifiants

DOI: 10.3390/ijms25158495 PMID: 39126066
pubmed: 39126066
pii: ijms25158495
doi: 10.3390/ijms25158495
pii:
doi:

Substances chimiques

Oligonucleotides, Antisense 0
RNA-Binding Protein FUS 0
FUS protein, human 0
Morpholinos 0

Types de publication

Journal Article

Langues

eng

Sous-ensembles de citation

IM

Subventions

Organisme : Motor Neurone Disease Research Australia
ID : 2322

Auteurs

Rita Mejzini (R)

Centre for Molecular Medicine and Innovative Therapeutics, Health Futures Institute, Murdoch University, Murdoch, WA 6150, Australia.
The Perron Institute for Neurological and Translational Science, Nedlands, WA 6009, Australia.
ORCID: 0000-0003-2249-1253

Marvin H Caruthers (MH)

Department of Biochemistry, University of Colorado, Boulder, CO 80309, USA.
ORCID: 0000-0002-4931-468X

Balazs Schafer (B)

Department of Biochemistry, University of Colorado, Boulder, CO 80309, USA.

Ondrej Kostov (O)

Department of Biochemistry, University of Colorado, Boulder, CO 80309, USA.
ORCID: 0000-0002-0194-1852

Kavitha Sudheendran (K)

Department of Biochemistry, University of Colorado, Boulder, CO 80309, USA.

Marija Ciba (M)

Department of Biochemistry, University of Colorado, Boulder, CO 80309, USA.
ORCID: 0009-0008-5179-6463

Mathias Danielsen (M)

Department of Biochemistry, University of Colorado, Boulder, CO 80309, USA.
ORCID: 0000-0001-9862-3967

Steve Wilton (S)

Centre for Molecular Medicine and Innovative Therapeutics, Health Futures Institute, Murdoch University, Murdoch, WA 6150, Australia.
The Perron Institute for Neurological and Translational Science, Nedlands, WA 6009, Australia.
Centre for Neuromuscular and Neurological Disorders, University of Western Australia, Crawley, WA 6009, Australia.

Patrick Anthony Akkari (PA)

Centre for Molecular Medicine and Innovative Therapeutics, Health Futures Institute, Murdoch University, Murdoch, WA 6150, Australia.
The Perron Institute for Neurological and Translational Science, Nedlands, WA 6009, Australia.
Black Swan Pharmaceuticals, Wake Forest, NC 27587, USA.
Department of Neurology, Duke University, Durham, NC 27708, USA.

Loren L Flynn (LL)

Centre for Molecular Medicine and Innovative Therapeutics, Health Futures Institute, Murdoch University, Murdoch, WA 6150, Australia.
The Perron Institute for Neurological and Translational Science, Nedlands, WA 6009, Australia.
Centre for Neuromuscular and Neurological Disorders, University of Western Australia, Crawley, WA 6009, Australia.
Black Swan Pharmaceuticals, Wake Forest, NC 27587, USA.
ORCID: 0000-0001-5249-547X

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Classifications MeSH

questionsmedicales.fr › Eucaryotes › Animaux › Chordés › Vertébrés › Mammifères › Eutheria › Primates › Haplorhini › Catarrhini › Hominidae › Humains › Allele-Selective Thiomorpholino Antisense...
questionsmedicales.fr › Actions chimiques et utilisations › Utilisations spécialisées de produits chimiques › Produits chimiques de laboratoire › Sondes moléculaires › Sondes d'acide nucléique › Éléments antisens (génétique) › Oligonucléotides antisens › Allele-Selective Thiomorpholino Antisense...
questionsmedicales.fr › Maladies métaboliques et nutritionnelles › Maladies métaboliques › Troubles de l'homéostasie des protéines › Protéinopathies TDP-43 › Sclérose latérale amyotrophique › Allele-Selective Thiomorpholino Antisense...
questionsmedicales.fr › Acides aminés, peptides et protéines › Protéines › Nucléoprotéines › Protéines de liaison à l'ARN › Protéines à motif de reconnaissance de l'ARN › Ribonucléoprotéines nucléaires hétérogènes › Protéine FUS de liaison à l'ARN › Allele-Selective Thiomorpholino Antisense...
questionsmedicales.fr › Phénomènes génétiques › Structures génétiques › Génome › Composants de génome › Gènes › Allèles › Allele-Selective Thiomorpholino Antisense...
questionsmedicales.fr › Cellules › Cellules du tissu conjonctif › Fibroblastes › Allele-Selective Thiomorpholino Antisense...
questionsmedicales.fr › Techniques d'investigation › Techniques génétiques › Ciblage de gène › Techniques de knock-down de gènes › Allele-Selective Thiomorpholino Antisense...
questionsmedicales.fr › Acides nucléiques, nucléotides et nucléosides › Nucléotides › Polynucléotides › Oligonucléotides › Morpholinos › Allele-Selective Thiomorpholino Antisense...