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"name": "Comment diagnostiquer une maladie ciblée par des oligonucleotides antisens ?",
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"text": "Le diagnostic repose sur des tests génétiques et l'analyse de l'expression des gènes."
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"@type": "Question",
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"position": 3,
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"name": "Peut-on utiliser l'imagerie pour diagnostiquer des maladies ciblées ?",
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"@type": "Question",
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"name": "Les oligonucleotides peuvent-ils soulager des symptômes ?",
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"text": "Des effets secondaires peuvent survenir, comme des réactions au site d'injection ou des nausées."
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"@type": "Question",
"name": "Comment les symptômes évoluent-ils avec le traitement ?",
"position": 9,
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"@type": "Question",
"name": "Les symptômes sont-ils différents selon les maladies ciblées ?",
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"text": "Oui, les symptômes varient selon la maladie génétique ou le type de cancer traité."
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"name": "Les oligonucleotides antisens peuvent-ils être utilisés préventivement ?",
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"text": "Ils sont principalement utilisés pour traiter, mais des études explorent leur usage préventif."
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"name": "Comment prévenir les maladies ciblées par des oligonucleotides ?",
"position": 12,
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"name": "Y a-t-il des vaccins associés aux oligonucleotides ?",
"position": 13,
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"name": "Les conseils génétiques sont-ils importants ?",
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"name": "Peut-on prévenir les effets secondaires des oligonucleotides ?",
"position": 15,
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"text": "Des stratégies de gestion des effets secondaires peuvent être mises en place par les médecins."
}
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"name": "Comment les oligonucleotides antisens sont-ils administrés ?",
"position": 16,
"acceptedAnswer": {
"@type": "Answer",
"text": "Ils sont généralement administrés par injection sous-cutanée ou intraveineuse."
}
},
{
"@type": "Question",
"name": "Quels types de maladies sont traités avec des oligonucleotides ?",
"position": 17,
"acceptedAnswer": {
"@type": "Answer",
"text": "Ils sont utilisés pour traiter des maladies génétiques, des cancers et des infections virales."
}
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{
"@type": "Question",
"name": "Les oligonucleotides sont-ils utilisés en monothérapie ?",
"position": 18,
"acceptedAnswer": {
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"text": "Ils peuvent être utilisés seuls ou en combinaison avec d'autres traitements."
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{
"@type": "Question",
"name": "Quelle est la durée du traitement avec des oligonucleotides ?",
"position": 19,
"acceptedAnswer": {
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"text": "La durée varie selon la maladie, mais peut aller de quelques semaines à plusieurs mois."
}
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{
"@type": "Question",
"name": "Y a-t-il des protocoles spécifiques pour le traitement ?",
"position": 20,
"acceptedAnswer": {
"@type": "Answer",
"text": "Oui, des protocoles sont établis selon la maladie et la réponse du patient au traitement."
}
},
{
"@type": "Question",
"name": "Quelles complications peuvent survenir avec les oligonucleotides ?",
"position": 21,
"acceptedAnswer": {
"@type": "Answer",
"text": "Des complications peuvent inclure des réactions allergiques et des infections au site d'injection."
}
},
{
"@type": "Question",
"name": "Les oligonucleotides peuvent-ils causer des mutations ?",
"position": 22,
"acceptedAnswer": {
"@type": "Answer",
"text": "Il n'y a pas de preuves solides que les oligonucleotides causent des mutations génétiques."
}
},
{
"@type": "Question",
"name": "Comment gérer les complications liées au traitement ?",
"position": 23,
"acceptedAnswer": {
"@type": "Answer",
"text": "La gestion implique un suivi régulier et des ajustements de traitement si nécessaire."
}
},
{
"@type": "Question",
"name": "Les complications sont-elles fréquentes ?",
"position": 24,
"acceptedAnswer": {
"@type": "Answer",
"text": "Les complications sont généralement rares, mais peuvent survenir chez certains patients."
}
},
{
"@type": "Question",
"name": "Y a-t-il des risques à long terme associés aux oligonucleotides ?",
"position": 25,
"acceptedAnswer": {
"@type": "Answer",
"text": "Les études à long terme sont nécessaires pour évaluer les risques potentiels sur la santé."
}
},
{
"@type": "Question",
"name": "Quels facteurs de risque sont associés aux maladies ciblées ?",
"position": 26,
"acceptedAnswer": {
"@type": "Answer",
"text": "Les facteurs incluent des antécédents familiaux, des mutations génétiques et des expositions environnementales."
}
},
{
"@type": "Question",
"name": "L'âge influence-t-il l'efficacité des oligonucleotides ?",
"position": 27,
"acceptedAnswer": {
"@type": "Answer",
"text": "L'âge peut influencer la réponse au traitement, mais cela dépend de la maladie spécifique."
}
},
{
"@type": "Question",
"name": "Les habitudes de vie affectent-elles le traitement ?",
"position": 28,
"acceptedAnswer": {
"@type": "Answer",
"text": "Oui, des habitudes comme le tabagisme ou l'alimentation peuvent influencer l'efficacité."
}
},
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"@type": "Question",
"name": "Y a-t-il des prédispositions génétiques ?",
"position": 29,
"acceptedAnswer": {
"@type": "Answer",
"text": "Certaines mutations génétiques peuvent augmenter le risque de maladies ciblées par les oligonucleotides."
}
},
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"@type": "Question",
"name": "Les facteurs environnementaux jouent-ils un rôle ?",
"position": 30,
"acceptedAnswer": {
"@type": "Answer",
"text": "Oui, l'exposition à des toxines ou à des agents pathogènes peut augmenter le risque de maladies."
}
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Drug Metabolism and Pharmacokinetics, Research and Early Development, Cardiovascular, Renal and Metabolism (CVRM), BioPharmaceuticals R&D, AstraZeneca, 431 83 Gothenburg, Sweden.
Department of Biomedical Engineering, Linköping University, 581 85 Linköping, Sweden.
From the Washington University School of Medicine, St. Louis (T.M.M., R.C.B.); the Sean M. Healey and AMG Center for ALS, Massachusetts General Hospital, Harvard Medical School, Boston (M.E.C., S.B.), and Biogen, Cambridge (T.C., S. Chary, S. Chew, H.Z., F.W., I.N., D.G., P.S., L.F., T.A.F., S.F.) - both in Massachusetts; Montreal Neurological Institute and Hospital, Montreal (A.G.); the Sheffield Institute for Translational Neuroscience, University of Sheffield, and the National Institute for Health and Care Research Sheffield Biomedical Research Centre and Clinical Research Facility, University of Sheffield and Sheffield Teaching Hospitals NHS Foundation Trust, Sheffield (P.J.S., C.J.M.), and Biogen, Maidenhead (M.M.) - both in the United Kingdom; Aichi Medical University, Aichi, Japan (G.S.); the University of Turin, Turin, Italy (A.C.); KU Leuven, VIB Center for Brain and Disease Research, University Hospitals Leuven, Leuven, Belgium (P.V.D.); the University of Ulm, Ulm, and Deutsches Zentrum für Neurodegenerative Erkrankungen, Bonn - both in Germany (A.C.L.); Emory University, Atlanta (J.D.G.); the Neurological Institute, Columbia University Irving Medical Center, New York (J.A.A.); and the Department of Neurology, University of Miami, Miami (M.B.).
From the Washington University School of Medicine, St. Louis (T.M.M., R.C.B.); the Sean M. Healey and AMG Center for ALS, Massachusetts General Hospital, Harvard Medical School, Boston (M.E.C., S.B.), and Biogen, Cambridge (T.C., S. Chary, S. Chew, H.Z., F.W., I.N., D.G., P.S., L.F., T.A.F., S.F.) - both in Massachusetts; Montreal Neurological Institute and Hospital, Montreal (A.G.); the Sheffield Institute for Translational Neuroscience, University of Sheffield, and the National Institute for Health and Care Research Sheffield Biomedical Research Centre and Clinical Research Facility, University of Sheffield and Sheffield Teaching Hospitals NHS Foundation Trust, Sheffield (P.J.S., C.J.M.), and Biogen, Maidenhead (M.M.) - both in the United Kingdom; Aichi Medical University, Aichi, Japan (G.S.); the University of Turin, Turin, Italy (A.C.); KU Leuven, VIB Center for Brain and Disease Research, University Hospitals Leuven, Leuven, Belgium (P.V.D.); the University of Ulm, Ulm, and Deutsches Zentrum für Neurodegenerative Erkrankungen, Bonn - both in Germany (A.C.L.); Emory University, Atlanta (J.D.G.); the Neurological Institute, Columbia University Irving Medical Center, New York (J.A.A.); and the Department of Neurology, University of Miami, Miami (M.B.).
From the Washington University School of Medicine, St. Louis (T.M.M., R.C.B.); the Sean M. Healey and AMG Center for ALS, Massachusetts General Hospital, Harvard Medical School, Boston (M.E.C., S.B.), and Biogen, Cambridge (T.C., S. Chary, S. Chew, H.Z., F.W., I.N., D.G., P.S., L.F., T.A.F., S.F.) - both in Massachusetts; Montreal Neurological Institute and Hospital, Montreal (A.G.); the Sheffield Institute for Translational Neuroscience, University of Sheffield, and the National Institute for Health and Care Research Sheffield Biomedical Research Centre and Clinical Research Facility, University of Sheffield and Sheffield Teaching Hospitals NHS Foundation Trust, Sheffield (P.J.S., C.J.M.), and Biogen, Maidenhead (M.M.) - both in the United Kingdom; Aichi Medical University, Aichi, Japan (G.S.); the University of Turin, Turin, Italy (A.C.); KU Leuven, VIB Center for Brain and Disease Research, University Hospitals Leuven, Leuven, Belgium (P.V.D.); the University of Ulm, Ulm, and Deutsches Zentrum für Neurodegenerative Erkrankungen, Bonn - both in Germany (A.C.L.); Emory University, Atlanta (J.D.G.); the Neurological Institute, Columbia University Irving Medical Center, New York (J.A.A.); and the Department of Neurology, University of Miami, Miami (M.B.).
From the Washington University School of Medicine, St. Louis (T.M.M., R.C.B.); the Sean M. Healey and AMG Center for ALS, Massachusetts General Hospital, Harvard Medical School, Boston (M.E.C., S.B.), and Biogen, Cambridge (T.C., S. Chary, S. Chew, H.Z., F.W., I.N., D.G., P.S., L.F., T.A.F., S.F.) - both in Massachusetts; Montreal Neurological Institute and Hospital, Montreal (A.G.); the Sheffield Institute for Translational Neuroscience, University of Sheffield, and the National Institute for Health and Care Research Sheffield Biomedical Research Centre and Clinical Research Facility, University of Sheffield and Sheffield Teaching Hospitals NHS Foundation Trust, Sheffield (P.J.S., C.J.M.), and Biogen, Maidenhead (M.M.) - both in the United Kingdom; Aichi Medical University, Aichi, Japan (G.S.); the University of Turin, Turin, Italy (A.C.); KU Leuven, VIB Center for Brain and Disease Research, University Hospitals Leuven, Leuven, Belgium (P.V.D.); the University of Ulm, Ulm, and Deutsches Zentrum für Neurodegenerative Erkrankungen, Bonn - both in Germany (A.C.L.); Emory University, Atlanta (J.D.G.); the Neurological Institute, Columbia University Irving Medical Center, New York (J.A.A.); and the Department of Neurology, University of Miami, Miami (M.B.).
From the Washington University School of Medicine, St. Louis (T.M.M., R.C.B.); the Sean M. Healey and AMG Center for ALS, Massachusetts General Hospital, Harvard Medical School, Boston (M.E.C., S.B.), and Biogen, Cambridge (T.C., S. Chary, S. Chew, H.Z., F.W., I.N., D.G., P.S., L.F., T.A.F., S.F.) - both in Massachusetts; Montreal Neurological Institute and Hospital, Montreal (A.G.); the Sheffield Institute for Translational Neuroscience, University of Sheffield, and the National Institute for Health and Care Research Sheffield Biomedical Research Centre and Clinical Research Facility, University of Sheffield and Sheffield Teaching Hospitals NHS Foundation Trust, Sheffield (P.J.S., C.J.M.), and Biogen, Maidenhead (M.M.) - both in the United Kingdom; Aichi Medical University, Aichi, Japan (G.S.); the University of Turin, Turin, Italy (A.C.); KU Leuven, VIB Center for Brain and Disease Research, University Hospitals Leuven, Leuven, Belgium (P.V.D.); the University of Ulm, Ulm, and Deutsches Zentrum für Neurodegenerative Erkrankungen, Bonn - both in Germany (A.C.L.); Emory University, Atlanta (J.D.G.); the Neurological Institute, Columbia University Irving Medical Center, New York (J.A.A.); and the Department of Neurology, University of Miami, Miami (M.B.).
From the Washington University School of Medicine, St. Louis (T.M.M., R.C.B.); the Sean M. Healey and AMG Center for ALS, Massachusetts General Hospital, Harvard Medical School, Boston (M.E.C., S.B.), and Biogen, Cambridge (T.C., S. Chary, S. Chew, H.Z., F.W., I.N., D.G., P.S., L.F., T.A.F., S.F.) - both in Massachusetts; Montreal Neurological Institute and Hospital, Montreal (A.G.); the Sheffield Institute for Translational Neuroscience, University of Sheffield, and the National Institute for Health and Care Research Sheffield Biomedical Research Centre and Clinical Research Facility, University of Sheffield and Sheffield Teaching Hospitals NHS Foundation Trust, Sheffield (P.J.S., C.J.M.), and Biogen, Maidenhead (M.M.) - both in the United Kingdom; Aichi Medical University, Aichi, Japan (G.S.); the University of Turin, Turin, Italy (A.C.); KU Leuven, VIB Center for Brain and Disease Research, University Hospitals Leuven, Leuven, Belgium (P.V.D.); the University of Ulm, Ulm, and Deutsches Zentrum für Neurodegenerative Erkrankungen, Bonn - both in Germany (A.C.L.); Emory University, Atlanta (J.D.G.); the Neurological Institute, Columbia University Irving Medical Center, New York (J.A.A.); and the Department of Neurology, University of Miami, Miami (M.B.).
From the Washington University School of Medicine, St. Louis (T.M.M., R.C.B.); the Sean M. Healey and AMG Center for ALS, Massachusetts General Hospital, Harvard Medical School, Boston (M.E.C., S.B.), and Biogen, Cambridge (T.C., S. Chary, S. Chew, H.Z., F.W., I.N., D.G., P.S., L.F., T.A.F., S.F.) - both in Massachusetts; Montreal Neurological Institute and Hospital, Montreal (A.G.); the Sheffield Institute for Translational Neuroscience, University of Sheffield, and the National Institute for Health and Care Research Sheffield Biomedical Research Centre and Clinical Research Facility, University of Sheffield and Sheffield Teaching Hospitals NHS Foundation Trust, Sheffield (P.J.S., C.J.M.), and Biogen, Maidenhead (M.M.) - both in the United Kingdom; Aichi Medical University, Aichi, Japan (G.S.); the University of Turin, Turin, Italy (A.C.); KU Leuven, VIB Center for Brain and Disease Research, University Hospitals Leuven, Leuven, Belgium (P.V.D.); the University of Ulm, Ulm, and Deutsches Zentrum für Neurodegenerative Erkrankungen, Bonn - both in Germany (A.C.L.); Emory University, Atlanta (J.D.G.); the Neurological Institute, Columbia University Irving Medical Center, New York (J.A.A.); and the Department of Neurology, University of Miami, Miami (M.B.).
The rapid emergence of antibiotic-resistant bacteria directly contributes to a wave of untreatable infections. The lack of new drug development is an important driver of this crisis. Most antibiotics ...
Three bacterial species (...
A total of 45,628 ASOs were generated from 348 unique essential genes in...
We report the first sequencing of morpholino antisense oligonucleotides (phosphorodiamidate morpholino oligomers, PMOs) using electron capture dissociation (ECD) mass spectrometry. In this research, w...
Long noncoding RNAs (lncRNAs) play key regulatory roles in gene expression at the transcriptional level. Experimental evidence has established that a substantial fraction of lncRNA preferentially accu...
Off-target interactions between antisense oligonucleotides (ASOs) with state-of-the-art modifications and biological components still pose clinical safety liabilities. To mitigate a broad spectrum of ...
To date, no medical therapy can slow the progression of aortic stenosis. Fibrocalcific stenosis is the most frequent form in the general population and affects about 6% of the elderly population. Over...
Exosomes are a type of extracellular vesicles that contain diverse molecules and are present in our body fluids. They play a crucial role in transporting materials and transmitting signals between cel...
We describe here the design, synthesis, physicochemical properties, and hepatitis B antiviral activity of new 2'-O-alkyl ribonucleotide N3'→P5' phosphoramidate (2'-O-alkyl-NPO) and (thio)-phosphoramid...
Pharmacokinetics (PK) of antisense oligonucleotides (ASOs) is characterized by rapid distribution from plasma to tissue and slow terminal plasma elimination driven by re-distribution from tissue. Quan...
Calmodulinopathies are rare inherited arrhythmia syndromes caused by dominant heterozygous variants in...
We tested this hypothesis using human induced pluripotent stem cell-derived cardiomyocyte and mouse models of...
Human...
These results provide proof of concept that ASOs targeting individual calmodulin genes are potentially effective and safe therapies for calmodulinopathies....