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AAV-PHP.B

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https://www.readbyqxmd.com/read/30291583/effects-of-neutralizing-antibody-production-on-aav-php-b-mediated-transduction-of-the-mouse-central-nervous-system
#1
Yoichiro Shinohara, Ayumu Konno, Keisuke Nitta, Yasunori Matsuzaki, Hiroyuki Yasui, Junya Suwa, Keiju Hiromura, Hirokazu Hirai
Adeno-associated virus (AAV)-PHP.B, a capsid variant of AAV serotype 9, is highly permeable to the blood-brain barrier. A major obstacle to the systemic use of AAV-PHP.B is the generation of neutralizing antibodies (NAbs); however, temporal profiles of NAb production after exposure to AAV-PHP.B, and the influence on later AAV-PHP.B administration, remains unknown. To address these, AAV-PHP.Bs expressing either GFP or mCherry by neuron-specific or astrocyte-specific promoters were intravenously administered to mice at various intervals, and brain expression was examined...
October 5, 2018: Molecular Neurobiology
https://www.readbyqxmd.com/read/30013186/more-expansive-gene-transfer-to-the-rat-cns-aav-php-eb-vector-dose-response-and-comparison-to-aav-php-b
#2
Robert D Dayton, Mychal S Grames, Ronald L Klein
Engineered recombinant adeno-associated virus (AAV) vectors have advanced the transduction of neurons in the CNS on an expansive, wide-scale basis since the papers first using AAV9 for this purpose. Wide-scale CNS expression is relevant to gene therapy as well as indispensable for basic studies such as disease modeling. For example, the wide-scale gene transfer approach could expedite hypothesis testing in vivo relative to the generation of germ-line transgenic mice for all of the genes of interest. Wide-scale gene transfer is more efficient in neonates than in adults, so improving gene transfer efficiency in adults is an important goal...
August 2018: Gene Therapy
https://www.readbyqxmd.com/read/29523880/widespread-transduction-of-astrocytes-and-neurons-in-the-mouse-central-nervous-system-after-systemic-delivery-of-a-self-complementary-aav-php-b-vector
#3
Melvin Y Rincon, Filip de Vin, Sandra I Duqué, Shelly Fripont, Stephanie A Castaldo, Jessica Bouhuijzen-Wenger, Matthew G Holt
Until recently, adeno-associated virus 9 (AAV9) was considered the AAV serotype most effective in crossing the blood-brain barrier (BBB) and transducing cells of the central nervous system (CNS), following systemic injection. However, a newly engineered capsid, AAV-PHP.B, is reported to cross the BBB at even higher efficiency. We investigated how much we could boost CNS transgene expression by using AAV-PHP.B carrying a self-complementary (sc) genome. To allow comparison, 6 weeks old C57BL/6 mice received intravenous injections of scAAV2/9-GFP or scAAV2/PHP...
April 2018: Gene Therapy
https://www.readbyqxmd.com/read/29281027/cas9-sgrna-selective-targeting-of-the-p23h-rhodopsin-mutant-allele-for-treating-retinitis-pigmentosa-by-intravitreal-aav9-php-b-based-delivery
#4
Serena G Giannelli, Mirko Luoni, Valerio Castoldi, Luca Massimino, Tommaso Cabassi, Debora Angeloni, Gian Carlo Demontis, Letizia Leocani, Massimiliano Andreazzoli, Vania Broccoli
P23H is the most common mutation in the RHODOPSIN (RHO) gene leading to a dominant form of retinitis pigmentosa (RP), a rod photoreceptor degeneration that invariably causes vision loss. Specific disruption of the disease P23H RHO mutant while preserving the wild-type (WT) functional allele would be an invaluable therapy for this disease. However, various technologies tested in the past failed to achieve effective changes and consequently therapeutic benefits. We validated a CRISPR/Cas9 strategy to specifically inactivate the P23H RHO mutant, while preserving the WT allele in vitro...
March 1, 2018: Human Molecular Genetics
https://www.readbyqxmd.com/read/29175632/intravenous-administration-of-the-adeno-associated-virus-php-b-capsid-fails-to-upregulate-transduction-efficiency-in-the-marmoset-brain
#5
Yasunori Matsuzaki, Ayumu Konno, Ryuta Mochizuki, Yoichiro Shinohara, Keisuke Nitta, Yukihiro Okada, Hirokazu Hirai
Intravenous administration of adeno-associated virus (AAV)-PHP.B, a capsid variant of AAV9 containing seven amino acid insertions, results in a greater permeability of the blood brain barrier (BBB) than standard AAV9 in mice, leading to highly efficient and global transduction of the central nervous system (CNS). The present study aimed to examine whether the enhanced BBB penetrance of AAV-PHP.B observed in mice also occurs in non-human primates. Thus, a young adult (age, 1.6 years) and an old adult (age, 7...
February 5, 2018: Neuroscience Letters
https://www.readbyqxmd.com/read/28882452/aav-php-b-mediated-global-scale-expression-in-the-mouse-nervous-system-enables-gba1-gene-therapy-for-wide-protection-from-synucleinopathy
#6
Giuseppe Morabito, Serena G Giannelli, Gabriele Ordazzo, Simone Bido, Valerio Castoldi, Marzia Indrigo, Tommaso Cabassi, Stefano Cattaneo, Mirko Luoni, Cinzia Cancellieri, Alessandro Sessa, Marco Bacigaluppi, Stefano Taverna, Letizia Leocani, José L Lanciego, Vania Broccoli
The lack of technology for direct global-scale targeting of the adult mouse nervous system has hindered research on brain processing and dysfunctions. Currently, gene transfer is normally achieved by intraparenchymal viral injections, but these injections target a restricted brain area. Herein, we demonstrated that intravenous delivery of adeno-associated virus (AAV)-PHP.B viral particles permeated and diffused throughout the neural parenchyma, targeting both the central and the peripheral nervous system in a global pattern...
December 6, 2017: Molecular Therapy: the Journal of the American Society of Gene Therapy
https://www.readbyqxmd.com/read/28671695/engineered-aavs-for-efficient-noninvasive-gene-delivery-to-the-central-and-peripheral-nervous-systems
#7
Ken Y Chan, Min J Jang, Bryan B Yoo, Alon Greenbaum, Namita Ravi, Wei-Li Wu, Luis Sánchez-Guardado, Carlos Lois, Sarkis K Mazmanian, Benjamin E Deverman, Viviana Gradinaru
Adeno-associated viruses (AAVs) are commonly used for in vivo gene transfer. Nevertheless, AAVs that provide efficient transduction across specific organs or cell populations are needed. Here, we describe AAV-PHP.eB and AAV-PHP.S, capsids that efficiently transduce the central and peripheral nervous systems, respectively. In the adult mouse, intravenous administration of 1 × 1011 vector genomes (vg) of AAV-PHP.eB transduced 69% of cortical and 55% of striatal neurons, while 1 × 1012 vg of AAV-PHP.S transduced 82% of dorsal root ganglion neurons, as well as cardiac and enteric neurons...
August 2017: Nature Neuroscience
https://www.readbyqxmd.com/read/28008310/corrigendum-better-targeting-better-efficiency-for-wide-scale-neuronal-transduction-with-the-synapsin-promoter-and-aav-php-b
#8
Kasey L Jackson, Robert D Dayton, Benjamin E Deverman, Ronald L Klein
[This corrects the article on p. 116 in vol. 9, PMID: 27867348.].
2016: Frontiers in Molecular Neuroscience
https://www.readbyqxmd.com/read/27867348/better-targeting-better-efficiency-for-wide-scale-neuronal-transduction-with-the-synapsin-promoter-and-aav-php-b
#9
Kasey L Jackson, Robert D Dayton, Benjamin E Deverman, Ronald L Klein
Widespread genetic modification of cells in the central nervous system (CNS) with a viral vector has become possible and increasingly more efficient. We previously applied an AAV9 vector with the cytomegalovirus/chicken beta-actin (CBA) hybrid promoter and achieved wide-scale CNS transduction in neonatal and adult rats. However, this method transduces a variety of tissues in addition to the CNS. Thus we studied intravenous AAV9 gene transfer with a synapsin promoter to better target the neurons. We noted in systematic comparisons that the synapsin promoter drives lower level expression than does the CBA promoter...
2016: Frontiers in Molecular Neuroscience
https://www.readbyqxmd.com/read/26829320/cre-dependent-selection-yields-aav-variants-for-widespread-gene-transfer-to-the-adult-brain
#10
Benjamin E Deverman, Piers L Pravdo, Bryan P Simpson, Sripriya Ravindra Kumar, Ken Y Chan, Abhik Banerjee, Wei-Li Wu, Bin Yang, Nina Huber, Sergiu P Pasca, Viviana Gradinaru
Recombinant adeno-associated viruses (rAAVs) are commonly used vehicles for in vivo gene transfer. However, the tropism repertoire of naturally occurring AAVs is limited, prompting a search for novel AAV capsids with desired characteristics. Here we describe a capsid selection method, called Cre recombination-based AAV targeted evolution (CREATE), that enables the development of AAV capsids that more efficiently transduce defined Cre-expressing cell populations in vivo. We use CREATE to generate AAV variants that efficiently and widely transduce the adult mouse central nervous system (CNS) after intravenous injection...
February 2016: Nature Biotechnology
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