Cargando…

Antibody-oligonucleotide conjugate achieves CNS delivery in animal models for spinal muscular atrophy

Antisense oligonucleotides (ASOs) have emerged as one of the most innovative new genetic drug modalities. However, their high molecular weight limits their bioavailability for otherwise-treatable neurological disorders. We investigated conjugation of ASOs to an antibody against the murine transferri...

Descripción completa

Detalles Bibliográficos
Autores principales:	Hammond, Suzan M., Abendroth, Frank, Goli, Larissa, Stoodley, Jessica, Burrell, Matthew, Thom, George, Gurrell, Ian, Ahlskog, Nina, Gait, Michael J., Wood, Matthew J.A., Webster, Carl I.
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	American Society for Clinical Investigation 2022
Materias:	Research Article
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7614086/ https://www.ncbi.nlm.nih.gov/pubmed/36346674 http://dx.doi.org/10.1172/jci.insight.154142

Ejemplares similares

Identification of a Peptide for Systemic Brain Delivery of a Morpholino Oligonucleotide in Mouse Models of Spinal Muscular Atrophy
por: Shabanpoor, Fazel, et al.
Publicado: (2017)

Combining multiomics and drug perturbation profiles to identify muscle-specific treatments for spinal muscular atrophy
por: Meijboom, Katharina E., et al.
Publicado: (2021)

Cell-Penetrating Peptide Conjugates of Steric Blocking Oligonucleotides as Therapeutics for Neuromuscular Diseases from a Historical Perspective to Current Prospects of Treatment
por: Gait, Michael J., et al.
Publicado: (2019)

Pip6-PMO, A New Generation of Peptide-oligonucleotide Conjugates With Improved Cardiac Exon Skipping Activity for DMD Treatment
por: Betts, Corinne, et al.
Publicado: (2012)

Bi-specific splice-switching PMO oligonucleotides conjugated via a single peptide active in a mouse model of Duchenne muscular dystrophy
por: Shabanpoor, Fazel, et al.
Publicado: (2015)

Muscle overexpression of Klf15 via an AAV8-Spc5-12 construct does not provide benefits in spinal muscular atrophy mice
por: Ahlskog, Nina, et al.
Publicado: (2020)

Enhanced delivery of IL-1 receptor antagonist to the central nervous system as a novel anti–transferrin receptor-IL-1RA fusion reverses neuropathic mechanical hypersensitivity
por: Webster, Carl I., et al.
Publicado: (2016)

A Short Antisense Oligonucleotide Ameliorates Symptoms of Severe Mouse Models of Spinal Muscular Atrophy
por: Keil, Jeffrey M, et al.
Publicado: (2014)

Development of a general methodology for labelling peptide–morpholino oligonucleotide conjugates using alkyne–azide click chemistry
por: Shabanpoor, Fazel, et al.
Publicado: (2013)

Interventions Targeting Glucocorticoid-Krüppel-like Factor 15-Branched-Chain Amino Acid Signaling Improve Disease Phenotypes in Spinal Muscular Atrophy Mice
por: Walter, Lisa M., et al.
Publicado: (2018)

Cell penetrating peptide conjugates of steric block oligonucleotides()
por: Lebleu, Bernard, et al.
Publicado: (2008)

Nanocarriers for Delivery of Oligonucleotides to the CNS
por: Male, David, et al.
Publicado: (2022)

Myostatin inhibition in combination with antisense oligonucleotide therapy improves outcomes in spinal muscular atrophy
por: Zhou, Haiyan, et al.
Publicado: (2020)

Dysregulation of Tweak and Fn14 in skeletal muscle of spinal muscular atrophy mice
por: Meijboom, Katharina E., et al.
Publicado: (2022)

Peptide Nanoparticle Delivery of Charge-Neutral Splice-Switching Morpholino Oligonucleotides
por: Järver, Peter, et al.
Publicado: (2015)

Light modulation ameliorates expression of circadian genes and disease progression in spinal muscular atrophy mice
por: Walter, Lisa M, et al.
Publicado: (2018)

Synthesis and Splice-Redirecting Activity of Branched, Arginine-Rich Peptide Dendrimer Conjugates of Peptide Nucleic Acid Oligonucleotides
por: Saleh, Amer F., et al.
Publicado: (2010)

Targeting the 5′ untranslated region of SMN2 as a therapeutic strategy for spinal muscular atrophy
por: Winkelsas, Audrey M., et al.
Publicado: (2021)

Histopathological Defects in Intestine in Severe Spinal Muscular Atrophy Mice Are Improved by Systemic Antisense Oligonucleotide Treatment
por: Sintusek, Palittiya, et al.
Publicado: (2016)

A Semi‐Mechanistic Population Pharmacokinetic Model of Nusinersen: An Antisense Oligonucleotide for the Treatment of Spinal Muscular Atrophy
por: Biliouris, Konstantinos, et al.
Publicado: (2018)

Cerebrospinal Fluid Parameters in Antisense Oligonucleotide-Treated Adult 5q-Spinal Muscular Atrophy Patients
por: Müschen, Lars Hendrik, et al.
Publicado: (2021)

Immortalized Canine Dystrophic Myoblast Cell Lines for Development of Peptide-Conjugated Splice-Switching Oligonucleotides
por: Tone, Yuichiro, et al.
Publicado: (2021)

Synthesis, cellular uptake and HIV-1 Tat-dependent trans-activation inhibition activity of oligonucleotide analogues disulphide-conjugated to cell-penetrating peptides
por: Turner, John J., et al.
Publicado: (2005)

Peptide-conjugated oligonucleotides evoke long-lasting myotonic dystrophy correction in patient-derived cells and mice
por: Klein, Arnaud F., et al.
Publicado: (2019)

Altered Levels of MicroRNA-9, -206, and -132 in Spinal Muscular Atrophy and Their Response to Antisense Oligonucleotide Therapy
por: Catapano, Francesco, et al.
Publicado: (2016)

Antisense Oligonucleotides Conjugated with Lipophilic Compounds: Synthesis and In Vitro Evaluation of Exon Skipping in Duchenne Muscular Dystrophy
por: Marchesi, Elena, et al.
Publicado: (2022)

Splice-Switching Therapy for Spinal Muscular Atrophy
por: Meijboom, Katharina E., et al.
Publicado: (2017)

Development of Multiexon Skipping Antisense Oligonucleotide Therapy for Duchenne Muscular Dystrophy
por: Aoki, Yoshitsugu, et al.
Publicado: (2013)

Peptide-mediated Cell and In Vivo Delivery of Antisense Oligonucleotides and siRNA
por: Järver, Peter, et al.
Publicado: (2012)

Spinal muscular atrophy
por: D'Amico, Adele, et al.
Publicado: (2011)

Progressive Muscular Atrophy
por: Omar, A.
Publicado: (1947)

Progressive Muscular Atrophy
por: Omar, A.
Publicado: (1947)

Progressive Muscular Atrophy
por: Omer, M. A.
Publicado: (1949)

Muscular Atrophy and Sclerodermia
por: Nixon, J. A.
Publicado: (1903)

Progressive Muscular Atrophy
Publicado: (1874)

Muscular Atrophy and Sclerodermia
Publicado: (1904)

Acute Muscular Atrophy
por: Haddon, John
Publicado: (1870)

Case of Muscular Atrophy
por: Kersey, V.
Publicado: (1871)

Comment on: “Myostatin inhibition in combination with antisense oligonucleotide therapy improves outcomes in spinal muscular atrophy” by Zhou et al.
por: Chen, Tai‐Heng
Publicado: (2020)

High Concentration or Combined Treatment of Antisense Oligonucleotides for Spinal Muscular Atrophy Perturbed SMN2 Splicing in Patient Fibroblasts
por: Wijaya, Yogik Onky Silvana, et al.
Publicado: (2022)

Cannot write session to /tmp/vufind_sessions/sess_psofv54big3rgr8b1cggsr75j5