Cargando…

Adeno-associated virus serotype 9 antibodies in patients screened for treatment with onasemnogene abeparvovec

Spinal muscular atrophy is a progressive, recessively inherited monogenic neurologic disease, the genetic root cause of which is the absence of a functional survival motor neuron 1 gene. Onasemnogene abeparvovec (formerly AVXS-101) is an adeno-associated virus serotype 9 vector-based gene therapy th...

Descripción completa

Detalles Bibliográficos
Autores principales:	Day, John W., Finkel, Richard S., Mercuri, Eugenio, Swoboda, Kathryn J., Menier, Melissa, van Olden, Rudolf, Tauscher-Wisniewski, Sitra, Mendell, Jerry R.
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	American Society of Gene & Cell Therapy 2021
Materias:	Original Article
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7973120/ https://www.ncbi.nlm.nih.gov/pubmed/33768131 http://dx.doi.org/10.1016/j.omtm.2021.02.014

Ejemplares similares

Clinical Trial and Postmarketing Safety of Onasemnogene Abeparvovec Therapy
por: Day, John W., et al.
Publicado: (2021)

Correction to: Clinical Trial and Postmarketing Safety of Onasemnogene Abeparvovec Therapy
por: Day, John W., et al.
Publicado: (2021)

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)

Onasemnogene abeparvovec for presymptomatic infants with three copies of SMN2 at risk for spinal muscular atrophy: the Phase III SPR1NT trial
por: Strauss, Kevin A., et al.
Publicado: (2022)

Onasemnogene abeparvovec for spinal muscular atrophy
Publicado: (2022)

Onasemnogene abeparvovec for presymptomatic infants with two copies of SMN2 at risk for spinal muscular atrophy type 1: the Phase III SPR1NT trial
por: Strauss, Kevin A., et al.
Publicado: (2022)

Onasemnogene Abeparvovec for Spinal Muscular Atrophy: The Costlier Drug Ever
por: Mahajan, Rajiv
Publicado: (2019)

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)

Onasemnogene abeparvovec in spinal muscular atrophy: an Australian experience of safety and efficacy
por: D'Silva, Arlene M., et al.
Publicado: (2022)

Adeno-associated virus serotype 9 antibody seroprevalence for patients in the United States with spinal muscular atrophy
por: Day, John W., et al.
Publicado: (2023)

Combination Therapy with Nusinersen and Onasemnogene Abeparvovec-xioi in Spinal Muscular Atrophy Type I
por: Mirea, Andrada, et al.
Publicado: (2021)

Ultraexpensive gene therapies, industry interests and the right to health: the case of onasemnogene abeparvovec in Brazil
por: Ivama-Brummell, Adriana Mitsue, et al.
Publicado: (2022)

Single-Dose Intrathecal Dorsal Root Ganglia Toxicity of Onasemnogene Abeparvovec in Cynomolgus Monkeys
por: Tukov, Francis Fonyuy, et al.
Publicado: (2022)

Expert recommendations and clinical considerations in the use of onasemnogene abeparvovec gene therapy for spinal muscular atrophy
por: Kichula, Elizabeth A., et al.
Publicado: (2021)

Expanding the Availability of Onasemnogene Abeparvovec to Older Patients: The Evolving Treatment Landscape for Spinal Muscular Atrophy
por: René, Charlotte A., et al.
Publicado: (2023)

Onasemnogene abeparvovec in spinal muscular atrophy: predictors of efficacy and safety in naïve patients with spinal muscular atrophy and following switch from other therapies
por: Pane, Marika, et al.
Publicado: (2023)

Safety Monitoring of Gene Therapy for Spinal Muscular Atrophy with Onasemnogene Abeparvovec –A Single Centre Experience
por: Friese, Johannes, et al.
Publicado: (2021)

Treatment of spinal muscular atrophy with Onasemnogene Abeparvovec in Switzerland: a prospective observational case series study
por: Stettner, Georg M., et al.
Publicado: (2023)

Review of cardiac safety in onasemnogene abeparvovec gene replacement therapy: translation from preclinical to clinical findings
por: Chand, Deepa H., et al.
Publicado: (2023)

Switching therapies: safety profile of Onasemnogene abeparvovec-xioi in a SMA1 patient previously treated with Risdiplam
por: Tosi, Michele, et al.
Publicado: (2022)

Onasemnogene abeparvovec gene replacement therapy for the treatment of spinal muscular atrophy: a real-world observational study
por: Bitetti, Ilaria, et al.
Publicado: (2022)

Patient and Caregiver Outcomes After Onasemnogene Abeparvovec Treatment: Findings from the Cure SMA 2021 Membership Survey
por: Toro, Walter, et al.
Publicado: (2023)

Gene Therapy for Spinal Muscular Atrophy (SMA): A Review of Current Challenges and Safety Considerations for Onasemnogene Abeparvovec (Zolgensma)
por: Ogbonmide, Tolu, et al.
Publicado: (2023)

Erratum to: Patients with Spinal Muscular Atrophy Type 1 Achieve and Maintain Bulbar Function Following Onasemnogene Abeparvovec Treatment
por: McGrattan, Katlyn E., et al.
Publicado: (2023)

Patients with Spinal Muscular Atrophy Type 1 Achieve and Maintain Bulbar Function Following Onasemnogene Abeparvovec Treatment(1)
por: McGrattan, Katlyn E., et al.
Publicado: (2023)

Changes in electrophysiological findings of spinal muscular atrophy type I after the administration of nusinersen and onasemnogene abeparvovec: two case reports
por: Mizuno, Tomoko, et al.
Publicado: (2023)

Early Development of Spinal Deformities in Children Severely Affected with Spinal Muscular Atrophy after Gene Therapy with Onasemnogene Abeparvovec—Preliminary Results
por: Soini, Venla, et al.
Publicado: (2023)

An updated cost-utility model for onasemnogene abeparvovec (Zolgensma®) in spinal muscular atrophy type 1 patients and comparison with evaluation by the Institute for Clinical and Effectiveness Review (ICER)
por: Dean, Rebecca, et al.
Publicado: (2021)

Adeno-associated virus serotype-9 efficiently transduces the retinal outer plexiform layer
por: Lei, Bo, et al.
Publicado: (2009)

Differential myofiber-type transduction preference of adeno-associated virus serotypes 6 and 9
por: Riaz, Muhammad, et al.
Publicado: (2015)

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

Biodistribution of adeno-associated virus serotype 9 (AAV9) vector after intrathecal and intravenous delivery in mouse
por: Schuster, Daniel J., et al.
Publicado: (2014)

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

Adeno-associated virus serotypes 9 and rh10 mediate strong neuronal transduction of the dog brain
por: Swain, G P, et al.
Publicado: (2014)

Adeno-associated virus serotype 9 efficiently targets ischemic skeletal muscle following systemic delivery
por: Katwal, Arabindra B., et al.
Publicado: (2013)

Cell-penetrating peptides enhance the transduction of adeno-associated virus serotype 9 in the central nervous system
por: Meng, Yuan, et al.
Publicado: (2021)

Cell-penetrating peptides enhance the transduction of adeno-associated virus serotype 9 in the central nervous system
por: Meng, Yuan, et al.
Publicado: (2022)

Axonal transport of adeno-associated viral vectors is serotype-dependent
por: Salegio, Ernesto A., et al.
Publicado: (2012)

Adeno-associated virus serotype 9 mediated vascular endothelial growth factor gene overexpression in mdx mice
por: Song, Xueqin, et al.
Publicado: (2018)

Efficacy of Adeno-Associated Virus Serotype 9-Mediated Gene Therapy for AB-Variant GM2 Gangliosidosis
por: Vyas, Meera, et al.
Publicado: (2023)

Cannot write session to /tmp/vufind_sessions/sess_tbad667pbhnc3qd2r738fcm68p