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1 siRYR2-U10), we evaluated the efficacy of an adeno-associated serotype 9 viral vector (AAV9) expressi
2 e potential utility of a novel combinatorial adeno-associated viral (AAV) gene therapy by expressing
4 gene therapy approach using liver-directed, adeno-associated viral (AAV) serotype 8 vector delivery
6 eived bilateral infusions of a Cre-dependent adeno-associated viral (AAV) vector expressing enhanced
7 ccessful clinical data on gene therapy using adeno-associated viral (AAV) vector for hemophilia B (HB
10 Rho specific transcriptional silencing upon adeno-associated viral (AAV) vector-mediated expression
15 of the new millennium it became evident that adeno-associated viral (AAV)-mediated gene transfer to t
16 shown that in vitro transduction with bovine adeno-associated viral (BAAV) vectors restores connexin
17 delivered to HSA(LR) mice using recombinant adeno-associated viral (rAAV) vectors injected intraveno
18 cute pharmacological studies and longer-term adeno-associated viral knockdown experiments indicate th
20 helial growth factor (VEGF) gene therapy via adeno-associated viral type-2 (AAV2) vector to produce s
21 eatment of affected forelimb muscles with an adeno-associated viral vector (AAV) encoding human Neuro
23 Recent clinical breakthroughs in recombinant adeno-associated viral vector (rAAV)-based gene therapy
25 a-synuclein 1-120 and rats injected with the adeno-associated viral vector carrying wild-type human a
26 with SMA1, a single intravenous infusion of adeno-associated viral vector containing DNA coding for
27 em that combines Cas9 ribonucleoproteins and adeno-associated viral vector delivery of a homologous d
28 cortical neuron cultures transduced with an adeno-associated viral vector driving the GCaMP6f report
29 genic retinal detachment for the delivery of adeno-associated viral vector encoding Rab Escort Protei
30 mal area was reduced after treatment with an adeno-associated viral vector expressing a short hairpin
31 itude changes following administration of an adeno-associated viral vector expressing Flag-micro-dyst
34 r fluorescent tag alone by using recombinant adeno-associated viral vector in the liver of three mous
35 in vivo for their functional properties with adeno-associated viral vector libraries and identify car
38 ear of Ush1c c.216G>A mice using a synthetic adeno-associated viral vector, Anc80L65, shown to transd
39 otent shRNA, S1, which, when delivered by an adeno-associated viral vector, effectively inhibits HBV
41 3 signaling axis, we used local injection of adeno-associated viral vectors (AAVs) encoding ligand-sp
42 kers was present in 2 patients who underwent adeno-associated viral vectors (serotype 2)-mediated NGF
43 feasibility of gene therapy via recombinant adeno-associated viral vectors as a viable treatment app
46 and cynomolgus macaques were transduced with adeno-associated viral vectors encoding hNTCP and subseq
48 LRRK2 rats were injected intracranially with adeno-associated viral vectors expressing human alpha-sy
49 his study seeks to enhance the capability of adeno-associated viral vectors for therapeutic gene deli
50 vivo intramuscular injections of recombinant adeno-associated viral vectors harbouring a myosin trans
53 s) were ovariectomized, and, 14 weeks later, adeno-associated viral vectors were used to express ERal
54 iver viral envelopes) and self-complementary adeno-associated viral vectors, serotype-9 (scAAV-9) in
60 finger nuclease mRNA via electroporation and adeno-associated virus (AAV) 6 delivery of donor constru
61 , we assessed the therapeutic efficacy of an adeno-associated virus (AAV) 9-mbetagal vector infused s
62 ession in 1-month old mdx:utr (-/-) mice via adeno-associated virus (AAV) 9-mediated RNA interference
66 g RNP delivery with naturally recombinogenic adeno-associated virus (AAV) donor vectors enables site-
67 roteostasis in AMD, we delivered recombinant adeno-associated virus (AAV) encoding Abeta42 and Abeta4
70 fective therapy for corneal vascularization, adeno-associated virus (AAV) gene therapy, exploiting a
71 al cord via intrathecal administration of an adeno-associated virus (AAV) gene transfer vector signif
79 therapy using nonintegrating viruses such as adeno-associated virus (AAV) is not optimal in this sett
85 ating protein (AAP) is a recently discovered adeno-associated virus (AAV) protein that promotes capsi
86 e (ZFN) mRNA with donor template delivery by adeno-associated virus (AAV) serotype 6 vectors directs
88 placement of SDS-PAGE for purity analysis of adeno-associated virus (AAV) therapeutic products of dif
92 previously reported compassionate use of an adeno-associated virus (AAV) vector containing the human
93 ateral subretinal injection of a recombinant adeno-associated virus (AAV) vector containing the RPE65
95 ssion in the dorsal striatum, we injected an adeno-associated virus (AAV) vector producing a short ha
96 d for IDUA production and function following adeno-associated virus (AAV) vector transduction of MPS1
97 dministered a single intravenous dose of the adeno-associated virus (AAV) vector, AAV-BR1-CAG-NEMO, d
98 ed in the brains of transgenic mice using an adeno-associated virus (AAV) vector, decreased parenchym
102 ants identified Anc80L65 from a set of other adeno-associated virus (AAV) vectors as a potent vector
103 reprogramming of MFs into hepatocytes using adeno-associated virus (AAV) vectors expressing hepatic
104 the astrocytic CN/NFAT pathway in rats using adeno-associated virus (AAV) vectors expressing the astr
110 eover, cardiac overexpression of HMGB1 using adeno-associated virus (AAV) vectors induced inflammatio
112 itution, Y704A, near the 2-fold interface of adeno-associated virus (AAV) was defective for transcrip
114 retrograde functionality into the capsid of adeno-associated virus (AAV), a vector that has shown pr
115 n mouse are systematically investigated with adeno-associated virus (AAV), an anterograde viral trace
116 ere we present two methods for radiolabeling adeno-associated virus (AAV), one of the most commonly u
117 ct design, RAM can be packaged into a single adeno-associated virus (AAV), providing great versatilit
118 teps in the life cycle of a human virus, the adeno-associated virus (AAV), that causes no known disea
122 ticospinal neurons specifically by injecting adeno-associated virus (AAV)-expressing Cre-dependent DR
126 we report a genome editing approach in which adeno-associated virus (AAV)-mediated CRISPR/Cas9 delive
127 enerated mice with elevated expression using adeno-associated virus (AAV)-mediated gene delivery.
132 plore the therapeutic potential of naked and adeno-associated virus (AAV)-packaged AONs in vitro and
133 ST input, and the right CST was treated with adeno-associated virus (AAV)-Sox11 or AAV-EBFP control,
134 lar dystrophy, dystrophin restoration during adeno-associated virus (AAV)-U7-mediated exon-skipping t
135 irect intrahippocampal administration of the adeno-associated virus (AAV)-vectored anti-phospho-tau a
139 c efficacy of telomerase activation by using adeno-associated virus (AAV)9 gene therapy vectors carry
141 ta pathology, we investigated the effects of adeno-associated virus (AAV2/1)-mediated expression of I
146 highly efficient CRISPR directed recombinant Adeno-Associated Virus (rAAV) mediated gene targeting ap
147 that gene therapy mediated by a recombinant adeno-associated virus (rAAV) vector expressing human G6
148 n mice receiving high doses of a recombinant adeno-associated virus (rAAV) vector expressing shRNAs (
149 that the non-tumor-bearing (NT), recombinant adeno-associated virus (rAAV) vector-treated GSD-Ia mice
150 lionic or intrathecal injection, recombinant adeno-associated virus (rAAV) vectors can also infect se
151 tional microdystrophin genes and recombinant adeno-associated virus (rAAV) vectors is an attractive s
152 ne therapy in G6pt-/- mice using recombinant adeno-associated virus (rAAV) vectors, directed by eithe
154 d safety of gene therapy through infusion of adeno-associated virus 1 (AAV1)/SERCA2a in patients with
155 time mice were intracranially injected with adeno-associated virus 1 expressing murine IL-6 (AAV1-mI
157 been studied mostly in the dependoparvovirus adeno-associated virus 2 (AAV2) and the protoparvovirus
159 che for either virus to replicate.IMPORTANCE Adeno-associated virus 2 (AAV2) differs from most other
161 ector, rAAV2/HBoV1, in which the recombinant adeno-associated virus 2 (rAAV2) genome is pseudopackage
164 efficacy of gene therapy with a recombinant adeno-associated virus 2/2 (rAAV2/2) vector carrying the
165 lso was performed in mice given infusions of adeno-associated virus 6-NF-kappaB-luciferase, to assess
167 ntrols, were given injections of recombinant adeno-associated virus 8 vector that expressed the prima
168 In proof-of-concept experiments, we used Adeno-Associated Virus 9 (AAV9) to deliver single-guide
170 or JNCL by generating two self-complementary adeno-associated virus 9 (scAAV9) constructs to address
171 livery approach using two self-complementary adeno-associated virus 9 (scAAV9) constructs to address
173 holesterolaemia liver chimeric mice using an adeno-associated virus 9-based gene therapy and restore
174 -type levels in a mouse model of I/R, as did adeno-associated virus 9-mediated ATF6 overexpression.
176 D1-MSNs versus D2-MSNs using a Cre-inducible adeno-associated virus and Cre lines during cocaine cond
177 urb a specific molecular interaction between adeno-associated virus and its host cell, which can be r
179 therapy include the emergence of recombinant adeno-associated virus as the vector of choice, capsid e
182 we lineage-labeled hepatocytes by injecting adeno-associated virus containing thyroxine-binding glob
183 iabetes and two models of type 2 diabetes by adeno-associated virus delivery of renin (ReninAAV).
185 ta signaling in the substantia nigra through adeno-associated virus expressing a constitutively activ
187 g 5-HT system by stereotaxic injection of an adeno-associated virus expressing Cre recombinase (AAV-C
188 of stress-susceptible or mice injected with adeno-associated virus expressing shRNA against Cldn5 ca
190 rate that particular groups of drugs enhance adeno-associated virus gene delivery by unknown mechanis
192 tential for FDA-approved drug enhancement of adeno-associated virus gene therapy, which could result
195 constructs in mouse rod photoreceptors using adeno-associated virus in Xenopus laevis rod photorecept
196 ng reporter genes into the safe-harbor locus adeno-associated virus integration site 1 in human embry
197 In addition, introduction of 3xMyc-FXR1 via adeno-associated virus into mice leads to the redistribu
198 We used a DREADD, hM3Dq, administered via adeno-associated virus into the LC under a synthetic pro
199 s when the packaging capacity of recombinant adeno-associated virus is limited while tissue-specific
201 n via stereotactic hippocampal injections of adeno-associated virus particles in mutant hAPP Tg mouse
202 teral, intranigral injections of recombinant adeno-associated virus pseudotype 2/5 to overexpress wil
204 pression of DISC1 in the hippocampus with an adeno-associated virus reduced the levels of BACE1, solu
207 teral dorsal amygdala Ce-region infusions of adeno-associated virus serotype 2 containing the CRF con
208 single intravenous dose of a codon-optimized adeno-associated virus serotype 5 (AAV5) vector encoding
209 rphysiological doses of MIS, using either an adeno-associated virus serotype 9 (AAV9) gene therapy ve
210 acy of gene therapy for NPC1, we constructed adeno-associated virus serotype 9 (AAV9) vectors to deli
211 h SMA1 received a single dose of intravenous adeno-associated virus serotype 9 carrying SMN complemen
212 a potent inducer of premature senescence, by adeno-associated virus serotype 9 gene transfer, resulte
213 CRISPR/Cas9-based cardiac gene editing using adeno-associated virus serotype 9 to deliver a single sh
216 ic implications, we found that prior in vivo Adeno-associated virus serotype 9-mediated gene delivery
218 the heart of 7-month-old SJ13 mice using an adeno-associated virus serotype-9 Cre recombinase vector
219 Cardiac-specific overexpression of FGF16 via adeno-associated virus subtype 9 (AAV9) in the mutant he
220 g within the VTA, we delivered Cre-inducible adeno-associated virus that drives the expression of flu
222 c retrograde tracing to label the inputs and adeno-associated virus to trace axonal projections, we i
223 rat spinal cord after dorsal root crush and adeno-associated virus transgene expression in dorsal ro
224 ene expression using aerosolized delivery of adeno-associated virus type 1 (AAV1) in a large animal m
227 G of CXCL10(-/-) mice, using the neurotropic adeno-associated virus type 8 (AAV8) vector, boosted the
228 ty and tolerability of escalated doses of an adeno-associated virus vector (AAV) expressing a normal
229 cus aureus) and guide RNA constructs into an adeno-associated virus vector and systemically delivered
230 t NOD scid gamma (NSG) mice by the use of an adeno-associated virus vector, followed by engraftment o
232 we show that expression of these genes from adeno-associated virus vectors in C57BL/6 mice is able t
233 gnaling in a common mouse model of AD, using adeno-associated virus vectors normalized glutamate sign
234 human patients, and gene augmentation using adeno-associated virus vectors robustly sustained the re
235 ts within the packaging limit of recombinant adeno-associated virus vectors that have been shown to b
238 n of c-Fos using photoreceptor-specific AAV (adeno-associated virus)-hRK (human rhodopsin kinase)-sh_
239 We investigated whether exosome-associated adeno-associated virus, (exo-AAV) enabled broad retinal
240 han HepG2 cells after HBV genome delivery by adeno-associated virus, and stable expression of NTCP in
241 ntify integrations of murine leukemia virus, adeno-associated virus, Tol2 transposons or Ac/Ds transp
243 in postnatal muscle tissue in vivo, we used adeno-associated virus-9 (AAV9) to deliver gene-editing
245 e treatment of a mouse model of OPMD with an adeno-associated virus-based gene therapy combining comp
248 ntramuscular administration of a recombinant adeno-associated virus-based vector (rAAV vector) expres
249 d deep sequencing results confirmed that our adeno-associated virus-CRISPR/Cas9 strategy was very eff
251 e, using a combination of electrophysiology, adeno-associated virus-delivered fluorescent proteins, a
254 n the R6/2 Huntington's disease mouse model, adeno-associated virus-mediated delivery of CYP46A1 into
255 n CYP46A1 expression in the striatum, via an adeno-associated virus-mediated delivery of selective sh
256 ethylmalonic encephalopathy, liver-targeted adeno-associated virus-mediated ETHE1 gene transfer dram
257 llel, we explored the effects of recombinant adeno-associated virus-mediated expression of Abeta38 an
259 llowing high over-expression of WT hAIPL1 by adeno-associated virus-mediated gene delivery, which was
260 u scFvs and tested their efficacy in vivo by adeno-associated virus-mediated gene transfer to the bra
275 R induced by intraparenchymal delivery of an adeno-associated virus-short hairpin RNA construct was s
276 We used mice with a mutation in Npas2 and adeno-associated virus-short hairpin RNA mediated knockd
277 liver-specific overexpression of human ZIP8 (adeno-associated virus-ZIP8 [AAV-ZIP8]) resulted in incr
283 ected strategies based on SNTRVAP-displaying adeno-associated virus/phage (AAVP) particles in mice be
284 R-155) in an in vivo model of PD produced by adeno-associated-virus-mediated expression of alpha-syn.
294 sting neutralizing antibodies (NAbs) against adeno-associated viruses (AAVs) pose a major, unresolved
298 particle-mediated delivery of Cas9 mRNA with adeno-associated viruses encoding a sgRNA and a repair t
300 l vectors can package larger transgenes than adeno-associated viruses, yet lentiviral vectors remain
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