Cargando…

Systemic AAVrh10 provides higher transgene expression than AAV9 in the brain and the spinal cord of neonatal mice

Systemic delivery of self-complementary (sc) adeno-associated-virus vector of serotype 9 (AAV9) was recently shown to provide robust and widespread gene transfer to the central nervous system (CNS), opening new avenues for practical, and non-invasive gene therapy of neurological diseases. More recen...

Descripción completa

Detalles Bibliográficos
Autores principales:	Tanguy, Yannick, Biferi, Maria G., Besse, Aurore, Astord, Stephanie, Cohen-Tannoudji, Mathilde, Marais, Thibaut, Barkats, Martine
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	Frontiers Media S.A. 2015
Materias:	Neuroscience
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4516891/ https://www.ncbi.nlm.nih.gov/pubmed/26283910 http://dx.doi.org/10.3389/fnmol.2015.00036

Ejemplares similares

AAV9-Mediated Expression of SMN Restricted to Neurons Does Not Rescue the Spinal Muscular Atrophy Phenotype in Mice
por: Besse, Aurore, et al.
Publicado: (2020)

Systemic Treatment of Fabry Disease Using a Novel AAV9 Vector Expressing α-Galactosidase A
por: Biferi, Maria Grazia, et al.
Publicado: (2020)

A Single Intravenous AAV9 Injection Mediates Bilateral Gene Transfer to the Adult Mouse Retina
por: Bemelmans, Alexis-Pierre, et al.
Publicado: (2013)

Intra-CSF AAV9 and AAVrh10 Administration in Nonhuman Primates: Promising Routes and Vectors for Which Neurological Diseases?
por: Bey, Karim, et al.
Publicado: (2020)

Gene Therapy for ALS—A Perspective
por: Cappella, Marisa, et al.
Publicado: (2019)

Efficacy and biodistribution analysis of intracerebroventricular administration of an optimized scAAV9-SMN1 vector in a mouse model of spinal muscular atrophy
por: Armbruster, Nicole, et al.
Publicado: (2016)

Lentiviral vector-mediated overexpression of mutant ataxin-7 recapitulates SCA7 pathology and promotes accumulation of the FUS/TLS and MBNL1 RNA-binding proteins
por: Alves, Sandro, et al.
Publicado: (2016)

Selective transduction of cerebellar Purkinje and granule neurons using delivery of AAV-PHP.eB and AAVrh10 vectors at axonal terminal locations
por: Surdyka, Magdalena, 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)

Targeting γ-secretase triggers the selective enrichment of oligomeric APP-CTFs in brain extracellular vesicles from Alzheimer cell and mouse models
por: Lauritzen, Inger, et al.
Publicado: (2019)

Development of capsid- and genome-modified optimized AAVrh74 vectors for muscle gene therapy
por: Shoti, Jakob, et al.
Publicado: (2023)

Implication of the SMN complex in the biogenesis and steady state level of the Signal Recognition Particle
por: Piazzon, Nathalie, et al.
Publicado: (2013)

Intraneuronal aggregation of the β-CTF fragment of APP (C99) induces Aβ-independent lysosomal-autophagic pathology
por: Lauritzen, Inger, et al.
Publicado: (2016)

Persistent Expression of Biologically Active Anti-HER2 Antibody by AAVrh.10-mediated Gene Transfer
por: Wang, Guoqing, et al.
Publicado: (2010)

Can early treatment of twitcher mice with high dose AAVrh10-GALC eliminate the need for BMT?
por: Rafi, Mohammad A, et al.
Publicado: (2021)

Selection of an Efficient AAV Vector for Robust CNS Transgene Expression
por: Hanlon, Killian S., et al.
Publicado: (2019)

Durability of transgene expression after rAAV gene therapy
por: Muhuri, Manish, et al.
Publicado: (2022)

RNAi-mediated rheostat for dynamic control of AAV-delivered transgenes
por: Subramanian, Megha, et al.
Publicado: (2023)

Planet of the AAVs: The Spinal Cord Injury Episode
por: Stepankova, Katerina, et al.
Publicado: (2021)

Widespread AAV1- and AAV2-mediated transgene expression in the nonhuman primate brain: implications for Huntington’s disease
por: Hadaczek, Piotr, et al.
Publicado: (2016)

Gene therapy with secreted acid alpha-glucosidase rescues Pompe disease in a novel mouse model with early-onset spinal cord and respiratory defects
por: Colella, Pasqualina, et al.
Publicado: (2020)

Intrathecal administration of IGF-I by AAVrh10 improves sensory and motor deficits in a mouse model of diabetic neuropathy
por: Homs, Judit, et al.
Publicado: (2014)

Validation of Anti-Adeno Associated Virus Serotype rh10 (AAVrh.10) Total and Neutralizing Antibody Immunogenicity Assays
por: Butala-Flores, Elizabeth, et al.
Publicado: (2023)

Pre Clinical Assessment of AAVrh74.MCK.GNE Viral Vector Therapeutic Potential: Robust Activity Despite Lack of Consistent Animal Model for GNE Myopathy
por: Mitrani-Rosenbaum, Stella, et al.
Publicado: (2022)

AAVrh.10-mediated Genetic Delivery of Bevacizumab to the Pleura to Provide Local Anti-VEGF to Suppress Growth of Metastatic Lung Tumors
por: Watanabe, Masaki, et al.
Publicado: (2010)

AAVrh10 Vector Corrects Disease Pathology in MPS IIIA Mice and Achieves Widespread Distribution of SGSH in Large Animal Brains
por: Hocquemiller, Michaël, et al.
Publicado: (2019)

Evaluation of rAAVrh74 gene therapy vector seroprevalence by measurement of total binding antibodies in patients with Duchenne muscular dystrophy
por: Goedeker, Natalie L., et al.
Publicado: (2023)

AAVrh10 vector corrects pathology in animal models of GM1 gangliosidosis and achieves widespread distribution in the CNS of nonhuman primates
por: Hocquemiller, Michaël, et al.
Publicado: (2022)

Therapeutic rAAVrh10 Mediated SOD1 Silencing in Adult SOD1(G93A) Mice and Nonhuman Primates
por: Borel, Florie, et al.
Publicado: (2016)

Therapeutic efficacy of rscAAVrh74.miniCMV.LIPA gene therapy in a mouse model of lysosomal acid lipase deficiency
por: Lam, Patricia, et al.
Publicado: (2022)

Optimization of AAV expression cassettes to improve packaging capacity and transgene expression in neurons
por: Choi, Jun-Hyeok, et al.
Publicado: (2014)

Kinetics and durability of transgene expression after intrastriatal injection of AAV9 vectors
por: Hollidge, Bradley S., et al.
Publicado: (2022)

AAV-p40 Bioengineering Platform for Variant Selection Based on Transgene Expression
por: Westhaus, Adrian, et al.
Publicado: (2022)

IL12-Mediated Liver Inflammation Reduces the Formation of AAV Transcriptionally Active Forms but Has No Effect over Preexisting AAV Transgene Expression
por: Gil-Fariña, Irene, et al.
Publicado: (2013)

An Isolated Limb Infusion Method Allows for Broad Distribution of rAAVrh74.MCK.GALGT2 to Leg Skeletal Muscles in the Rhesus Macaque
por: Xu, Rui, et al.
Publicado: (2018)

Assessment of Systemic Delivery of rAAVrh74.MHCK7.micro-dystrophin in Children With Duchenne Muscular Dystrophy: A Nonrandomized Controlled Trial
por: Mendell, Jerry R., et al.
Publicado: (2020)

Dose-Escalation Study of Systemically Delivered rAAVrh74.MHCK7.micro-dystrophin in the mdx Mouse Model of Duchenne Muscular Dystrophy
por: Potter, Rachael A., et al.
Publicado: (2021)

Assessment of rAAVrh.74.MHCK7.micro-dystrophin Gene Therapy Using Magnetic Resonance Imaging in Children With Duchenne Muscular Dystrophy
por: Willcocks, Rebecca J., et al.
Publicado: (2021)

Rational design of AAVrh10-vectored ACE2 functional domain to broadly block the cell entry of SARS-CoV-2 variants
por: Li, Minchao, et al.
Publicado: (2022)

Are Child and Adolescent Responses to Placebo Higher in Major Depression than in Anxiety Disorders? A Systematic Review of Placebo-Controlled Trials
por: Cohen, David, et al.
Publicado: (2008)

Cannot write session to /tmp/vufind_sessions/sess_bodbvn0c8u2rrjl22irvo0k456