Cargando…

Targeting ON-bipolar cells by AAV gene therapy stably reverses LRIT3-congenital stationary night blindness

Adeno-associated virus (AAV)–based gene therapies aimed at curing inherited retinal diseases to date have typically focused on photoreceptors and retinal pigmented epithelia within the relatively accessible outer retina. However, therapeutic targeting in diseases such as congenital stationary night...

Descripción completa

Detalles Bibliográficos
Autores principales:	Miyadera, Keiko, Santana, Evelyn, Roszak, Karolina, Iffrig, Sommer, Visel, Meike, Iwabe, Simone, Boyd, Ryan F., Bartoe, Joshua T., Sato, Yu, Gray, Alexa, Ripolles-Garcia, Ana, Dufour, Valérie L., Byrne, Leah C., Flannery, John G., Beltran, William A., Aguirre, Gustavo D.
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	National Academy of Sciences 2022
Materias:	Biological Sciences
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9060458/ https://www.ncbi.nlm.nih.gov/pubmed/35316139 http://dx.doi.org/10.1073/pnas.2117038119

Ejemplares similares

Genome-wide association study and whole-genome sequencing identify a deletion in LRIT3 associated with canine congenital stationary night blindness
por: Das, Rueben G., et al.
Publicado: (2019)

Lrit3 Deficient Mouse (nob6): A Novel Model of Complete Congenital Stationary Night Blindness (cCSNB)
por: Neuillé, Marion, et al.
Publicado: (2014)

scAAVengr, a transcriptome-based pipeline for quantitative ranking of engineered AAVs with single-cell resolution
por: Öztürk, Bilge E, et al.
Publicado: (2021)

A Naturally Occurring Canine Model of Autosomal Recessive Congenital Stationary Night Blindness
por: Kondo, Mineo, et al.
Publicado: (2015)

AAV-Mediated, Optogenetic Ablation of Müller Glia Leads to Structural and Functional Changes in the Mouse Retina
por: Byrne, Leah C., et al.
Publicado: (2013)

LRIT1 Modulates Adaptive Changes in Synaptic Communication of Cone Photoreceptors
por: Sarria, Ignacio, et al.
Publicado: (2018)

LRIT3 is Required for Nyctalopin Expression and Normal ON and OFF Pathway Signaling in the Retina
por: Hasan, Nazarul, et al.
Publicado: (2020)

AAV Induced Expression of Human Rod and Cone Opsin in Bipolar Cells of a Mouse Model of Retinal Degeneration
por: McClements, Michelle E., et al.
Publicado: (2021)

LRIT3 Differentially Affects Connectivity and Synaptic Transmission of Cones to ON- and OFF-Bipolar Cells
por: Neuillé, Marion, et al.
Publicado: (2017)

Assessment of Night Vision Problems in Patients with Congenital Stationary Night Blindness
por: Bijveld, Mieke M. C., et al.
Publicado: (2013)

Restoration of mGluR6 Localization Following AAV-Mediated Delivery in a Mouse Model of Congenital Stationary Night Blindness
por: Varin, Juliette, et al.
Publicado: (2021)

Substantial restoration of night vision in adult mice with congenital stationary night blindness
por: Varin, Juliette, et al.
Publicado: (2021)

Congenital Stationary Night Blindness: Clinical and Genetic Features
por: Kim, Angela H., et al.
Publicado: (2022)

Presynaptic Expression of LRIT3 Transsynaptically Organizes the Postsynaptic Glutamate Signaling Complex Containing TRPM1
por: Hasan, Nazarul, et al.
Publicado: (2019)

rAAV-mediated overexpression of TGF-β stably restructures human osteoarthritic articular cartilage in situ
por: Venkatesan, Jagadeesh K, et al.
Publicado: (2013)

Recombinant AAV-Mediated BEST1 Transfer to the Retinal Pigment Epithelium: Analysis of Serotype-Dependent Retinal Effects
por: Guziewicz, Karina E., et al.
Publicado: (2013)

AAV retinal transduction in a large animal model species: Comparison of a self-complementary AAV2/5 with a single-stranded AAV2/5 vector
por: Petersen-Jones, S.M., et al.
Publicado: (2009)

Clinical and Genetic Characteristics of Korean Congenital Stationary Night Blindness Patients
por: Kim, Hyeong-Min, et al.
Publicado: (2021)

TRPM1 mutations are associated with the complete form of congenital stationary night blindness
por: Nakamura, Makoto, et al.
Publicado: (2010)

Cav1.4 dysfunction and congenital stationary night blindness type 2
por: Koschak, Alexandra, et al.
Publicado: (2021)

Author Correction: Variabilities in retinal function and structure in a canine model of cone-rod dystrophy associated with RPGRIP1 support multigenic etiology
por: Das, Rueben G., et al.
Publicado: (2018)

Variabilities in retinal function and structure in a canine model of cone-rod dystrophy associated with RPGRIP1 support multigenic etiology
por: Das, Rueben G., et al.
Publicado: (2017)

Spectrum of Cav1.4 dysfunction in congenital stationary night blindness type 2()
por: Burtscher, Verena, et al.
Publicado: (2014)

Color vision abnormality as an initial presentation of the complete type of congenital stationary night blindness
por: Tan, Xue, et al.
Publicado: (2013)

Photoreceptor Degeneration in Two Mouse Models for Congenital Stationary Night Blindness Type 2
por: Regus-Leidig, Hanna, et al.
Publicado: (2014)

Mutations in GRM6 identified in consanguineous Pakistani families with congenital stationary night blindness
por: Naeem, Muhammad Asif, et al.
Publicado: (2015)

Long-Term Clinical Course in a Patient with Complete Congenital Stationary Night Blindness
por: Kurata, Kentaro, et al.
Publicado: (2017)

In vitro analysis of promoter activity in Müller cells
por: Geller, Scott F., et al.
Publicado: (2008)

Molecular profiling of complete congenital stationary night blindness: A pilot study on an Indian cohort
por: Malaichamy, Sivasankar, et al.
Publicado: (2014)

Tyrosine capsid-mutant AAV vectors for gene delivery to the canine retina from a subretinal or intravitreal approach
por: Mowat, FM, et al.
Publicado: (2013)

Evaluation of Lateral Spread of Transgene Expression following Subretinal AAV–Mediated Gene Delivery in Dogs
por: Bruewer, Ashlee R., et al.
Publicado: (2013)

Erratum: Molecular profiling of complete congenital stationary night blindness: A pilot study on an Indian cohort
Publicado: (2014)

Two Novel NYX Gene Mutations in the Chinese Families with X-linked Congenital Stationary Night Blindness
por: Dai, Shuzhen, et al.
Publicado: (2015)

Psychophysical measures of visual function and everyday perceptual experience in a case of congenital stationary night blindness
por: Cammack, Jocelyn, et al.
Publicado: (2016)

Congenital Stationary Night Blindness due to Novel TRPM1 Gene Mutations in a Korean Patient
por: Lee, Yun Jeong, et al.
Publicado: (2020)

Optic Atrophy and Inner Retinal Thinning in CACNA1F-Related Congenital Stationary Night Blindness
por: Leahy, Kate E, et al.
Publicado: (2021)

A New Mouse Model for Complete Congenital Stationary Night Blindness Due to Gpr179 Deficiency
por: Orhan, Elise, et al.
Publicado: (2021)

Ca(v)1.4 congenital stationary night blindness is associated with an increased rate of proteasomal degradation
por: Sadeh, Tal T., et al.
Publicado: (2023)

Mice Lacking Gpr179 with Complete Congenital Stationary Night Blindness Are a Good Model for Myopia
por: Wilmet, Baptiste, et al.
Publicado: (2022)

Retinoschisin gene therapy in photoreceptors, Müller glia, or all retinal cells in the Rs1h−/− mouse
por: Byrne, Leah C., et al.
Publicado: (2014)

Cannot write session to /tmp/vufind_sessions/sess_bkp8ej2kdma3qtkoh56k5fn2a9