Cargando…

Effective delivery of large genes to the retina by dual AAV vectors

Retinal gene therapy with adeno-associated viral (AAV) vectors is safe and effective in humans. However, AAV's limited cargo capacity prevents its application to therapies of inherited retinal diseases due to mutations of genes over 5 kb, like Stargardt's disease (STGD) and Usher syndrome...

Descripción completa

Detalles Bibliográficos
Autores principales:	Trapani, Ivana, Colella, Pasqualina, Sommella, Andrea, Iodice, Carolina, Cesi, Giulia, de Simone, Sonia, Marrocco, Elena, Rossi, Settimio, Giunti, Massimo, Palfi, Arpad, Farrar, Gwyneth J, Polishchuk, Roman, Auricchio, Alberto
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	Blackwell Publishing Ltd 2014
Materias:	Research Article
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3927955/ https://www.ncbi.nlm.nih.gov/pubmed/24150896 http://dx.doi.org/10.1002/emmm.201302948

Ejemplares similares

Improved dual AAV vectors with reduced expression of truncated proteins are safe and effective in the retina of a mouse model of Stargardt disease
por: Trapani, Ivana, et al.
Publicado: (2015)

Recombinant Vectors Based on Porcine Adeno-Associated Viral Serotypes Transduce the Murine and Pig Retina
por: Puppo, Agostina, et al.
Publicado: (2013)

Triple Vectors Expand AAV Transfer Capacity in the Retina
por: Maddalena, Andrea, et al.
Publicado: (2018)

Inclusion of a degron reduces levelsof undesired inteins after AAV-mediated proteintrans-splicing in the retina
por: Tornabene, Patrizia, et al.
Publicado: (2021)

Myosin7a Deficiency Results in Reduced Retinal Activity Which Is Improved by Gene Therapy
por: Colella, Pasqualina, et al.
Publicado: (2013)

AAV-PHP.eB transduces both the inner and outer retina with high efficacy in mice
por: Palfi, Arpad, et al.
Publicado: (2022)

AAV-mediated photoreceptor transduction of the pig cone-enriched retina
por: Mussolino, C, et al.
Publicado: (2011)

AAV-Delivered Tulp1 Supplementation Therapy Targeting Photoreceptors Provides Minimal Benefit in Tulp1−/− Retinas
por: Palfi, Arpad, et al.
Publicado: (2020)

A Novel Retinal Ganglion Cell Promoter for Utility in AAV Vectors
por: Hanlon, Killian S., et al.
Publicado: (2017)

MicroRNA-Restricted Transgene Expression in the Retina
por: Karali, Marianthi, et al.
Publicado: (2011)

Adeno-Associated Viral Vectors as a Tool for Large Gene Delivery to the Retina
por: Trapani, Ivana
Publicado: (2019)

Efficacy, pharmacokinetics, and safety in the mouse and primate retina of dual AAV vectors for Usher syndrome type 1B
por: Ferla, Rita, et al.
Publicado: (2023)

Emerging Issues in AAV-Mediated In Vivo Gene Therapy
por: Colella, Pasqualina, et al.
Publicado: (2017)

Proteosomal Degradation Impairs Transcytosis of AAV vectors from Suprachoroidal Space to Retina
por: Ding, Kun, et al.
Publicado: (2021)

Therapeutic homology-independent targeted integration in retina and liver
por: Tornabene, Patrizia, et al.
Publicado: (2022)

Efficient gene delivery to photoreceptors using AAV2/rh10 and rescue of the Rho(–/–) mouse
por: Palfi, Arpad, et al.
Publicado: (2015)

Corrigendum to “Efficient gene delivery to photoreceptors using AAV2/rh10 and rescue of the Rho–/– mouse”
por: Palfi, Arpad, et al.
Publicado: (2016)

Retinal transduction profiles by high-capacity viral vectors
por: Puppo, Agostina, et al.
Publicado: (2014)

Optimisation of AAV-NDI1 Significantly Enhances Its Therapeutic Value for Correcting Retinal Mitochondrial Dysfunction
por: Chadderton, Naomi, et al.
Publicado: (2023)

AAV-mediated transcription factor EB (TFEB) gene delivery ameliorates muscle pathology and function in the murine model of Pompe Disease
por: Gatto, Francesca, et al.
Publicado: (2017)

T Cell-Mediated Immune Responses to AAV and AAV Vectors
por: Ertl, Hildegund C. J.
Publicado: (2021)

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)

Exosome-associated AAV2 vector mediates robust gene delivery into the murine retina upon intravitreal injection
por: Wassmer, Sarah J., et al.
Publicado: (2017)

Non-erythropoietic erythropoietin derivatives protect from light-induced and genetic photoreceptor degeneration
por: Colella, Pasqualina, et al.
Publicado: (2011)

Intravitreal delivery of AAV-NDI1 provides functional benefit in a murine model of Leber hereditary optic neuropathy
por: Chadderton, Naomi, et al.
Publicado: (2013)

AAV-8 and AAV-9 Vectors Cooperate with Serum Proteins Differently Than AAV-1 and AAV-6
por: Denard, Jérôme, et al.
Publicado: (2018)

Human stem cell-based retina on chip as new translational model for validation of AAV retinal gene therapy vectors
por: Achberger, Kevin, et al.
Publicado: (2021)

AAV9 Targets Cone Photoreceptors in the Nonhuman Primate Retina
por: Vandenberghe, Luk H., et al.
Publicado: (2013)

AAV‐mediated gene therapy improving mitochondrial function provides benefit in age‐related macular degeneration models
por: Millington‐Ward, Sophia, et al.
Publicado: (2022)

Syngeneic AAV Pseudo-particles Potentiate Gene Transduction of AAV Vectors
por: Wang, Qizhao, et al.
Publicado: (2016)

Innate Immune Responses to AAV Vectors
por: Rogers, Geoffrey L., et al.
Publicado: (2011)

AAV Vectored Immunoprophylaxis for Filovirus Infections
por: Rghei, Amira D., et al.
Publicado: (2020)

AAV gene therapy vectors in the TMJ
por: Brouxhon, Sabine M., et al.
Publicado: (2022)

Correction: AAV9 Targets Cone Photoreceptors in the Nonhuman Primate Retina
por: Vandenberghe, Luk H., et al.
Publicado: (2013)

Non-photoreceptor Expression of Tulp1 May Contribute to Extensive Retinal Degeneration in Tulp1-/- Mice
por: Palfi, Arpad, et al.
Publicado: (2020)

Targeting the somatosensory system with AAV9 and AAV2retro viral vectors
por: Skorput, Alexander G. J., et al.
Publicado: (2022)

Has retinal gene therapy come of age? From bench to bedside and back to bench
por: Trapani, Ivana, et al.
Publicado: (2019)

Improvement of Photoreceptor Targeting via Intravitreal Delivery in Mouse and Human Retina Using Combinatory rAAV2 Capsid Mutant Vectors
por: Reid, Christopher A., et al.
Publicado: (2017)

High-Throughput Screening Identifies Kinase Inhibitors That Increase Dual Adeno-Associated Viral Vector Transduction In Vitro and in Mouse Retina
por: Maddalena, Andrea, et al.
Publicado: (2018)

Near-perfect infectivity of wild-type AAV as benchmark for infectivity of recombinant AAV vectors
por: Zeltner, Nadja, et al.
Publicado: (2010)

Cannot write session to /tmp/vufind_sessions/sess_7tfmohss1mibgdq6ahc1bud0m8