Cargando…

Dual Supramolecular Nanoparticle Vectors Enable CRISPR/Cas9‐Mediated Knockin of Retinoschisin 1 Gene—A Potential Nonviral Therapeutic Solution for X‐Linked Juvenile Retinoschisis

The homology‐independent targeted integration (HITI) strategy enables effective CRISPR/Cas9‐mediated knockin of therapeutic genes in nondividing cells in vivo, promising general therapeutic solutions for treating genetic diseases like X‐linked juvenile retinoschisis. Herein, supramolecular nanoparti...

Descripción completa

Detalles Bibliográficos
Autores principales:	Chou, Shih‐Jie, Yang, Peng, Ban, Qian, Yang, Yi‐Ping, Wang, Mong‐Lien, Chien, Chian‐Shiu, Chen, Shih‐Jen, Sun, Na, Zhu, Yazhen, Liu, Hongtao, Hui, Wenqiao, Lin, Tai‐Chi, Wang, Fang, Zhang, Ryan Yue, Nguyen, Viet Q., Liu, Wenfei, Chen, Mengxiang, Jonas, Steve J., Weiss, Paul S., Tseng, Hsian‐Rong, Chiou, Shih‐Hwa
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	John Wiley and Sons Inc. 2020
Materias:	Communications
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7237855/ https://www.ncbi.nlm.nih.gov/pubmed/32440478 http://dx.doi.org/10.1002/advs.201903432

Ejemplares similares

Gene Therapy: Dual Supramolecular Nanoparticle Vectors Enable CRISPR/Cas9‐Mediated Knockin of Retinoschisin 1 Gene—A Potential Nonviral Therapeutic Solution for X‐Linked Juvenile Retinoschisis (Adv. Sci. 10/2020)
por: Chou, Shih‐Jie, et al.
Publicado: (2020)

Supramolecular nanosubstrate–mediated delivery system enables CRISPR-Cas9 knockin of hemoglobin beta gene for hemoglobinopathies
por: Yang, Peng, et al.
Publicado: (2020)

Structural analysis of X-linked retinoschisis mutations reveals distinct classes which differentially effect retinoschisin function
por: Ramsay, Ewan P., et al.
Publicado: (2016)

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

The X‐linked juvenile retinoschisis protein retinoschisin is a novel regulator of mitogen‐activated protein kinase signalling and apoptosis in the retina
por: Plössl, Karolina, et al.
Publicado: (2016)

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)

Morphological and Molecular Defects in Human Three-Dimensional Retinal Organoid Model of X-Linked Juvenile Retinoschisis
por: Huang, Kang-Chieh, et al.
Publicado: (2019)

Generation of Functional Dopaminergic Neurons from Reprogramming Fibroblasts by Nonviral-based Mesoporous Silica Nanoparticles
por: Chang, Jen-Hsuan, et al.
Publicado: (2018)

Retinoschisin is linked to retinal Na/K-ATPase signaling and localization
por: Plössl, Karolina, et al.
Publicado: (2017)

Identification of the retinoschisin-binding site on the retinal Na/K-ATPase
por: Plössl, Karolina, et al.
Publicado: (2019)

Retinoschisin and Cardiac Glycoside Crosstalk at the Retinal Na/K-ATPase
por: Schmid, Verena, et al.
Publicado: (2020)

Cisplatin-selected resistance is associated with increased motility and stem-like properties via activation of STAT3/Snail axis in atypical teratoid/rhabdoid tumor cells
por: Liu, Wei-Hsiu, et al.
Publicado: (2015)

Generation of GLA-Knockout Human Embryonic Stem Cell Lines to Model Autophagic Dysfunction and Exosome Secretion in Fabry Disease-Associated Hypertrophic Cardiomyopathy
por: Song, Hui-Yung, et al.
Publicado: (2019)

Targeting cancer stem cells: emerging role of Nanog transcription factor
por: Wang, Mong-Lien, et al.
Publicado: (2013)

Retinoschisin Facilitates the Function of L-Type Voltage-Gated Calcium Channels
por: Shi, Liheng, et al.
Publicado: (2017)

A Review of SARS-CoV-2 and the Ongoing Clinical Trials
por: Tu, Yung-Fang, et al.
Publicado: (2020)

Nonviral gene delivery system
por: Yang, Ningning
Publicado: (2012)

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

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

Expression of Endogenous Angiotensin-Converting Enzyme 2 in Human Induced Pluripotent Stem Cell-Derived Retinal Organoids
por: Ahmad Mulyadi Lai, Henkie Isahwan, et al.
Publicado: (2021)

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

Using CRISPR/Cas9-Mediated GLA Gene Knockout as an In Vitro Drug Screening Model for Fabry Disease
por: Song, Hui-Yung, et al.
Publicado: (2016)

Nonviral Interferon Inducers
por: Merigan, Thomas C., et al.
Publicado: (1970)

Highlight of Immune Pathogenic Response and Hematopathologic Effect in SARS-CoV, MERS-CoV, and SARS-Cov-2 Infection
por: Liang, Yanwen, et al.
Publicado: (2020)

Circular RNA hsa_circ_0000190 Facilitates the Tumorigenesis and Immune Evasion by Upregulating the Expression of Soluble PD-L1 in Non-Small-Cell Lung Cancer
por: Luo, Yung-Hung, et al.
Publicado: (2021)

Regulation of Retinoschisin Secretion in Weri-Rb1 Cells by the F-Actin and Microtubule Cytoskeleton
por: Kitamura, Eiko, et al.
Publicado: (2011)

Ash2l interacts with Oct4-stemness circuitry to promote super-enhancer-driven pluripotency network
por: Tsai, Ping-Hsing, et al.
Publicado: (2019)

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

Impaired ROS Scavenging System in Human Induced Pluripotent Stem Cells Generated from Patients with MERRF Syndrome
por: Chou, Shih-Jie, et al.
Publicado: (2016)

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)

Truncation of retinoschisin protein associated with a novel splice site mutation in the RS1 gene
por: Lesch, Balázs, et al.
Publicado: (2008)

C-terminal tensin-like (CTEN) knockin alleviates cystic kidney defects in Tensin-1 knockout mice
por: Chiu, Chun-Lung, et al.
Publicado: (2022)

An overview of viral and nonviral delivery systems for microRNA
por: Yang, Ningning
Publicado: (2015)

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

Role of dipeptidyl peptidase-4 inhibitors in patients with diabetes infected with coronavirus-19
por: Chen, Chun-Fan, et al.
Publicado: (2020)

Oncostatin M suppresses metastasis of lung adenocarcinoma by inhibiting SLUG expression through coordination of STATs and PIASs signalings
por: Pan, Chih-Ming, et al.
Publicado: (2016)

Protective effect of metformin against retinal vein occlusions in diabetes mellitus – A nationwide population-based study
por: Lin, Tai-Chi, et al.
Publicado: (2017)

Cannot write session to /tmp/vufind_sessions/sess_93t37gvtjvula0njfgmile5ro0