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1 ll enough to be packaged efficiently into an adeno-associated virus.
2 er a single systemic delivery of recombinant adeno-associated virus.
3 ckout mice and mice injected with 3xMyc-FXR1 adeno-associated virus.
4 he murine heart was performed by means of an adeno-associated virus.
5 nd a donor template delivered by recombinant adeno-associated virus.
6 ns of somatic brain transgenesis mediated by adeno-associated virus.
7 2 and a reporter through the injection of an adeno-associated virus.
8 d safety of gene therapy through infusion of adeno-associated virus 1 (AAV1)/SERCA2a in patients with
9 time mice were intracranially injected with adeno-associated virus 1 expressing murine IL-6 (AAV1-mI
11 been studied mostly in the dependoparvovirus adeno-associated virus 2 (AAV2) and the protoparvovirus
13 che for either virus to replicate.IMPORTANCE Adeno-associated virus 2 (AAV2) differs from most other
15 ector, rAAV2/HBoV1, in which the recombinant adeno-associated virus 2 (rAAV2) genome is pseudopackage
18 efficacy of gene therapy with a recombinant adeno-associated virus 2/2 (rAAV2/2) vector carrying the
20 lso was performed in mice given infusions of adeno-associated virus 6-NF-kappaB-luciferase, to assess
22 ntrols, were given injections of recombinant adeno-associated virus 8 vector that expressed the prima
25 or JNCL by generating two self-complementary adeno-associated virus 9 (scAAV9) constructs to address
26 livery approach using two self-complementary adeno-associated virus 9 (scAAV9) constructs to address
28 holesterolaemia liver chimeric mice using an adeno-associated virus 9-based gene therapy and restore
29 -type levels in a mouse model of I/R, as did adeno-associated virus 9-mediated ATF6 overexpression.
31 in postnatal muscle tissue in vivo, we used adeno-associated virus-9 (AAV9) to deliver gene-editing
34 finger nuclease mRNA via electroporation and adeno-associated virus (AAV) 6 delivery of donor constru
35 , we assessed the therapeutic efficacy of an adeno-associated virus (AAV) 9-mbetagal vector infused s
36 ession in 1-month old mdx:utr (-/-) mice via adeno-associated virus (AAV) 9-mediated RNA interference
41 g RNP delivery with naturally recombinogenic adeno-associated virus (AAV) donor vectors enables site-
43 icistronic gene transfer vector derived from Adeno-associated virus (AAV) enables a wide range of app
44 roteostasis in AMD, we delivered recombinant adeno-associated virus (AAV) encoding Abeta42 and Abeta4
48 fective therapy for corneal vascularization, adeno-associated virus (AAV) gene therapy, exploiting a
49 al cord via intrathecal administration of an adeno-associated virus (AAV) gene transfer vector signif
56 n, we show that overexpression of Arg1 using adeno-associated virus (AAV) in the CNS of rTg4510 tau t
61 therapy using nonintegrating viruses such as adeno-associated virus (AAV) is not optimal in this sett
67 ating protein (AAP) is a recently discovered adeno-associated virus (AAV) protein that promotes capsi
68 e (ZFN) mRNA with donor template delivery by adeno-associated virus (AAV) serotype 6 vectors directs
70 placement of SDS-PAGE for purity analysis of adeno-associated virus (AAV) therapeutic products of dif
76 previously reported compassionate use of an adeno-associated virus (AAV) vector containing the human
77 ateral subretinal injection of a recombinant adeno-associated virus (AAV) vector containing the RPE65
80 ssion in the dorsal striatum, we injected an adeno-associated virus (AAV) vector producing a short ha
81 vity-dependent neurotrophic factor, using an adeno-associated virus (AAV) vector significantly increa
82 d for IDUA production and function following adeno-associated virus (AAV) vector transduction of MPS1
83 dministered a single intravenous dose of the adeno-associated virus (AAV) vector, AAV-BR1-CAG-NEMO, d
84 ed in the brains of transgenic mice using an adeno-associated virus (AAV) vector, decreased parenchym
85 ood, Crudele et al describe a novel study of adeno-associated virus (AAV) vector-mediated gene therap
90 ants identified Anc80L65 from a set of other adeno-associated virus (AAV) vectors as a potent vector
92 reprogramming of MFs into hepatocytes using adeno-associated virus (AAV) vectors expressing hepatic
93 the astrocytic CN/NFAT pathway in rats using adeno-associated virus (AAV) vectors expressing the astr
100 eover, cardiac overexpression of HMGB1 using adeno-associated virus (AAV) vectors induced inflammatio
102 itution, Y704A, near the 2-fold interface of adeno-associated virus (AAV) was defective for transcrip
104 retrograde functionality into the capsid of adeno-associated virus (AAV), a vector that has shown pr
105 n mouse are systematically investigated with adeno-associated virus (AAV), an anterograde viral trace
106 ere we present two methods for radiolabeling adeno-associated virus (AAV), one of the most commonly u
107 ct design, RAM can be packaged into a single adeno-associated virus (AAV), providing great versatilit
108 teps in the life cycle of a human virus, the adeno-associated virus (AAV), that causes no known disea
112 ticospinal neurons specifically by injecting adeno-associated virus (AAV)-expressing Cre-dependent DR
116 we report a genome editing approach in which adeno-associated virus (AAV)-mediated CRISPR/Cas9 delive
117 on ARVC cardiac manifestations in mice after adeno-associated virus (AAV)-mediated gene delivery of m
118 enerated mice with elevated expression using adeno-associated virus (AAV)-mediated gene delivery.
123 plore the therapeutic potential of naked and adeno-associated virus (AAV)-packaged AONs in vitro and
124 ST input, and the right CST was treated with adeno-associated virus (AAV)-Sox11 or AAV-EBFP control,
125 lar dystrophy, dystrophin restoration during adeno-associated virus (AAV)-U7-mediated exon-skipping t
126 irect intrahippocampal administration of the adeno-associated virus (AAV)-vectored anti-phospho-tau a
131 c efficacy of telomerase activation by using adeno-associated virus (AAV)9 gene therapy vectors carry
132 We performed intramuscular injection of an adeno-associated virus (AAV)9 vector expressing GAA (AAV
138 ta pathology, we investigated the effects of adeno-associated virus (AAV2/1)-mediated expression of I
145 9 presents challenges in packaging it within adeno-associated viruses (AAVs) for clinical application
147 sting neutralizing antibodies (NAbs) against adeno-associated viruses (AAVs) pose a major, unresolved
150 amine neurons in both the MPP(+)-lesioned or adeno-associated virus alpha-synuclein rat models of Par
151 D1-MSNs versus D2-MSNs using a Cre-inducible adeno-associated virus and Cre lines during cocaine cond
152 urb a specific molecular interaction between adeno-associated virus and its host cell, which can be r
154 han HepG2 cells after HBV genome delivery by adeno-associated virus, and stable expression of NTCP in
156 therapy include the emergence of recombinant adeno-associated virus as the vector of choice, capsid e
158 e treatment of a mouse model of OPMD with an adeno-associated virus-based gene therapy combining comp
162 ntramuscular administration of a recombinant adeno-associated virus-based vector (rAAV vector) expres
167 we lineage-labeled hepatocytes by injecting adeno-associated virus containing thyroxine-binding glob
168 d deep sequencing results confirmed that our adeno-associated virus-CRISPR/Cas9 strategy was very eff
170 e, using a combination of electrophysiology, adeno-associated virus-delivered fluorescent proteins, a
171 iabetes and two models of type 2 diabetes by adeno-associated virus delivery of renin (ReninAAV).
173 2 expression in the rostral striatum through adeno-associated virus effectively disrupted or restored
176 particle-mediated delivery of Cas9 mRNA with adeno-associated viruses encoding a sgRNA and a repair t
177 We investigated whether exosome-associated adeno-associated virus, (exo-AAV) enabled broad retinal
178 ta signaling in the substantia nigra through adeno-associated virus expressing a constitutively activ
180 g 5-HT system by stereotaxic injection of an adeno-associated virus expressing Cre recombinase (AAV-C
181 of stress-susceptible or mice injected with adeno-associated virus expressing shRNA against Cldn5 ca
183 rate that particular groups of drugs enhance adeno-associated virus gene delivery by unknown mechanis
185 tential for FDA-approved drug enhancement of adeno-associated virus gene therapy, which could result
187 n of c-Fos using photoreceptor-specific AAV (adeno-associated virus)-hRK (human rhodopsin kinase)-sh_
189 constructs in mouse rod photoreceptors using adeno-associated virus in Xenopus laevis rod photorecept
190 ng reporter genes into the safe-harbor locus adeno-associated virus integration site 1 in human embry
191 In addition, introduction of 3xMyc-FXR1 via adeno-associated virus into mice leads to the redistribu
192 We used a DREADD, hM3Dq, administered via adeno-associated virus into the LC under a synthetic pro
193 s when the packaging capacity of recombinant adeno-associated virus is limited while tissue-specific
197 nuclein in the adult rat substantia nigra by adeno-associated virus-mediated delivery of a short hair
198 n the R6/2 Huntington's disease mouse model, adeno-associated virus-mediated delivery of CYP46A1 into
199 n CYP46A1 expression in the striatum, via an adeno-associated virus-mediated delivery of selective sh
200 ethylmalonic encephalopathy, liver-targeted adeno-associated virus-mediated ETHE1 gene transfer dram
201 llel, we explored the effects of recombinant adeno-associated virus-mediated expression of Abeta38 an
203 Here we have used anterograde tracing with adeno-associated virus-mediated expression of green fluo
204 llowing high over-expression of WT hAIPL1 by adeno-associated virus-mediated gene delivery, which was
205 u scFvs and tested their efficacy in vivo by adeno-associated virus-mediated gene transfer to the bra
220 R-155) in an in vivo model of PD produced by adeno-associated-virus-mediated expression of alpha-syn.
222 d via stereotactic hippocampal injections of adeno-associated virus particles in APP/PS1 mice, locali
223 n via stereotactic hippocampal injections of adeno-associated virus particles in mutant hAPP Tg mouse
224 ected strategies based on SNTRVAP-displaying adeno-associated virus/phage (AAVP) particles in mice be
227 teral, intranigral injections of recombinant adeno-associated virus pseudotype 2/5 to overexpress wil
229 on of a replication-incompetent, recombinant adeno-associated virus (rAAV) designed to express an ant
232 highly efficient CRISPR directed recombinant Adeno-Associated Virus (rAAV) mediated gene targeting ap
233 that gene therapy mediated by a recombinant adeno-associated virus (rAAV) vector expressing human G6
234 n mice receiving high doses of a recombinant adeno-associated virus (rAAV) vector expressing shRNAs (
235 that the non-tumor-bearing (NT), recombinant adeno-associated virus (rAAV) vector-treated GSD-Ia mice
236 lionic or intrathecal injection, recombinant adeno-associated virus (rAAV) vectors can also infect se
237 tional microdystrophin genes and recombinant adeno-associated virus (rAAV) vectors is an attractive s
238 ne therapy in G6pt-/- mice using recombinant adeno-associated virus (rAAV) vectors, directed by eithe
239 ology, we explored the effect of recombinant adeno-associated virus (rAAV)-mediated overexpression of
241 sought to investigate this using recombinant adeno-associated viruses (rAAV)-mediated expression of g
244 pression of DISC1 in the hippocampus with an adeno-associated virus reduced the levels of BACE1, solu
246 c brain transgenesis was utilized to deliver adeno-associated virus serotype 1 (AAV1) encoding human
248 the inverted terminal repeats (ITRs) in the adeno-associated virus serotype 2 (AAV2) genome signific
250 rated the virus particle, self-complementary adeno-associated virus serotype 2 carrying the mutated g
251 teral dorsal amygdala Ce-region infusions of adeno-associated virus serotype 2 containing the CRF con
252 control of energy homeostasis, via specific adeno-associated virus serotype 2-mediated overexpressio
253 single intravenous dose of a codon-optimized adeno-associated virus serotype 5 (AAV5) vector encoding
254 rphysiological doses of MIS, using either an adeno-associated virus serotype 9 (AAV9) gene therapy ve
255 acy of gene therapy for NPC1, we constructed adeno-associated virus serotype 9 (AAV9) vectors to deli
256 h SMA1 received a single dose of intravenous adeno-associated virus serotype 9 carrying SMN complemen
257 a potent inducer of premature senescence, by adeno-associated virus serotype 9 gene transfer, resulte
258 CRISPR/Cas9-based cardiac gene editing using adeno-associated virus serotype 9 to deliver a single sh
262 ic implications, we found that prior in vivo Adeno-associated virus serotype 9-mediated gene delivery
264 the heart of 7-month-old SJ13 mice using an adeno-associated virus serotype-9 Cre recombinase vector
267 R induced by intraparenchymal delivery of an adeno-associated virus-short hairpin RNA construct was s
268 We used mice with a mutation in Npas2 and adeno-associated virus-short hairpin RNA mediated knockd
269 Cardiac-specific overexpression of FGF16 via adeno-associated virus subtype 9 (AAV9) in the mutant he
270 g within the VTA, we delivered Cre-inducible adeno-associated virus that drives the expression of flu
272 on subretinal or intravitreal injections of adeno-associated virus to deliver the therapeutic gene.
273 c retrograde tracing to label the inputs and adeno-associated virus to trace axonal projections, we i
275 ntify integrations of murine leukemia virus, adeno-associated virus, Tol2 transposons or Ac/Ds transp
276 rat spinal cord after dorsal root crush and adeno-associated virus transgene expression in dorsal ro
277 ene expression using aerosolized delivery of adeno-associated virus type 1 (AAV1) in a large animal m
278 d with our therapeutic vector, a recombinant adeno-associated virus type 1 (rAAV1) expressing our RNA
280 lind sham-surgery-controlled trial assessing adeno-associated virus type 2 (AAV2)-neurturin injected
281 integration site (IS) analysis of wild-type adeno-associated virus type 2 (wtAAV2) in human dermal f
284 G of CXCL10(-/-) mice, using the neurotropic adeno-associated virus type 8 (AAV8) vector, boosted the
285 ation of RS1 via retina-specific delivery of adeno-associated virus type 8-RS1 (AAV8-RS1) vector resc
286 ty and tolerability of escalated doses of an adeno-associated virus vector (AAV) expressing a normal
287 cus aureus) and guide RNA constructs into an adeno-associated virus vector and systemically delivered
288 the use of a dominant-negative forkhead boxO adeno-associated virus vector delivered directly to the
289 We transduced NAcore astrocytes with an adeno-associated virus vector expressing hM3D designer r
290 t NOD scid gamma (NSG) mice by the use of an adeno-associated virus vector, followed by engraftment o
292 we show that expression of these genes from adeno-associated virus vectors in C57BL/6 mice is able t
293 gnaling in a common mouse model of AD, using adeno-associated virus vectors normalized glutamate sign
294 human patients, and gene augmentation using adeno-associated virus vectors robustly sustained the re
295 ts within the packaging limit of recombinant adeno-associated virus vectors that have been shown to b
299 l vectors can package larger transgenes than adeno-associated viruses, yet lentiviral vectors remain
300 liver-specific overexpression of human ZIP8 (adeno-associated virus-ZIP8 [AAV-ZIP8]) resulted in incr
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