Cargando…

Adeno-associated virus serotype 9 antibody seroprevalence for patients in the United States with spinal muscular atrophy

Onasemnogene abeparvovec is a recombinant adeno-associated virus serotype 9 (AAV9) vector-based gene therapy for spinal muscular atrophy (SMA). Patients with elevated titers of anti-AAV9 antibodies (AAV9-Ab) should not receive onasemnogene abeparvovec because of potential safety and efficacy implica...

Descripción completa

Detalles Bibliográficos
Autores principales:	Day, John W., Mendell, Jerry R., Burghes, Arthur H.M., van Olden, Rudolf W., Adhikary, Rishi R., Dilly, Keith W.
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	American Society of Gene & Cell Therapy 2023
Materias:	Original Article
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10562739/ https://www.ncbi.nlm.nih.gov/pubmed/37822718 http://dx.doi.org/10.1016/j.omtm.2023.101117

Ejemplares similares

Adeno-associated virus serotype 9 antibodies in patients screened for treatment with onasemnogene abeparvovec
por: Day, John W., et al.
Publicado: (2021)

Recombinant Adeno-Associated Virus Serotype 9 Gene Therapy in Spinal Muscular Atrophy
por: Kotulska, Katarzyna, et al.
Publicado: (2021)

Electrophysiological biomarkers in spinal muscular atrophy: proof of concept
por: David Arnold, W, et al.
Publicado: (2014)

Base editing rescue of spinal muscular atrophy in cells and in mice
por: Arbab, Mandana, et al.
Publicado: (2023)

Persistent neuromuscular junction transmission defects in adults with spinal muscular atrophy treated with nusinersen
por: Arnold, W David, et al.
Publicado: (2021)

SMA-EUROPE workshop report: opportunities and challenges in developing clinical trials for spinal muscular atrophy in Europe
por: Kayadjanian, Nathalie, et al.
Publicado: (2013)

What Genetics Has Told Us and How It Can Inform Future Experiments for Spinal Muscular Atrophy, a Perspective
por: Blatnik, Anton J., et al.
Publicado: (2021)

Evaluation of the orally bioavailable 4-phenylbutyrate-tethered trichostatin A analogue AR42 in models of spinal muscular atrophy
por: Lumpkin, Casey J., et al.
Publicado: (2023)

Ribonucleoprotein Assembly Defects Correlate with Spinal Muscular Atrophy Severity and Preferentially Affect a Subset of Spliceosomal snRNPs
por: Gabanella, Francesca, et al.
Publicado: (2007)

Advances and limitations for the treatment of spinal muscular atrophy
por: Day, John W., et al.
Publicado: (2022)

Plastin 3 Expression Does Not Modify Spinal Muscular Atrophy Severity in the ∆7 SMA Mouse
por: McGovern, Vicki L., et al.
Publicado: (2015)

Baseline results of the NeuroNEXT spinal muscular atrophy infant biomarker study
por: Kolb, Stephen J., et al.
Publicado: (2016)

Carrier screening for spinal muscular atrophy
por: Prior, Thomas W.
Publicado: (2008)

A transgene carrying an A2G missense mutation in the SMN gene modulates phenotypic severity in mice with severe (type I) spinal muscular atrophy
por: Monani, Umrao R., et al.
Publicado: (2003)

Intrathecal Onasemnogene Abeparvovec for Sitting, Nonambulatory Patients with Spinal Muscular Atrophy: Phase I Ascending-Dose Study (STRONG)
por: Finkel, Richard S., et al.
Publicado: (2023)

Improved Antisense Oligonucleotide Design to Suppress Aberrant SMN2 Gene Transcript Processing: Towards a Treatment for Spinal Muscular Atrophy
por: Mitrpant, Chalermchai, et al.
Publicado: (2013)

Health outcomes in spinal muscular atrophy type 1 following AVXS‐101 gene replacement therapy
por: Al‐Zaidy, Samiah, et al.
Publicado: (2018)

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

Nusinersen Treatment in Adults With Spinal Muscular Atrophy
por: Duong, Tina, et al.
Publicado: (2021)

Infectivity of adeno-associated virus serotypes in mouse testis
por: Rajasekaran, Santhanasabapathy, et al.
Publicado: (2018)

Five-Year Extension Results of the Phase 1 START Trial of Onasemnogene Abeparvovec in Spinal Muscular Atrophy
por: Mendell, Jerry R., et al.
Publicado: (2021)

Modeling Spinal Muscular Atrophy in Drosophila
por: Chang, Howard Chia-Hao, et al.
Publicado: (2008)

Nusinersen for spinal muscular atrophy
por: Wurster, Claudia D., et al.
Publicado: (2018)

Spinal Muscular Atrophy and Ependymoma
por: Albakr, Aishah, et al.
Publicado: (2017)

Risdiplam for spinal muscular atrophy
Publicado: (2022)

Spinal Muscular Atrophy: Mutations, Testing, and Clinical Relevance
por: Keinath, Melissa C, et al.
Publicado: (2021)

Impaired kidney structure and function in spinal muscular atrophy
por: Nery, Flávia C., et al.
Publicado: (2019)

Innovating spinal muscular atrophy models in the therapeutic era
por: Signoria, Ilaria, et al.
Publicado: (2023)

Clinical features of spinal and bulbar muscular atrophy
por: Rhodes, Lindsay E., et al.
Publicado: (2009)

Cardiac pathology in spinal muscular atrophy: a systematic review
por: Wijngaarde, C. A., et al.
Publicado: (2017)

Supraspinal gene transfer by intrathecal adeno-associated virus serotype 5
por: Schuster, Daniel J., et al.
Publicado: (2014)

Nerve sprouting capacity in a pharmacologically induced mouse model of spinal muscular atrophy
por: Rimer, Mendell, et al.
Publicado: (2019)

Burden of Spinal Muscular Atrophy (SMA) on Patients and Caregivers in Canada
por: McMillan, H.J., et al.
Publicado: (2021)

Androgens in spinal and bulbar muscular atrophy
Publicado: (2012)

Spinal Muscular Atrophy: The Treatment Approved
por: Tabet, Rabih, et al.
Publicado: (2017)

Advances in spinal muscular atrophy therapeutics
por: Parente, Valeria, et al.
Publicado: (2018)

Euglycemic Ketoacidosis in Spinal Muscular Atrophy
por: Stoimenis, Dimitrios, et al.
Publicado: (2019)

Metabolic Dysfunction in Spinal Muscular Atrophy
por: Deguise, Marc-Olivier, et al.
Publicado: (2021)

Update on Biomarkers in Spinal Muscular Atrophy
por: Pino, Megan G, et al.
Publicado: (2021)

Onasemnogene abeparvovec for spinal muscular atrophy
Publicado: (2022)

Cannot write session to /tmp/vufind_sessions/sess_3j1q6cns9itm4un71j9qh47ai3