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1 CI-MPR in muscle, was administered with the AAV vector.
2 ansgenes separately, or a single bicistronic AAV vector.
3 ne therapy clinical trials using recombinant AAV vectors.
4 decreased avidity favors systemic spread of AAV vectors.
5 aB in HeLa cells transduced with recombinant AAV vectors.
6 tocol for gene targeting in human cells with AAV vectors.
7 argeted integration of these double-stranded AAV vectors.
8 ignificant hurdle in clinical application of AAV vectors.
9 ization of cell clones containing integrated AAV vectors.
10 s have been developed to better characterize AAV vectors.
11 pecific memory CD8(+) T cells reactivated by AAV vectors.
12 and raise concerns over the clinical use of AAV vectors.
13 ld be co-delivered to TG neurons by separate AAV vectors.
14 a substantial improvement over conventional AAV vectors.
15 relative inaccessibility of BCs to standard AAV vectors.
16 and multiple gRNA expression cassettes with AAV vectors.
17 t for single-stranded and self-complementary AAV vectors.
18 hrough the use of an adeno-associated viral (AAV) vector.
19 igen delivered by an adeno-associated virus (AAV) vector.
20 a single functional adeno-associated virus (AAV) vector.
21 were delivered by an adeno-associated virus (AAV) vector.
22 livery, such as with adeno-associated viral (AAV) vectors.
23 r vectorization into adeno-associated virus (AAV) vectors.
24 replacement genes in adenoassociated virus (AAV) vectors.
25 uced higher transduction than their parental AAV vectors (2- to 9-fold over AAV2), with the highest o
26 th a human SMN-expressing self-complementary AAV vector - a vector that leads to earlier onset of gen
27 ravenous dose of the adeno-associated virus (AAV) vector, AAV-BR1-CAG-NEMO, delivering the Nemo gene
28 fficiency of a novel adeno-associated virus (AAV) vector, AAV2/9, across murine nasal and lung airway
30 ngly, the antibody response was prevented by AAV vector administration during the 12 wk of ERT, and t
31 in the liver was found after single-stranded AAV vector administration, regardless of the capsid sequ
32 overexpressed via an adeno-associated virus (AAV) vector after the onset of spontaneous recurrent sei
33 ficiency of multiple adeno-associated virus (AAV) vectors after a single injection via intraperitonea
34 guide RNA expression cassette into a single AAV vector and targeted the cholesterol regulatory gene
35 to identify photoreceptors transduced by the AAV vector and to localize cone arrestin within cone cel
36 lt mouse brains via stereotaxic injection of AAV vectors and found that it also preferentially accumu
38 ish a profile of insertional mutagenesis for AAV vectors and provide unique insight into the chromoso
39 outcome of liver-directed gene therapy using AAV vectors and showed in a proof-of-principle study how
40 ging, new approaches to engineer and improve AAV vectors and their genetic cargo are increasingly hel
41 s were packaged into adeno-associated virus (AAV) vectors and injected into the ventricles of postnat
42 ing of the determinants of immunogenicity of AAV vectors, and of potential associated toxicities, is
43 dministration of the adeno-associated virus (AAV)-vectored anti-phospho-tau antibody PHF1 to P301S ta
46 aging capacity of the vector, trans-splicing AAV vectors are able to package twice the size of the ve
49 Furthermore, we show that single-stranded AAV vectors are better substrates for site-specific inte
57 nsduction potential, adeno-associated virus (AAV) vectors are leading candidates for gene therapy in
59 multiple pseudotyped adeno-associated virus (AAV) vectors as a means for achieving systemic distribut
60 from a set of other adeno-associated virus (AAV) vectors as a potent vector for the cochlear cell ta
62 ce by intraperitoneal injection of the PQBP1-AAV vector at E10 successfully rescued microcephaly with
64 adjuvant; in contrast, administration of the AAV vector before the GAA challenge prevented the antibo
68 a mechanism for integration and suggest that AAV vectors can integrate at existing chromosome breaks
69 been shown that the adeno-associated virus (AAV) vector can deliver the VEGF gene efficiently into t
71 ators to dissect the immune responses to the AAV vector capsid and to the transgene product, and to d
72 orchid donor testes were exposed in vitro to AAV vectors carrying a GFP transgene and transplanted to
73 To overcome this obstacle, we constructed AAV vectors carrying the channelrhodopsin-2 (ChR2) gene
74 a self-complementary adeno-associated viral (AAV) vector carrying human RP2-coding sequence and demon
76 eletion of mTOR with adeno-associated viral (AAV) vector carrying the Cre recombinase in the hippocam
77 systemic delivery of adeno-associated virus (AAV) vectors carrying human delta-sarcoglycan (delta-SG)
78 ere amplified from human DNA, cloned into an AAV vector cassette upstream of the green fluorescent pr
80 tor system to isolate and analyze 977 unique AAV vector-chromosome integration junctions from normal
81 ed a single-stranded adeno-associated viral (AAV) vector consisting of a bioengineered capsid, liver-
83 served in these mice after injection with an AAV vector containing a lacZ gene fragment, and precise
86 e results highlight the potential utility of AAV vectors containing serotype 5 capsid to deliver and
87 A either in a single vector or in 2 separate AAV vectors containing the heavy- and light-chain cDNAs.
88 gene therapy with an adeno-associated virus (AAV) vector containing a liver-specific promoter elevate
89 passionate use of an adeno-associated virus (AAV) vector containing the human AADC gene (AAV2-hAADC)
90 ion of a recombinant adeno-associated virus (AAV) vector containing the RPE65 gene (AAV2-hRPE65v2) in
93 s study, we injected adeno-associated virus (AAV) vectors containing the human CLN2 cDNA into the bra
94 IIa (mFVIIa) from an adeno-associated viral (AAV) vector corrected abnormal hemostatic parameters in
96 ery of costimulatory inhibitor transgenes by AAV vectors could prevent and reverse lupus in this muri
97 with stereotaxic injection of 5-HT2CR shRNA AAV vector decreased vocalizations and anxiety- and depr
98 sgenic mice using an adeno-associated virus (AAV) vector, decreased parenchymal Abeta amyloid deposit
100 strophic mdx(4cv) mice using single and dual AAV vector delivery of a muscle-specific Cas9 cassette t
103 report that systemic adeno-associated virus (AAV) vector delivery of zinc finger nucleases (ZFNs) and
114 adapted an intravascular delivery system of AAV vectors encoding the FIX transgene to skeletal muscl
115 inal injection of an adeno-associated virus (AAV) vector encoding ARES, luciferase expression can be
116 ly manipulated by an adeno-associated virus (AAV) vector encoding BMP4 delivered by a clinically appl
118 Introduction of an adeno-associated viral (AAV) vector encoding IL-4 into the hippocampus resulted
119 gene therapy with an adeno-associated viral (AAV) vector encoding REP1 (AAV.REP1) in patients with th
120 ) with CRE-dependent adeno-associated viral (AAV) vector encoding the engineered Gi/o-coupled human m
121 administration of an adeno-associated virus (AAV) vector encoding the human DOK7 gene resulted in an
123 long-term effect of adeno-associated viral (AAV) vector-encoding vascular endothelial growth factor
125 we demonstrate that adeno-associated viral (AAV) vectors, especially serotypes 8 and 9, mediated eff
127 eral infusions of a retrogradely transported AAV vector expressing Cre recombinase (Retro-Cre-GFP) in
130 arate cohort was injected with CRE-dependent AAV vectors expressing diphtheria toxin (DTA) to selecti
131 f-complementary adenovirus-associated virus (AAV) vector expressing a codon-optimized human factor IX
132 y to the VMH with an adeno-associated viral (AAV) vector expressing a short hairpin RNA for AMPKalpha
133 6 was depleted using adeno-associated viral (AAV) vector expressing CD36 short hairpin RNA (shRNA) in
134 s of a Cre-dependent adeno-associated viral (AAV) vector expressing enhanced halorhodopsin 3.0 fused
136 iated by AAV-GPE, an adeno-associated virus (AAV) vector expressing G6Pase-alpha directed by the huma
137 In this study, an adeno-associated virus (AAV) vector expressing human SMN (AAV8-hSMN) was injecte
138 to hepatocytes using adeno-associated virus (AAV) vectors expressing hepatic transcription factors.
139 jection, recombinant adeno-associated virus (AAV) vectors expressing short hairpin RNAs profoundly im
140 athway in rats using adeno-associated virus (AAV) vectors expressing the astrocyte-specific promoter
141 sduction patterns of adeno-associated virus (AAV) vectors following delivery to the developing retina
146 ut also provide a general approach to tailor AAV vectors for systemic or hepatic gene transfer by ree
147 eering and payload design aimed at tailoring AAV vectors for transduction and treatment of cancer cel
148 n gene therapy using adeno-associated viral (AAV) vector for hemophilia B (HB) showed that the risk o
149 ommercially available adenoassociated virus (AAV) vector for siRNA delivery into mammalian cells.
150 we review the use of adeno-associated virus (AAV) vectors for delivery of HIV bNAbs and antibody-like
151 We investigated adeno-associated virus (AAV) vectors for gene delivery to the TG after intraderm
153 f seven serotypes of adeno-associated virus (AAV) vectors for genetic manipulation of primary culture
157 With this in mind, adeno-associated virus (AAV) vector gene delivery was used to localize IL-2 expr
158 g second-strand synthesis in single-stranded AAV vector genomes and to facilitate robust transgene ex
160 efficiency, significantly higher numbers of AAV vector genomes were successfully delivered to the nu
161 Gene targeting with adeno-associated virus (AAV) vectors has been demonstrated in multiple human cel
162 (less than 5 kb) of adeno-associated virus (AAV) vectors has been effectively doubled with the devel
163 Gene transfer using adeno-associated virus (AAV) vectors has great potential for treating human dise
166 me receptor.IMPORTANCE Over the past decade, AAV vectors have emerged as leading gene delivery tools
170 in gene therapy with adeno-associated virus (AAV) vectors have been the object of almost two decades
175 therapies utilizing adeno-associated viral (AAV) vectors hold great promise for treating Duchenne mu
180 greatly improved transduction efficiency of AAV vectors in human and mouse hepatocytes independent o
185 in trans, we designed a system for producing AAV vectors in which expression of one capsid protein is
187 n be transduced with adeno-associated virus (AAV) vectors in vivo, in a manner that would be useful f
188 nger genetic elements to be packaged into an AAV vector including tissue-specific promoters, multiple
189 both single-stranded and self-complementary AAV vectors indicate that the genomes are largely packag
190 ssion of HMGB1 using adeno-associated virus (AAV) vectors induced inflammation in the hearts of both
191 smid transfection and 4- to 6-fold lower for AAV vector infection), but they still represented a sign
192 novel high-throughput method for identifying AAV vector integration sites was developed and used to c
193 ensive evaluation of adeno-associated virus (AAV) vector integration sites has not been completed, de
195 We found that a direct microinjection of AAV vectors into the vagal nodose ganglia in vivo leads
197 nactive, efficient transgene expression from AAV vectors is dependent upon viral second-strand DNA sy
199 luation of nNOS binding mini-dystrophin dual AAV vectors is warranted in dystrophic dogs and eventual
200 ion with recombinant adeno-associated virus (AAV) vectors is limited by the need to convert its singl
201 y the status of ss AAV genomes, we generated AAV vectors labeled with bromodeoxyuridine (BrdU), a nuc
203 et of gene expression compared with standard AAV vectors - led to improved efficacy of gene therapy,
204 ted in humans and additional modification to AAV vectors may be required for further study in order t
205 humoral immunity to adeno-associated virus (AAV) vectors may limit their clinical utility in gene de
208 ance between tolerance and immunogenicity in AAV vector-mediated gene transfer are not fully understo
211 ibe a novel study of adeno-associated virus (AAV) vector-mediated gene therapy that induced immune to
212 nt tissue target for adeno-associated viral (AAV) vector-mediated gene transfer of the factor IX (FIX
213 f IFN-beta following adeno-associated virus (AAV) vector-mediated gene transfer resulted in significa
224 ibrillary tangles following a single dose of AAV-vectored PHF1 compared with mice treated with an AAV
227 atum, we injected an adeno-associated virus (AAV) vector producing a short hairpin RNA (AAV.sh.p11).
228 th as a raw material (e.g. in lentiviral and AAV vector production) as well as an active ingredient (
229 poxia-regulated, retinal glial cell-specific AAV vector provides a platform for gene therapy within r
231 Moreover, intracerebral infusion of the AAV vector resulted in robust AIP expression in the hipp
232 jection of 2 x 10(11) vg/kg of the hFVIIcoop AAV vector resulted in therapeutic levels of hFVII expre
233 imilarly, coadministration of rapamycin with AAV vectors resulted in markedly enhanced expression of
234 mic and intravitreal injection of engineered AAV vectors resulted in RdCVF and RdCVFL expression in t
235 iated by recombinant adeno-associated virus (AAV) vectors resulted in significant antiapoptotic activ
236 as well as to a novel, structurally distinct AAV vector, rh32.33, in an in vitro transduction inhibit
239 To evaluate systemic toxicity, we measured AAV vector sequestration in the liver using qPCR, and fo
242 this approach in generating custom-designed AAV vectors should be of significant value to the field
243 F-kappaB inhibitors before transduction with AAV vectors should lead to a dampened immune response, w
244 ons of HBV delivered by a self-complementary AAV vector showed better antiviral effects than single s
245 ophilia B (HB) using adeno-associated viral (AAV) vectors showed that the safety of a given strategy
246 hic factor, using an adeno-associated virus (AAV) vector significantly increased the neuritin level a
247 y, questions have arisen about the safety of AAV vectors, specifically, whether integration of vector
249 c biology of AAV, the history of progress in AAV vector technology, and some of the clinical and rese
251 These studies have led to the development of AAV vectors that are capable of high-efficiency transduc
252 in mdx and mdx4cv mice using a pair of dual AAV vectors that expressed a 6 kb nNOS-binding mini-dyst
253 ts may lend insight into the design of novel AAV vectors that have an enhanced nuclear entry capabili
254 h the circular episomal forms of recombinant AAV vectors that have been isolated and characterized fr
255 he development of an adeno-associated viral (AAV) vector that rescues rhodopsin mislocalization, main
256 the effect of HBV on adeno-associated viral (AAV) vectors, the most frequently applied gene transfer
257 ific integration than are self-complementary AAV vectors; the absence of DNAPKcs did not affect the t
258 ly cross the blood-brain barrier, we used an AAV vector to deliver antibody directly to the hippocamp
259 ese studies show that the use of recombinant AAV vector to deliver genes is a promising approach for
260 y is the first to use a recombinant chimeric AAV vector to knockout a gene in porcine fibroblasts for
262 This review describes the applications of AAV vectors to cancer models and presents developments i
265 The goal was to investigate the ability of AAV vectors to induce long-term, safe delivery of transg
269 We have used an adeno-associated viral (AAV) vector to deliver the genes encoding an anti-phosph
270 further injected an adeno-associated viral (AAV) vector to express IVS-AAA in the brain of a knock-i
272 , we created several adeno-associated virus (AAV) vectors to deliver genes that combat oxidation.
274 n this study, we use adeno-associated viral (AAV) vectors to increase Perm1 expression in skeletal mu
275 and guide RNAs using adeno-associated viral (AAV) vectors to target single (Mecp2) as well as multipl
277 potential strategies to redirect recombinant AAV vectors toward more productive trafficking pathways
280 sented here provide the first description of AAV vectors transducing neurons following delivery at th
283 following a corneal intrastromal injection, AAV vector transduction kinetics, using a chimeric AAV c
284 d function following adeno-associated virus (AAV) vector transduction of MPS1 patient fibroblasts.
286 fficiently regulated adeno-associated virus (AAV)-vectored transgene expression in cultured mammalian
287 None of the dogs (n = 14) receiving the AAV vector under transient IS developed inhibitory antib
291 MRI-guided technology for administration of AAV vectors we have developed and now employ in current
295 led to the development of novel recombinant AAV vectors which are more efficient in allowing increas
298 ion to date from a parenterally administered AAV vector, with broad implications for the future of mu
299 er-specific expression of human GAA with the AAV vector would induce immune tolerance and enhance the
300 doses of recombinant adeno-associated virus (AAV) vectors would allow for therapeutic levels of trans
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