Cargando…

Novel AAV capsids for intravitreal gene therapy of photoreceptor disorders

Gene therapy using recombinant adeno‐associated virus (rAAV) vectors to treat blinding retinal dystrophies has become clinical reality. Therapeutically impactful targeting of photoreceptors still relies on subretinal vector delivery, which detaches the retina and harbours substantial risks of collat...

Descripción completa

Detalles Bibliográficos
Autores principales:	Pavlou, Marina, Schön, Christian, Occelli, Laurence M, Rossi, Axel, Meumann, Nadja, Boyd, Ryan F, Bartoe, Joshua T, Siedlecki, Jakob, Gerhardt, Maximilian J, Babutzka, Sabrina, Bogedein, Jacqueline, Wagner, Johanna E, Priglinger, Siegfried G, Biel, Martin, Petersen‐Jones, Simon M, Büning, Hildegard, Michalakis, Stylianos
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	John Wiley and Sons Inc. 2021
Materias:	Articles
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8033523/ https://www.ncbi.nlm.nih.gov/pubmed/33616280 http://dx.doi.org/10.15252/emmm.202013392

Ejemplares similares

Immunogenicity of Novel AAV Capsids for Retinal Gene Therapy
por: Gehrke, Miranda, et al.
Publicado: (2022)

A Bioengineered In Vitro Model to Assess AAV-Based Gene Therapies for Cyclic GMP-Related Disorders
por: Pavlou, Marina, et al.
Publicado: (2022)

Capsid Modifications for Targeting and Improving the Efficacy of AAV Vectors
por: Büning, Hildegard, et al.
Publicado: (2019)

Biology, Pathobiology and Gene Therapy of CNG Channel-Related Retinopathies
por: Gerhardt, Maximilian J., et al.
Publicado: (2023)

Targeting Photoreceptors via Intravitreal Delivery Using Novel, Capsid-Mutated AAV Vectors
por: Kay, Christine N., et al.
Publicado: (2013)

AAV Vectors for FRET-Based Analysis of Protein-Protein Interactions in Photoreceptor Outer Segments
por: Becirovic, Elvir, et al.
Publicado: (2016)

Enhancing the Clinical Potential of AAV Vectors by Capsid Engineering to Evade Pre-Existing Immunity
por: Bartel, Melissa, et al.
Publicado: (2011)

Correction: Targeting Photoreceptors via Intravitreal Delivery Using Novel, Capsid-Mutated AAV Vectors
Publicado: (2014)

Correction: Targeting Photoreceptors via Intravitreal Delivery Using Novel, Capsid-Mutated AAV Vectors
por: Kay, Christine N., et al.
Publicado: (2013)

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)

Achromatopsia: Genetics and Gene Therapy
por: Michalakis, Stylianos, et al.
Publicado: (2021)

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)

Immunobiology of a rationally-designed AAV2 capsid following intravitreal delivery in mice
por: Whitehead, Michael, et al.
Publicado: (2023)

Ca(v)1.4 L-Type Calcium Channels Contribute to Calpain Activation in Degenerating Photoreceptors of rd1 Mice
por: Schön, Christian, et al.
Publicado: (2016)

AAV-Mediated Gene Supplementation Therapy in Achromatopsia Type 2: Preclinical Data on Therapeutic Time Window and Long-Term Effects
por: Mühlfriedel, Regine, et al.
Publicado: (2017)

More than meets the eye: The role of microglia in healthy and diseased retina
por: Murenu, Elisa, et al.
Publicado: (2022)

Long-term ocular damage after recovery from COVID-19: lack of evidence at three months
por: Brantl, Victor, et al.
Publicado: (2021)

Early Microglia Activation Precedes Photoreceptor Degeneration in a Mouse Model of CNGB1-Linked Retinitis Pigmentosa
por: Blank, Thomas, et al.
Publicado: (2018)

Hepatocellular Carcinoma Is a Natural Target for Adeno-Associated Virus (AAV) 2 Vectors
por: Meumann, Nadja, et al.
Publicado: (2022)

Gene Therapy with Voretigene Neparvovec Improves Vision and Partially Restores Electrophysiological Function in Pre-School Children with Leber Congenital Amaurosis
por: Gerhardt, Maximilian J., et al.
Publicado: (2022)

Risk of transient vision loss after intravitreal aflibercept using vial-prepared vs. the novel prefilled syringe formulation
por: Klaas, Julian E., et al.
Publicado: (2023)

A universal protocol for isolating retinal ON bipolar cells across species via fluorescence-activated cell sorting
por: Murenu, Elisa, et al.
Publicado: (2021)

Towards Clinical Implementation of Adeno-Associated Virus (AAV) Vectors for Cancer Gene Therapy: Current Status and Future Perspectives
por: Hacker, Ulrich T., et al.
Publicado: (2020)

AAV capsid CD8+ T-cell epitopes are highly conserved across AAV serotypes
por: Hui, Daniel J, et al.
Publicado: (2015)

Tailoring the AAV2 capsid vector for bone-targeting
por: Alméciga-Díaz, Carlos J., et al.
Publicado: (2018)

The HLA class-II immunopeptidomes of AAV capsids proteins
por: Brito-Sierra, Carlos A., et al.
Publicado: (2022)

The HLA class I immunopeptidomes of AAV capsid proteins
por: Brito-Sierra, Carlos A., et al.
Publicado: (2023)

The hunt for novel AAV capsids with improved cardiac tropism
por: Miguel-dos-Santos, Rodrigo, et al.
Publicado: (2023)

Hif1a inactivation rescues photoreceptor degeneration induced by a chronic hypoxia-like stress
por: Barben, Maya, et al.
Publicado: (2018)

Retinal Cyclic Nucleotide-Gated Channels: From Pathophysiology to Therapy
por: Michalakis, Stylianos, et al.
Publicado: (2018)

Adeno‐associated virus serotype 2 capsid variants for improved liver‐directed gene therapy
por: Meumann, Nadja, et al.
Publicado: (2023)

A multiplexed barcode approach to simultaneously evaluate gene delivery by adeno-associated virus capsid variants in nonhuman primates
por: Stone, Daniel, et al.
Publicado: (2023)

Site-Specific N-Glycosylation on the AAV8 Capsid Protein
por: Aloor, Arya, et al.
Publicado: (2018)

Cross-Packaging and Capsid Mosaic Formation in Multiplexed AAV Libraries
por: Schmit, Pauline F., et al.
Publicado: (2019)

AAV- based vector improvements unrelated to capsid protein modification
por: Shitik, Ekaterina M., et al.
Publicado: (2023)

Erratum: The HLA class-II immunopeptidomes of AAV capsids proteins
Publicado: (2023)

Capsid-expressing DNA in AAV vectors and its elimination by use of an oversize capsid gene for vector production
por: Halbert, Christine L., et al.
Publicado: (2010)

Novel Mutant AAV2 Rep Proteins Support AAV2 Replication without Blocking HSV-1 Helpervirus Replication
por: Seyffert, Michael, et al.
Publicado: (2017)

Adeno-associated Virus (AAV) Capsid Chimeras with Enhanced Infectivity Reveal a Core Element in the AAV Genome Critical for both Cell Transduction and Capsid Assembly
por: Viney, Lydia, et al.
Publicado: (2021)

GluA4-Targeted AAV Vectors Deliver Genes Selectively to Interneurons while Relying on the AAV Receptor for Entry
por: Hartmann, Jessica, et al.
Publicado: (2019)

Cannot write session to /tmp/vufind_sessions/sess_3m7rnja9um848j3giivfi7q977