Cargando…

mRNA trans-splicing dual AAV vectors for (epi)genome editing and gene therapy

Large genes including several CRISPR-Cas modules like gene activators (CRISPRa) require dual adeno-associated viral (AAV) vectors for an efficient in vivo delivery and expression. Current dual AAV vector approaches have important limitations, e.g., low reconstitution efficiency, production of alien...

Descripción completa

Detalles Bibliográficos
Autores principales:	Riedmayr, Lisa Maria, Hinrichsmeyer, Klara Sonnie, Thalhammer, Stefan Bernhard, Mittas, David Manuel, Karguth, Nina, Otify, Dina Yehia, Böhm, Sybille, Weber, Valentin Johannes, Bartoschek, Michael David, Splith, Victoria, Brümmer, Manuela, Ferreira, Raphael, Boon, Nanda, Wögenstein, Gabriele Maria, Grimm, Christian, Wijnholds, Jan, Mehlfeld, Verena, Michalakis, Stylianos, Fenske, Stefanie, Biel, Martin, Becirovic, Elvir
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	Nature Publishing Group UK 2023
Materias:	Article
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10584818/ https://www.ncbi.nlm.nih.gov/pubmed/37852949 http://dx.doi.org/10.1038/s41467-023-42386-0

Ejemplares similares

A gene therapy for inherited blindness using dCas9-VPR–mediated transcriptional activation
por: Böhm, Sybille, et al.
Publicado: (2020)

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

Antisense Oligonucleotide- and CRISPR-Cas9-Mediated Rescue of mRNA Splicing for a Deep Intronic CLRN1 Mutation
por: Panagiotopoulos, Anna-Lena, et al.
Publicado: (2020)

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

Peripherin-2 and Rom-1 have opposing effects on rod outer segment targeting of retinitis pigmentosa-linked peripherin-2 mutants
por: Böhm, Sybille, et al.
Publicado: (2017)

In Vivo Analysis of Disease-Associated Point Mutations Unveils Profound Differences in mRNA Splicing of Peripherin-2 in Rod and Cone Photoreceptors
por: Becirovic, Elvir, et al.
Publicado: (2016)

Physiological roles of cGMP-gated channels: lessons from mouse models and human channelopathies
por: Biel, Martin, et al.
Publicado: (2009)

The Retinitis Pigmentosa Mutation c.3444+1G>A in CNGB1 Results in Skipping of Exon 32
por: Becirovic, Elvir, et al.
Publicado: (2010)

Maybe you can turn me on: CRISPRa-based strategies for therapeutic applications
por: Becirovic, Elvir
Publicado: (2022)

Disturbed Processing of Contextual Information in HCN3 Channel Deficient Mice
por: Stieglitz, Marc S., et al.
Publicado: (2018)

cAMP-dependent regulation of HCN4 controls the tonic entrainment process in sinoatrial node pacemaker cells
por: Fenske, Stefanie, et al.
Publicado: (2020)

A deep intronic CLRN1 (USH3A) founder mutation generates an aberrant exon and underlies severe Usher syndrome on the Arabian Peninsula
por: Khan, Arif O., et al.
Publicado: (2017)

Optimizing AAV2/6 microglial targeting identified enhanced efficiency in the photoreceptor degenerative environment
por: Maes, Margaret E., et al.
Publicado: (2021)

Recombinant Adeno-Associated Viral Vectors (rAAV)-Vector Elements in Ocular Gene Therapy Clinical Trials and Transgene Expression and Bioactivity Assays
por: Buck, Thilo M., 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-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)

Novel AAV capsids for intravitreal gene therapy of photoreceptor disorders
por: Pavlou, Marina, et al.
Publicado: (2021)

AAV-mediated gene augmentation therapy of CRB1 patient-derived retinal organoids restores the histological and transcriptional retinal phenotype
por: Boon, Nanda, et al.
Publicado: (2023)

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

Xenon’s Sedative Effect Is Mediated by Interaction with the Cyclic Nucleotide-Binding Domain (CNBD) of HCN2 Channels Expressed by Thalamocortical Neurons of the Ventrobasal Nucleus in Mice
por: Kassab, Nour El Dine, et al.
Publicado: (2023)

AAV-CRB2 protects against vision loss in an inducible CRB1 retinitis pigmentosa mouse model
por: Buck, Thilo M., et al.
Publicado: (2020)

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

AAV-mediated gene augmentation therapy of CRB1 patient-derived retinal organoids restores the histological and transcriptional retinal phenotype
por: Boon, Nanda, et al.
Publicado: (2023)

PREDICTORS FOR POST- STROKE DELIRIUM OUTCOME
por: Dostovic, Zikrija, et al.
Publicado: (2016)

Post-traumatic Stress Disorder, Metacognitions, Cognitive and Global Functioning in Bosnian War Veterans
por: Tupkovic, Emir, et al.
Publicado: (2019)

Defining Phenotype, Tropism, and Retinal Gene Therapy Using Adeno-Associated Viral Vectors (AAVs) in New-Born Brown Norway Rats with a Spontaneous Mutation in Crb1
por: Boon, Nanda, et al.
Publicado: (2021)

Research Models and Gene Augmentation Therapy for CRB1 Retinal Dystrophies
por: Boon, Nanda, et al.
Publicado: (2020)

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

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

Epi-fingerprinting and epi-interventions for improved crop production and food quality
por: Rodríguez López, Carlos M., et al.
Publicado: (2015)

Applications and potentials of nanopore sequencing in the (epi)genome and (epi)transcriptome era
por: Xie, Shangqian, et al.
Publicado: (2021)

The cGMP-Dependent Protein Kinase II Is an Inhibitory Modulator of the Hyperpolarization-Activated HCN2 Channel
por: Hammelmann, Verena, et al.
Publicado: (2011)

A30P α-Synuclein interferes with the stable integration of adult-born neurons into the olfactory network
por: Neuner, Johanna, et al.
Publicado: (2014)

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

Paradigm shift: new concepts for HCN4 function in cardiac pacemaking
por: Hennis, Konstantin, et al.
Publicado: (2022)

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

EpiGRAPH: user-friendly software for statistical analysis and prediction of (epi)genomic data
por: Bock, Christoph, et al.
Publicado: (2009)

ActivEpi companion textbook: a supplement for use with the ActivEpi CD-ROM
por: Kleinbaum, David G, et al.
Publicado: (2013)

Epi‐immunotherapy for cancers: rationales of epi‐drugs in combination with immunotherapy and advances in clinical trials
por: Xu, Yang, et al.
Publicado: (2022)

Comparison of single-shot EPI and multi-shot EPI in prostate DWI at 3.0 T
por: Tamada, Tsutomu, et al.
Publicado: (2022)

Cannot write session to /tmp/vufind_sessions/sess_o54q4jdsh5j9kf9h6fubmoirfb