Cargando…

Induced Pluripotent Stem Cells and Genome-Editing Tools in Determining Gene Function and Therapy for Inherited Retinal Disorders

Inherited retinal disorders (IRDs) affect millions of people worldwide and are a major cause of irreversible blindness. Therapies based on drugs, gene augmentation or transplantation approaches have been widely investigated and proposed. Among gene therapies for retinal degenerative diseases, the fa...

Descripción completa

Detalles Bibliográficos
Autores principales:	Benati, Daniela, Leung, Amy, Perdigao, Pedro, Toulis, Vasileios, van der Spuy, Jacqueline, Recchia, Alessandra
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	MDPI 2022
Materias:	Review
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9735568/ https://www.ncbi.nlm.nih.gov/pubmed/36499601 http://dx.doi.org/10.3390/ijms232315276

Ejemplares similares

On the Wrong Track: Alterations of Ciliary Transport in Inherited Retinal Dystrophies
por: Sánchez-Bellver, Laura, et al.
Publicado: (2021)

Retinal Organoids from an AIPL1 CRISPR/Cas9 Knockout Cell Line Successfully Recapitulate the Molecular Features of LCA4 Disease
por: Perdigão, Pedro R. L., et al.
Publicado: (2023)

Allele-specific editing ameliorates dominant retinitis pigmentosa in a transgenic mouse model
por: Patrizi, Clarissa, et al.
Publicado: (2021)

New Editing Tools for Gene Therapy in Inherited Retinal Dystrophies
por: Pulman, Juliette, et al.
Publicado: (2022)

Prime Editing for Inherited Retinal Diseases
por: da Costa, Bruna Lopes, et al.
Publicado: (2021)

In vivo Editing of the Human Mutant Rhodopsin Gene by Electroporation of Plasmid-based CRISPR/Cas9 in the Mouse Retina
por: Latella, Maria Carmela, et al.
Publicado: (2016)

The Role of Hsp90 in Retinal Proteostasis and Disease
por: Ziaka, Kalliopi, et al.
Publicado: (2022)

Future Perspectives of Prime Editing for the Treatment of Inherited Retinal Diseases
por: Hansen, Silja, et al.
Publicado: (2023)

Investigation of PTC124-mediated translational readthrough in a retinal organoid model of AIPL1-associated Leber congenital amaurosis
por: Leung, Amy, et al.
Publicado: (2022)

Attitudes of people with inherited retinal conditions toward gene editing technology
por: Hoffman‐Andrews, Lily, et al.
Publicado: (2019)

Increasing the Genetic Diagnosis Yield in Inherited Retinal Dystrophies: Assigning Pathogenicity to Novel Non-canonical Splice Site Variants
por: Toulis, Vasileios, et al.
Publicado: (2020)

Inherited cardiac diseases, pluripotent stem cells, and genome editing combined—the past, present, and future
por: van den Brink, Lettine, et al.
Publicado: (2019)

Commentary: CRISPR gene editing for inherited retinal dystrophies: Towards clinical translation
por: Bansal, Mayank, et al.
Publicado: (2022)

Application of patient-derived induced pluripotent stem cells and organoids in inherited retinal diseases
por: Liang, Yuqin, et al.
Publicado: (2023)

The Inherited Blindness Protein AIPL1 Regulates the Ubiquitin-Like FAT10 Pathway
por: Bett, John S., et al.
Publicado: (2012)

Guiding Lights in Genome Editing for Inherited Retinal Disorders: Implications for Gene and Cell Therapy
por: Sanjurjo-Soriano, Carla, et al.
Publicado: (2018)

In Vitro Gene Delivery in Retinal Pigment Epithelium Cells by Plasmid DNA-Wrapped Gold Nanoparticles
por: Trigueros, Sònia, et al.
Publicado: (2019)

Primate retinal cones express phosphorylated tau associated with neuronal degeneration yet survive in old age
por: Aboelnour, Asmaa, et al.
Publicado: (2017)

Inherited Retinal Diseases
por: Ben-Yosef, Tamar
Publicado: (2022)

Retinal imaging in inherited retinal diseases
por: Georgiou, Michalis, et al.
Publicado: (2020)

Harnessing the Potential of Human Pluripotent Stem Cells and Gene Editing for the Treatment of Retinal Degeneration
por: Ovando-Roche, Patrick, et al.
Publicado: (2017)

Programmable RNA editing with endogenous ADAR enzymes – a feasible option for the treatment of inherited retinal disease?
por: Bellingrath, Julia-Sophia, et al.
Publicado: (2023)

Nonviral base editing of KCNJ13 mutation preserves vision in a model of inherited retinal channelopathy
por: Kabra, Meha, et al.
Publicado: (2023)

CRISPR-Cas9 correction of a nonsense mutation in LCA5 rescues lebercilin expression and localization in human retinal organoids
por: Afanasyeva, Tess A.V., et al.
Publicado: (2023)

Application of CRISPR Tools for Variant Interpretation and Disease Modeling in Inherited Retinal Dystrophies
por: Fuster-García, Carla, et al.
Publicado: (2020)

 Potential of Gene Editing and Induced Pluripotent Stem Cells (iPSCs) in Treatment of Retinal Diseases 
por: Chuang, Katherine, et al.
Publicado: (2017)

Engineered Sleeping Beauty Transposon as Efficient System to Optimize Chimp Adenoviral Production
por: Baldassarri, Samantha, et al.
Publicado: (2022)

INHERITED RETINAL DYSTROPHY IN THE RAT
por: Dowling, John E., et al.
Publicado: (1962)

Approach to inherited retinal diseases
por: Ratra, Dhanashree, et al.
Publicado: (2022)

Genome Editing for the Understanding and Treatment of Inherited Cardiomyopathies
por: Nguyen, Quynh, et al.
Publicado: (2020)

In vivo base editing rescues cone photoreceptors in a mouse model of early-onset inherited retinal degeneration
por: Choi, Elliot H., et al.
Publicado: (2022)

Utf1 contributes to intergenerational epigenetic inheritance of pluripotency
por: Bao, Qiuye, et al.
Publicado: (2017)

Epigenetic inheritance is gated by naïve pluripotency and Dppa2
por: Carlini, Valentina, et al.
Publicado: (2022)

Retinal Inflammation, Cell Death and Inherited Retinal Dystrophies
por: Olivares-González, Lorena, et al.
Publicado: (2021)

IROme, a New High-Throughput Molecular Tool for the Diagnosis of Inherited Retinal Dystrophies
por: Schorderet, Daniel F., et al.
Publicado: (2013)

Global spectrum of USH2A mutation in inherited retinal dystrophies: Prompt message for development of base editing therapy
por: Su, Bing-Nan, et al.
Publicado: (2022)

Gene Editing for Inherited Red Blood Cell Diseases
por: Quintana-Bustamante, Oscar, et al.
Publicado: (2022)

Systemic inflammation induced by lipopolysaccharide aggravates inherited retinal dystrophy
por: Noailles, Agustina, et al.
Publicado: (2018)

Retinal Oxygenation in Inherited Diseases of the Retina
por: Türksever, Cengiz, et al.
Publicado: (2021)

Management and treatment of inherited retinal dystrophies
por: Levi, Sarah R., et al.
Publicado: (2021)

Cannot write session to /tmp/vufind_sessions/sess_n42aml6tb2jiuk9sh3vssoui1s