Cargando…

Prospect of retinal gene therapy following commercialization of voretigene neparvovec-rzyl for retinal dystrophy mediated by RPE65 mutation

Detalles Bibliográficos
Autor principal:	Ameri, Hossein
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	Elsevier 2018
Materias:	Article
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5859497/ https://www.ncbi.nlm.nih.gov/pubmed/29564403 http://dx.doi.org/10.1016/j.joco.2018.01.006

Ejemplares similares

Cost-effectiveness of Voretigene Neparvovec-rzyl vs Standard Care for RPE65-Mediated Inherited Retinal Disease
por: Johnson, Scott, et al.
Publicado: (2019)

Bilateral exudative retinal detachments after subretinal gene therapy with voretigene neparvovec-rzyl for RPE65 Leber Congenital Amaurosis
por: Lidder, Alcina K., et al.
Publicado: (2023)

Retinal Pigment Epithelium Atrophy After Subretinal Voretigene Neparvovec-rzyl for RPE65-Related Disease: A 6-Month Follow-Up
por: Giansanti, Fabrizio, et al.
Publicado: (2022)

Voretigene neparvovec for inherited retinal dystrophy
Publicado: (2021)

An Economic Evaluation of Voretigene Neparvovec for the Treatment of Biallelic RPE65-Mediated Inherited Retinal Dystrophies in the UK
por: Viriato, Daniel, et al.
Publicado: (2020)

Cost Effectiveness of Voretigene Neparvovec for RPE65-Mediated Inherited Retinal Degeneration in Germany
por: Uhrmann, Matthias Fritz, et al.
Publicado: (2020)

Visual function and retinal changes after voretigene neparvovec treatment in children with biallelic RPE65-related inherited retinal dystrophy
por: Testa, Francesco, et al.
Publicado: (2022)

Voretigene Neparvovec for the Treatment of RPE65-associated Retinal Dystrophy: Consensus and Recommendations from the Korea RPE65-IRD Consensus Paper Committee
por: Han, Jinu, et al.
Publicado: (2023)

Cost-effectiveness of voretigene neparvovec in the treatment of patients with inherited retinal disease with RPE65 mutation in Switzerland
por: Bhadhuri, Arjun, et al.
Publicado: (2022)

Improved Rod Sensitivity as Assessed by Two-Color Dark-Adapted Perimetry in Patients With RPE65-Related Retinopathy Treated With Voretigene Neparvovec-rzyl
por: Ku, Cristy A., et al.
Publicado: (2023)

Outcomes and Adverse Effects of Voretigene Neparvovec Treatment for Biallelic RPE65-Mediated Inherited Retinal Dystrophies in a Cohort of Patients from a Single Center
por: Kiraly, Peter, et al.
Publicado: (2023)

Voretigene Neparvovec in Retinal Diseases: A Review of the Current Clinical Evidence
por: Gao, Jie, et al.
Publicado: (2020)

Chorioretinal atrophy following voretigene neparvovec despite the presence of fundus autofluorescence
por: Kolesnikova, Masha, et al.
Publicado: (2022)

Retinal Structure in RPE65-Associated Retinal Dystrophy
por: Kumaran, Neruban, et al.
Publicado: (2020)

Frequency of RPE65 Gene Mutation in Patients with Hereditary Retinal Dystrophy
por: Sinim Kahraman, Neslihan, et al.
Publicado: (2022)

Voretigene Neparvovec and Gene Therapy for Leber’s Congenital Amaurosis: Review of Evidence to Date
por: Padhy, Srikanta Kumar, et al.
Publicado: (2020)

Spatial and temporal resolution of the photoreceptors rescue dynamics after treatment with voretigene neparvovec
por: Stingl, Krunoslav, et al.
Publicado: (2022)

Novel mutations in RPE65 identified in consanguineous Pakistani families with retinal dystrophy
por: Kabir, Firoz, et al.
Publicado: (2013)

Long-Term Follow-Up of a Family with Retinal Dystrophy Caused by RPE65 Mutation
por: Jánossy, Ágnes, et al.
Publicado: (2023)

The interplay of environmental luminance and genetics in the retinal dystrophy induced by the dominant RPE65 mutation
por: Wu, Wenjing, et al.
Publicado: (2022)

Voretigene Neparvovec Gene Therapy in Clinical Practice: Treatment of the First Two Italian Pediatric Patients
por: Testa, Francesco, et al.
Publicado: (2021)

Epidemiology of Mutations in the 65-kDa Retinal Pigment Epithelium (RPE65) Gene-Mediated Inherited Retinal Dystrophies: A Systematic Literature Review
por: Sallum, Juliana M. F., et al.
Publicado: (2022)

Assessing the Accuracy, Quality, and Readability of Patient Accessible Online Resources Regarding Ocular Gene Therapy and Voretigene Neparvovec
por: Davuluri, Swetha, et al.
Publicado: (2021)

Validation of a Vision-Guided Mobility Assessment for RPE65-Associated Retinal Dystrophy
por: Kumaran, Neruban, et al.
Publicado: (2020)

Early onset flecked retinal dystrophy associated with new compound heterozygous RPE65 variants
por: Katagiri, Satoshi, et al.
Publicado: (2018)

Gene Therapy with Voretigene Neparvovec Improves Vision and Partially Restores Electrophysiological Function in Pre-School Children with Leber Congenital Amaurosis
por: Gerhardt, Maximilian J., et al.
Publicado: (2022)

The clinical features of retinal disease due to a dominant mutation in RPE65
por: Hull, Sarah, et al.
Publicado: (2016)

Pathogenicity Reclassification of RPE65 Missense Variants Related to Leber Congenital Amaurosis and Early-Onset Retinal Dystrophy
por: Motta, Fabiana L., et al.
Publicado: (2019)

Clinical Features and Natural History in a Cohort of Chinese Patients with RPE65-Associated Inherited Retinal Dystrophy
por: Shi, Jie, et al.
Publicado: (2021)

Long-Term Follow-Up of Retinal Degenerations Associated With LRAT Mutations and Their Comparability to Phenotypes Associated With RPE65 Mutations
por: Talib, Mays, et al.
Publicado: (2019)

The First Homozygote Mutation c.499G>T (Asp167Tyr) in the RPE65 Gene Encoding Retinoid Isomerohydrolase Causing Retinal Dystrophy
por: Bjeloš, Mirjana, et al.
Publicado: (2022)

A Comprehensive Study of the Retinal Phenotype of Rpe65-Deficient Dogs
por: Annear, Matthew J, et al.
Publicado: (2021)

Evaluation for Retinal Therapy for RPE65 Variation Assessed in hiPSC Retinal Pigment Epithelial Cells
por: Nash, Benjamin M., et al.
Publicado: (2021)

Phenotypic expansion of autosomal dominant retinitis pigmentosa associated with the D477G mutation in RPE65
por: Jauregui, Ruben, et al.
Publicado: (2020)

Differential neuroglycan C expression during retinal degeneration in Rpe65(−/−) mice
por: Escher, Pascal, et al.
Publicado: (2008)

Short-Term Outcomes of the First in Vivo Gene Therapy for RPE65-Mediated Retinitis Pigmentosa
por: Kwak, Jay Jiyong, et al.
Publicado: (2022)

Canine and Human Visual Cortex Intact and Responsive Despite Early Retinal Blindness from RPE65 Mutation
por: Aguirre, Geoffrey K, et al.
Publicado: (2007)

Neuroplasticity of the Lateral Geniculate Nucleus in Response to Retinal Gene Therapy in a Group of Patients with RPE65 Mutations
por: Ashtari, Manzar, et al.
Publicado: (2022)

RPE65-associated inherited retinal diseases: consensus recommendations for eligibility to gene therapy
por: Sodi, Andrea, et al.
Publicado: (2021)

Loss of Extracellular Signal-Regulated Kinase 1/2 in the Retinal Pigment Epithelium Leads to RPE65 Decrease and Retinal Degeneration
por: Pyakurel, Aswin, et al.
Publicado: (2017)

Cannot write session to /tmp/vufind_sessions/sess_8dn5kk0kpvhi4jhuno6daam5ls