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1 AAV has emerged as the vector of choice for gene deliver
2 AAV status was associated with a 60% reduction in odds o
3 AAV-hepcidin injected eyes had increased ferritin immuno
4 AAV-NT mice also expressed increased levels of E-cadheri
6 biopsy specimens from 44 patients with acute AAV had more mast cells in the interstitium, which corre
7 Hippocampal antibody levels achieved after AAV delivery were approximately 50-fold more than those
10 toms and complications are less common among AAV pertussis patients, demonstrating that the positive
13 by evolving murine antigenic epitopes on an AAV serotype 1 capsid template can evade NAbs without co
15 ly cross the blood-brain barrier, we used an AAV vector to deliver antibody directly to the hippocamp
20 or templates, we show that combining RNP and AAV donor delivery increases the efficiency of gene addi
21 yploid AAV vectors can be generated from any AAV serotype, whether natural, rational, library derived
23 Together with an intersectional approach, AAV-mediated anterograde transsynaptic tagging can categ
25 r GLP-1R-mediated effects on energy balance, AAV-GLP-1R was injected into the NTS to examine the role
27 added insight into the possible link between AAV integration events and the multifactorial pathogenes
31 2161380), the study drug (self-complementary AAV [scAAV]2(Y444,500,730F)-P1ND4v2) was intravitreally
34 ons of HBV delivered by a self-complementary AAV vector showed better antiviral effects than single s
35 both single-stranded and self-complementary AAV vectors indicate that the genomes are largely packag
37 To overcome this obstacle, we constructed AAV vectors carrying the channelrhodopsin-2 (ChR2) gene
38 reporter protein (NpHR-mCherry) or a control AAV (mCherry) plus optic fiber implants into the lOFC (E
42 me receptor.IMPORTANCE Over the past decade, AAV vectors have emerged as leading gene delivery tools
47 levels in the germ line (Vgf+/-) or in dHc (AAV-Cre-injected floxed mice) showed increased susceptib
48 r, through the investigation of 12 different AAV serotypes (AAV1 to -12), we find that AAP is not an
49 AAVR as a critical entry receptor, different AAV serotypes have evolved distinctive interactions with
50 y and biochemically, and show that different AAV serotypes have discrete interactions with the Ig-lik
52 ted viral particles to generate two distinct AAV interactomes, and identify several functional classe
53 strophic mdx(4cv) mice using single and dual AAV vector delivery of a muscle-specific Cas9 cassette t
56 ploration of effective strategies to enhance AAV transduction and escape from Nab activity is still i
60 some-associated adeno-associated virus, (exo-AAV) enabled broad retinal targeting following intravitr
62 By contrast, optogenetic activation or focal AAV-Cre-mediated knockdown of striatopallidal A(2A)R in
67 have shown the compatibility of capsids from AAV serotypes and homology of recognition sites of AAV N
68 AAV-2/9 containing a CMV-driven MMP-3 gene (AAV-MMP-3) into wild type mice resulted in efficient tra
69 aspects of the AAV life cycle, including how AAV interacts with host cellular factors to facilitate i
70 n more detail is important to understand how AAV engages with its cellular receptor and how the recep
73 T2 mice using imaging, immunohistochemistry, AAV-FLEX-GFP microinjections, and crosses to RiboTag, Ai
75 ormation is important for efforts to improve AAV genome packaging and will also inform the engineerin
76 t the importance of the nucleolus and AAP in AAV assembly and show the heterogeneous nature of the as
79 ession was approximately fourfold greater in AAV-TrkB- vs. AAV-GFP-treated SH animals (P < 0.004) and
80 ance between tolerance and immunogenicity in AAV vector-mediated gene transfer are not fully understo
81 ment pathway (AP) was recently implicated in AAV, and C5a inhibition is currently being tested in cli
82 ipsilateral diaphragm activity increased in AAV-TrkB-treated (9/9) compared with untreated (3/5) or
87 A number of nonenveloped viruses, including AAV, carry proteases that are needed for capsid maturati
88 following a corneal intrastromal injection, AAV vector transduction kinetics, using a chimeric AAV c
91 Better understanding of neutrophil-mediated AAV disease mechanisms may reveal novel treatment strate
92 ted virus (rAAV) vector-treated GSD-Ia mice (AAV-NT mice) expressing a wide range (0.9-63%) of normal
93 o previous work on Alzheimer's mouse models, AAV-Gfa2-VIVIT had no effects on the levels of GFAP and
95 ransduction of vectors derived from multiple AAV serotypes, including the evolutionarily distant sero
96 esent a platform that integrates multiplexed AAV/Cas9-mediated homology-directed repair (HDR) with DN
102 he tropism repertoire of naturally occurring AAVs is limited, prompting a search for novel AAV capsid
105 et by intravitreal delivery, but delivery of AAV by this route results in poor transduction outcomes.
107 sented here provide the first description of AAV vectors transducing neurons following delivery at th
108 ibrillary tangles following a single dose of AAV-vectored PHF1 compared with mice treated with an AAV
109 riodic induction, via use of an eye drop, of AAV-mediated secretion of MMP-3 into AH could have thera
112 greatly improved transduction efficiency of AAV vectors in human and mouse hepatocytes independent o
113 ging and will also inform the engineering of AAV capsid variants for improved tropism, specific tissu
114 s from parental serotypes for enhancement of AAV transduction and evasion of Nab recognition without
115 ing cell lines and the directed evolution of AAV capsids.IMPORTANCE We first report that an autonomou
116 in KO-TgAC1 mice with a single injection of AAV-Clrn1-UTR vector showed correlative preservation of
117 After region-specific viral injections of AAV-GFP or AAV-CRE in CREB(loxP/loxP) animals, behaviora
118 exogenous MMP-3.Intracameral inoculation of AAV-2/9 containing a CMV-driven MMP-3 gene (AAV-MMP-3) i
119 e-treatment allowed efficient maintenance of AAV genomes in mdx muscles and enhanced the AAV-U7 thera
124 This effect was preceded by sequestration of AAV within enlarged vesicles that were dispersed through
126 rotypes and homology of recognition sites of AAV Nab located on different capsid subunits from one vi
128 y of the dystrophic myofibers at the time of AAV-U7 injection, mdx muscles were pre-treated with a si
130 of striatal neurons, while 1 x 10(12) vg of AAV-PHP.S transduced 82% of dorsal root ganglion neurons
131 tration of 1 x 10(11) vector genomes (vg) of AAV-PHP.eB transduced 69% of cortical and 55% of striata
132 cell response determining the efficiency of AAVs intracellular processing in hepatocytes and thus th
136 y recognition sites or antigenic hotspots on AAVs and other related parvoviruses might be evolutionar
138 gion-specific viral injections of AAV-GFP or AAV-CRE in CREB(loxP/loxP) animals, behavioral testing m
139 ndent of the transgene, driving promoter, or AAV serotype and was subsequently confirmed in vivo.
141 uate if HS binding could be applied to other AAV serotypes to enhance their transduction, AAV1 and AA
143 uced higher transduction than their parental AAV vectors (2- to 9-fold over AAV2), with the highest o
147 ome may be useful for generating recombinant AAV-packaging cell lines and the directed evolution of A
148 or suppression by food intake), recombinant AAV-mediated transgene expression was markedly reduced,
149 lications for the improvement of recombinant AAV production in HEK293 cells and cell types that do no
152 Specifically, EerI treatment redirected AAV particles toward large vesicles positive for late en
156 d virus containing the CRRY coding sequence (AAV-CRRY) into the subretinal space of 4-wk-old Abca4(-/
157 with stereotaxic injection of 5-HT2CR shRNA AAV vector decreased vocalizations and anxiety- and depr
158 bition of c-Fos using photoreceptor-specific AAV (adeno-associated virus)-hRK (human rhodopsin kinase
162 ng ligands on the viral capsid can re-target AAVs to new cell types, but limited sites have been iden
169 cid substitution can significantly alter the AAV capsid integrity to the extent of reducing its stabi
170 e, we further biochemically characterize the AAV-AAVR interaction and define the domains within the e
173 AAV genomes in mdx muscles and enhanced the AAV-U7 therapy effect with a ten-fold increase of the pr
174 tes a strategy for inserting motifs into the AAV capsid without compromising viral titer or infectivi
175 ite-specific tetracysteine sequence into the AAV serotype 9 (AAV9) capsid, to permit labelling of vir
176 in the 163-nt common insertion region of the AAV genome, which has been implicated in the dysregulati
178 chment of receptor-targeting agents onto the AAV capsid holds potential to alter its tropism, but is
179 Neonate mice intravenously injected with the AAV-BR1-CAG-NEMO vector developed no hepatocellular carc
182 correlated with the loss of the therapeutic AAV genomes, probably due to alterations of the dystroph
183 type-specific promoters and enhancers, these AAVs enable efficient and targetable genetic modificatio
186 PPMO pre-treatment was also beneficial to AAV-mediated gene therapy with transfer of micro-dystrop
190 For purity analysis, only 25 ng of total AAV capsid proteins (4.3 femtomole virus particles) were
191 virally encoded synaptic anterograde tracer, AAV-SynaptoTag, followed by 3D reconstruction of the cor
192 eral infusions of a retrogradely transported AAV vector expressing Cre recombinase (Retro-Cre-GFP) in
196 gene as well as for the rescue of wild-type AAV genomes from tissues during directed evolution in th
197 nown to prevent degradation of ubiquitinated AAV capsids, thereby leading to increased nuclear accumu
201 outcome of liver-directed gene therapy using AAV vectors and showed in a proof-of-principle study how
203 ge-appropriate number of pertussis vaccines (AAV) (for persons aged >/=3 months) was associated with
205 asmic antibody (ANCA)-associated vasculitis (AAV) constitutes life-threatening autoimmune diseases af
207 peroxidase (MPO)-ANCA-associated vasculitis (AAV) show mast cells degranulate, thus enhancing injury
208 scles with an adeno-associated viral vector (AAV) encoding human Neurotrophin-3 at a clinically-feasi
209 of the adeno-associated virus (AAV) vector, AAV-BR1-CAG-NEMO, delivering the Nemo gene to the brain
210 injection of adeno-associated viral vectors (AAVs) encoding ligand-specific antagonists into the tibi
212 novel combinatorial adeno-associated viral (AAV) gene therapy by expressing DAT selectively in DA ne
214 ed a single-stranded adeno-associated viral (AAV) vector consisting of a bioengineered capsid, liver-
215 s of a Cre-dependent adeno-associated viral (AAV) vector expressing enhanced halorhodopsin 3.0 fused
217 therapies utilizing adeno-associated viral (AAV) vectors hold great promise for treating Duchenne mu
218 nal overexpression of adenoassociated virus (AAV)-ACE2 prevents or reverses diabetic retinopathy.
219 , in combination with adenoassociated virus (AAV)-mediated overexpression of human alpha-syn, at leve
220 aneous or enhanced by adenoassociated virus (AAV)-mediated upregulation of TrkB receptor expression,
221 pplied STN-DBS in an adeno-associated virus (AAV) 1/2-driven human mutated A53T alpha-synuclein (aSyn
222 electroporation and adeno-associated virus (AAV) 6 delivery of donor constructs in human HSPCs appro
224 e Rep68 protein from adeno-associated virus (AAV) combines a DNA binding and endonuclease domain with
226 rally recombinogenic adeno-associated virus (AAV) donor vectors enables site-specific gene insertion
227 elivered recombinant adeno-associated virus (AAV) encoding Abeta42 and Abeta40 peptides fused to BRI2
229 Stable delivery of adeno-associated virus (AAV) expressing cKL to diabetic endothelial nitric oxide
230 eal vascularization, adeno-associated virus (AAV) gene therapy, exploiting a natural immune tolerance
231 administration of an adeno-associated virus (AAV) gene transfer vector significantly prevented pathol
232 SPR-Cas9 delivery by adeno-associated virus (AAV) holds promise for gene therapy but faces critical b
233 livery of mAgrin via adeno-associated virus (AAV) into FKRP mutant mice was unable to improve dystrop
236 ting viruses such as adeno-associated virus (AAV) is not optimal in this setting because the noninteg
238 recently discovered adeno-associated virus (AAV) protein that promotes capsid assembly and provides
240 r purity analysis of adeno-associated virus (AAV) therapeutic products of different serotypes and tra
241 bility to target the adeno-associated virus (AAV) to specific types of cells, by altering the cell-su
242 ously shown that the adeno-associated virus (AAV) variant, ShH10, transduces Muller cells in the Dp71
243 passionate use of an adeno-associated virus (AAV) vector containing the human AADC gene (AAV2-hAADC)
244 atum, we injected an adeno-associated virus (AAV) vector producing a short hairpin RNA (AAV.sh.p11).
245 d function following adeno-associated virus (AAV) vector transduction of MPS1 patient fibroblasts.
246 ravenous dose of the adeno-associated virus (AAV) vector, AAV-BR1-CAG-NEMO, delivering the Nemo gene
247 from a set of other adeno-associated virus (AAV) vectors as a potent vector for the cochlear cell ta
248 athway in rats using adeno-associated virus (AAV) vectors expressing the astrocyte-specific promoter
249 we review the use of adeno-associated virus (AAV) vectors for delivery of HIV bNAbs and antibody-like
250 We investigated adeno-associated virus (AAV) vectors for gene delivery to the TG after intraderm
251 in gene therapy with adeno-associated virus (AAV) vectors have been the object of almost two decades
254 2-fold interface of adeno-associated virus (AAV) was defective for transcription of the packaged gen
255 We combined the adeno-associated virus (AAV) with the Cre-loxP site-specific recombination syste
256 y into the capsid of adeno-associated virus (AAV), a vector that has shown promise in neuroscience re
258 ds for radiolabeling adeno-associated virus (AAV), one of the most commonly used viral vectors for ge
259 age the library into adeno-associated virus (AAV), thereby allowing delivery to target organs in vivo
261 a lineage-specific, adeno-associated virus (AAV)-derived endogenous viral element (mAAV-EVE1) found
262 fically by injecting adeno-associated virus (AAV)-expressing Cre-dependent DREADD (designer receptor
264 ort that a system of adeno-associated virus (AAV)-mediated clustered regularly interspaced short pali
265 ng approach in which adeno-associated virus (AAV)-mediated CRISPR/Cas9 delivery to postmitotic photor
269 tential of naked and adeno-associated virus (AAV)-packaged AONs in vitro and in vivo In both cases, A
270 n restoration during adeno-associated virus (AAV)-U7-mediated exon-skipping therapy was shown to decr
271 dministration of the adeno-associated virus (AAV)-vectored anti-phospho-tau antibody PHF1 to P301S ta
272 fficiently regulated adeno-associated virus (AAV)-vectored transgene expression in cultured mammalian
275 activation by using adeno-associated virus (AAV)9 gene therapy vectors carrying the telomerase Tert
276 we developed a novel adeno-associated virus (AAV-GLP-1R) that utilizes short hairpin RNA to chronical
277 croinjection of a Cre-inducible ErbB4 virus (AAV-ErbB4.DIO) into the mesencephalon of TH-Cre;ErbB4(f/
278 gative, deacetylase-dead point mutant virus (AAV-HDAC3(Y298H)-v5), we found that selectively blocking
280 ase of recombinant adeno-associated viruses (AAV), proteasome inhibitors are known to prevent degrada
284 ies (NAbs) against adeno-associated viruses (AAVs) pose a major, unresolved challenge that restricts
286 roximately fourfold greater in AAV-TrkB- vs. AAV-GFP-treated SH animals (P < 0.004) and in animals di
288 s expected in control lesioned mice, whereas AAV.sh.p11 mice remained at or near normal baseline.
289 AAV are known to have nerve tropism, whether AAV can distribute to sensory nerves that innervate the
290 to the eyes of hepcidin knockout mice, while AAV-lacZ was injected into the contralateral eyes as a c
291 ogy triggered by fibrils in combination with AAV-mediated overexpression of alpha-syn reproduced many
292 for unifying nuclease protein delivery with AAV donor vectors for homology-directed genome editing.
293 ced in BDNF floxed mice injected in dHc with AAV-Cre, and in NBQX- and rapamycin-pretreated wild-type
294 his article, we describe our experience with AAV viral vector delivery system, that allows us to opti
296 we show that hepatic disruption of Ldlr with AAV-CRISPR results in severe hypercholesterolemia and at
297 Behaviorally, KO mice as well as mice with AAV-mediated deletion of CK2alpha in the PFC show a robu
298 helial phospho-p65 staining in patients with AAV indicated that NF-kappaB is activated in human NCGN
299 imilarly, coadministration of rapamycin with AAV vectors resulted in markedly enhanced expression of
300 of human ZIP8 (adeno-associated virus-ZIP8 [AAV-ZIP8]) resulted in increased tissue and whole blood
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