Cargando…

Ocular and systemic safety of a recombinant AAV8 vector for X-linked retinoschisis gene therapy: GLP studies in rabbits and Rs1-KO mice

X-linked retinoschisis (XLRS) is a retinal disease caused by mutations in the gene encoding the protein retinoschisin (RS1) and is one of the most common causes of macular degeneration in young men. Our therapeutic approach for XLRS is based on the administration of AAV8-scRS/IRBPhRS, an adeno-assoc...

Descripción completa

Detalles Bibliográficos
Autores principales:	Marangoni, Dario, Bush, Ronald A, Zeng, Yong, Wei, Lisa L, Ziccardi, Lucia, Vijayasarathy, Camasamudram, Bartoe, Joshua T, Palyada, Kiran, Santos, Maria, Hiriyanna, Suja, Wu, Zhijian, Colosi, Peter, Sieving, Paul A
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	Nature Publishing Group 2016
Materias:	Article
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5008245/ https://www.ncbi.nlm.nih.gov/pubmed/27626041 http://dx.doi.org/10.1038/mtm.2016.11

Ejemplares similares

Genetic Rescue of X-Linked Retinoschisis Mouse (Rs1(−/y)) Retina Induces Quiescence of the Retinal Microglial Inflammatory State Following AAV8-RS1 Gene Transfer and Identifies Gene Networks Underlying Retinal Recovery
por: Vijayasarathy, Camasamudram, et al.
Publicado: (2021)

Rearing Light Intensity Affects Inner Retinal Pathology in a Mouse Model of X-Linked Retinoschisis but Does Not Alter Gene Therapy Outcome
por: Marangoni, Dario, et al.
Publicado: (2017)

Targeted Expression of Retinoschisin by Retinal Bipolar Cells in XLRS Promotes Resolution of Retinoschisis Cysts Sans RS1 From Photoreceptors
por: Vijayasarathy, Camasamudram, et al.
Publicado: (2022)

Rs1h(−/y) exon 3-del rat model of X-linked retinoschisis with early onset and rapid phenotype is rescued by RS1 supplementation
por: Zeng, Yong, et al.
Publicado: (2021)

Retinal Structure and Gene Therapy Outcome in Retinoschisin-Deficient Mice Assessed by Spectral-Domain Optical Coherence Tomography
por: Zeng, Yong, et al.
Publicado: (2016)

NADPH Oxidase Contributes to Photoreceptor Degeneration in Constitutively Active RAC1 Mice
por: Song, Hongman, et al.
Publicado: (2016)

Trans-Ocular Electric Current In Vivo Enhances AAV-Mediated Retinal Transduction in Large Animal Eye After Intravitreal Vector Administration
por: Song, Hongman, et al.
Publicado: (2020)

Characterization of novel RS1 exonic deletions in juvenile X-linked retinoschisis
por: D’Souza, Leera, et al.
Publicado: (2013)

AAV2/4-RS1 gene therapy in the retinoschisin knockout mouse model of X-linked retinoschisis
por: Scruggs, Brittni A., et al.
Publicado: (2022)

Nrl knockdown by AAV-delivered CRISPR/Cas9 prevents retinal degeneration in mice
por: Yu, Wenhan, et al.
Publicado: (2017)

Cryo-EM of retinoschisin branched networks suggests an intercellular adhesive scaffold in the retina
por: Heymann, J. Bernard, et al.
Publicado: (2019)

Analysis of Anatomic and Functional Measures in X-Linked Retinoschisis
por: Cukras, Catherine A., et al.
Publicado: (2018)

Trans-ocular Electric Current In Vivo Enhances AAV-Mediated Retinal Gene Transduction after Intravitreal Vector Administration
por: Song, Hongman, et al.
Publicado: (2018)

Cog-Wheel Octameric Structure of RS1, the Discoidin Domain Containing Retinal Protein Associated with X-Linked Retinoschisis
por: Bush, Martin, et al.
Publicado: (2016)

Novel RS1 mutations associated with X-linked juvenile retinoschisis
por: YI, JUNHUI, et al.
Publicado: (2012)

“Para-retinal” Vector Administration into the Deep Vitreous Enhances Retinal Transgene Expression
por: Zeng, Yong, et al.
Publicado: (2020)

Bilateral foveal retinoschisis accompanying unilateral peripheral retinoschisis
por: Kocak, Nilufer, et al.
Publicado: (2014)

Bullous retinoschisis
por: Handa, Sabia, et al.
Publicado: (2019)

CRX controls retinal expression of the X-linked juvenile retinoschisis (RS1) gene
por: Langmann, Thomas, et al.
Publicado: (2008)

Novel mutations in the RS1 gene in Japanese patients with X-linked congenital retinoschisis
por: Kondo, Hiroyuki, et al.
Publicado: (2019)

Clinical findings and RS1 genotype in 90 Chinese families with X-linked retinoschisis
por: Chen, Chunjie, et al.
Publicado: (2020)

Characterization and AAV-mediated CRB gene augmentation in human-derived CRB1(KO) and CRB1(KO)CRB2(+/−) retinal organoids
por: Boon, Nanda, et al.
Publicado: (2023)

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)

Rétinoschisis maculaire bilatéral associé à un rétinoschisis périphérique unilatéral
por: Oummad, Hanane, et al.
Publicado: (2017)

Novel mutations of the RS1 gene in a cohort of Chinese families with X-linked retinoschisis
por: Chen, Jieqiong, et al.
Publicado: (2014)

Four novel RS1 gene mutations in Polish patients with X-linked juvenile retinoschisis
por: Skorczyk, Anna, et al.
Publicado: (2012)

Clinical features of a toddler with bilateral bullous retinoschisis with a novel RS1 mutation
por: Katagiri, Satoshi, et al.
Publicado: (2016)

Multimodal imaging in a pedigree of X-linked Retinoschisis with a novel RS1 variant
por: Stephenson, Kirk, et al.
Publicado: (2018)

Neovascularization of the iris in retinoschisis
por: Slean, Geraldine R., et al.
Publicado: (2017)

Traumatic Macular Retinoschisis
por: Tripathy, Koushik
Publicado: (2018)

Multimodal imaging in retinoschisis
por: Poornachandra, B., et al.
Publicado: (2019)

X-linked retinoschisis
por: Dubey, Devashish, et al.
Publicado: (2020)

Middle-Inner Macular Layers Dysfunction in a Case of Stellate Foveomacular Retinoschisis Detected by Abnormal Multifocal Photopic Negative Response Recordings
por: Barbano, Lucilla, et al.
Publicado: (2022)

Genetic variations in the hotspot region of RS1 gene in Indian patients with juvenile X-linked retinoschisis
por: Suganthalakshmi, Balasubbu, et al.
Publicado: (2007)

A novel mutation in the RS1 gene in a Chinese family with X-linked congenital retinoschisis
por: Zhang, Na, et al.
Publicado: (2021)

Whole-exome sequencing identifies an RS1 variant in a Chinese family with X-linked retinoschisis
por: Chen, Doudou, et al.
Publicado: (2021)

A novel mutation in RS1 and clinical manifestations in a Chinese twin family with congenital retinoschisis
por: Wang, Xiao-Fang, et al.
Publicado: (2022)

Rd9 Is a Naturally Occurring Mouse Model of a Common Form of Retinitis Pigmentosa Caused by Mutations in RPGR-ORF15
por: Thompson, Debra A., et al.
Publicado: (2012)

AAV1.NT‐3 gene therapy in the SOD1KO mouse model of accelerated sarcopenia
por: Tong, Lingying, et al.
Publicado: (2023)

Inhibition of pathological brain angiogenesis through systemic delivery of AAV vector expressing soluble FLT1
por: Shen, Fanxia, et al.
Publicado: (2015)

Cannot write session to /tmp/vufind_sessions/sess_1psofqkdos7u5h50rvtepf3eoh