Red Blood Cell Anchoring Enables Targeted Transduction and Re‐Administration of AAV‐Mediated Gene Therapy
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DOI: 10.1002/advs.202201293
Abstract: Adeno‐associated virus (AAV)‐mediated gene therapy is a promising therapeutic modality for curing many diseases including monogenic diseases. However, limited tissue‐targeting and restricted re‐administration due to the vector immunogenicity largely restrict its therapeutic potential. Here, using… read more here.
Keywords: aav mediated; administration; gene therapy; transduction ... See more keywords
AAV-Mediated Gene Delivery to the Enteric Nervous System by Intracolonic Injection.
Sign Up to like & getrecommendations! Published in 2019 at "Methods in molecular biology"
DOI: 10.1007/978-1-4939-9139-6_24
Abstract: The enteric nervous system of the lower gastrointestinal tract comprises intrinsic neural circuits as well as extrinsic afferent and efferent innervation. The development of strategies for neuronal gene transfer has created new opportunities for functional… read more here.
Keywords: nervous system; mediated gene; enteric nervous; aav mediated ... See more keywords
AAV-Mediated Anterograde Transsynaptic Tagging: Mapping Corticocollicular Input-Defined Neural Pathways for Defense Behaviors
Sign Up to like & getrecommendations! Published in 2017 at "Neuron"
DOI: 10.1016/j.neuron.2016.11.045
Abstract: To decipher neural circuits underlying brain functions, viral tracers are widely applied to map input and output connectivity of neuronal populations. Despite the successful application of retrograde transsynaptic viruses for identifying presynaptic neurons of transduced… read more here.
Keywords: aav mediated; input defined; input; anterograde transsynaptic ... See more keywords
AAV-mediated direct in vivo CRISPR screen identifies functional suppressors in glioblastoma
Sign Up to like & getrecommendations! Published in 2017 at "Nature neuroscience"
DOI: 10.1038/nn.4620
Abstract: A causative understanding of genetic factors that regulate glioblastoma pathogenesis is of central importance. Here we developed an adeno-associated virus–mediated, autochthonous genetic CRISPR screen in glioblastoma. Stereotaxic delivery of a virus library targeting genes commonly… read more here.
Keywords: mediated direct; crispr; direct vivo; aav mediated ... See more keywords
Rescue of retinal ganglion cells in optic nerve injury using cell-selective AAV mediated delivery of SIRT1
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DOI: 10.1038/s41434-021-00219-z
Abstract: SIRT1 prevents retinal ganglion cell (RGC) loss in models of optic neuropathy following pharmacologic activation or genetic overexpression. The exact mechanism of loss is not known, prior evidence suggests this is through oxidative stress to… read more here.
Keywords: sirt1; aav mediated; retinal ganglion; optic nerve ... See more keywords
AAV-mediated gene transfer of inducible nitric oxide synthase (iNOS) to an animal model of pulmonary hypertension.
Sign Up to like & getrecommendations! Published in 2022 at "Human gene therapy"
DOI: 10.1089/hum.2021.230
Abstract: Pulmonary hypertension (PH) is characterized by progressive obstruction of pulmonary arteries due to inflammatory processes, cellular proliferation, and extracellular matrix deposition and vasoconstriction. As treatment options are limited, we studied gene transfer of an inducible… read more here.
Keywords: transfer inducible; pulmonary hypertension; aav mediated; gene transfer ... See more keywords
Therapeutic potential of AAV-mediated MMP-3 secretion from corneal endothelium in treating glaucoma
Sign Up to like & getrecommendations! Published in 2017 at "Human Molecular Genetics"
DOI: 10.1093/hmg/ddx028
Abstract: Abstract Intraocular pressure (IOP) is maintained as a result of the balance between production of aqueous humour (AH) by the ciliary processes and hydrodynamic resistance to its outflow through the conventional outflow pathway comprising the… read more here.
Keywords: aav mediated; corneal endothelium; mmp; glaucoma ... See more keywords
Coupling AAV-mediated promoterless gene targeting to SaCas9 nuclease to efficiently correct liver metabolic diseases.
Sign Up to like & getrecommendations! Published in 2019 at "JCI insight"
DOI: 10.1172/jci.insight.128863
Abstract: Non-integrative AAV-mediated gene therapy in the liver is effective in adult patients, but faces limitations in pediatric settings due to episomal DNA loss during hepatocyte proliferation. Gene targeting is a promising approach by permanently modifying… read more here.
Keywords: aav mediated; gene targeting; coupling aav; mice ... See more keywords
Analyzing efficacy, stability, and safety of AAV-mediated optogenetic hearing restoration in mice
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DOI: 10.26508/lsa.202101338
Abstract: This longitudinal study revealed stable expression of the fast-gating channelrhodopsin f-Chrimson in cochlear neurons of mice over at least 2 yr upon a single postnatal AAV dosing of the cochlea. AAV-mediated optogenetic neural stimulation has… read more here.
Keywords: aav mediated; aav; hearing restoration; expression ... See more keywords
Adeno-Associated Virus (AAV)-Mediated Gene Therapy for Duchenne Muscular Dystrophy: The Issue of Transgene Persistence
Sign Up to like & getrecommendations! Published in 2022 at "Frontiers in Neurology"
DOI: 10.3389/fneur.2021.814174
Abstract: Duchenne muscular dystrophy (DMD) is an X-linked recessive, infancy-onset neuromuscular disorder characterized by progressive muscle weakness and atrophy, leading to delay of motor milestones, loss of autonomous ambulation, respiratory failure, cardiomyopathy, and premature death. DMD… read more here.
Keywords: aav mediated; therapy; gene therapy; mediated gene ... See more keywords