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1 ugh to be packaged efficiently into an adeno-associated virus.
2 ingle systemic delivery of recombinant adeno-associated virus.
3 mice and mice injected with 3xMyc-FXR1 adeno-associated virus.
4 ine heart was performed by means of an adeno-associated virus.
5 onor template delivered by recombinant adeno-associated virus.
6 -229E may constitute a descendant of camelid-associated viruses.
7 ty of gene therapy through infusion of adeno-associated virus 1 (AAV1)/SERCA2a in patients with heart
8 mice were intracranially injected with adeno-associated virus 1 expressing murine IL-6 (AAV1-mIL-6).
10 tudied mostly in the dependoparvovirus adeno-associated virus 2 (AAV2) and the protoparvovirus minute
12 r either virus to replicate.IMPORTANCE Adeno-associated virus 2 (AAV2) differs from most other viruse
14 rAAV2/HBoV1, in which the recombinant adeno-associated virus 2 (rAAV2) genome is pseudopackaged by t
17 acy of gene therapy with a recombinant adeno-associated virus 2/2 (rAAV2/2) vector carrying the RPE65
18 determine the effects of Grapevine Leafroll associated Virus 3 (GLRaV-3) on fruit composition and ch
20 s performed in mice given infusions of adeno-associated virus 6-NF-kappaB-luciferase, to assess activ
22 , were given injections of recombinant adeno-associated virus 8 vector that expressed the primary miR
23 proof-of-concept experiments, we used Adeno-Associated Virus 9 (AAV9) to deliver single-guide RNA (s
25 L by generating two self-complementary adeno-associated virus 9 (scAAV9) constructs to address CLN3 d
26 approach using two self-complementary adeno-associated virus 9 (scAAV9) constructs to address CLN3 d
27 In the present study, we delivered adeno-associated virus 9 carrying green fluorescent protein-CI
28 erolaemia liver chimeric mice using an adeno-associated virus 9-based gene therapy and restore normal
31 stnatal muscle tissue in vivo, we used adeno-associated virus-9 (AAV9) to deliver gene-editing compon
34 nuclease mRNA via electroporation and adeno-associated virus (AAV) 6 delivery of donor constructs in
35 ssessed the therapeutic efficacy of an adeno-associated virus (AAV) 9-mbetagal vector infused systemi
40 delivery with naturally recombinogenic adeno-associated virus (AAV) donor vectors enables site-specif
41 tasis in AMD, we delivered recombinant adeno-associated virus (AAV) encoding Abeta42 and Abeta40 pept
44 e therapy for corneal vascularization, adeno-associated virus (AAV) gene therapy, exploiting a natura
45 d via intrathecal administration of an adeno-associated virus (AAV) gene transfer vector significantl
51 owever, in vivo delivery of mAgrin via adeno-associated virus (AAV) into FKRP mutant mice was unable
55 y using nonintegrating viruses such as adeno-associated virus (AAV) is not optimal in this setting be
56 The life cycle of the human parvovirus adeno-associated virus (AAV) is orchestrated by four Rep prote
57 Intracellular transport of recombinant adeno-associated virus (AAV) is still incompletely understood.
61 protein (AAP) is a recently discovered adeno-associated virus (AAV) protein that promotes capsid asse
62 ) mRNA with donor template delivery by adeno-associated virus (AAV) serotype 6 vectors directs effici
64 ent of SDS-PAGE for purity analysis of adeno-associated virus (AAV) therapeutic products of different
69 ously reported compassionate use of an adeno-associated virus (AAV) vector containing the human AADC
70 subretinal injection of a recombinant adeno-associated virus (AAV) vector containing the RPE65 gene
72 in the dorsal striatum, we injected an adeno-associated virus (AAV) vector producing a short hairpin
73 IDUA production and function following adeno-associated virus (AAV) vector transduction of MPS1 patie
74 tered a single intravenous dose of the adeno-associated virus (AAV) vector, AAV-BR1-CAG-NEMO, deliver
75 the brains of transgenic mice using an adeno-associated virus (AAV) vector, decreased parenchymal Abe
76 rudele et al describe a novel study of adeno-associated virus (AAV) vector-mediated gene therapy that
80 dentified Anc80L65 from a set of other adeno-associated virus (AAV) vectors as a potent vector for th
82 gramming of MFs into hepatocytes using adeno-associated virus (AAV) vectors expressing hepatic transc
83 trocytic CN/NFAT pathway in rats using adeno-associated virus (AAV) vectors expressing the astrocyte-
86 Immune responses in gene therapy with adeno-associated virus (AAV) vectors have been the object of a
90 cardiac overexpression of HMGB1 using adeno-associated virus (AAV) vectors induced inflammation in t
92 n, Y704A, near the 2-fold interface of adeno-associated virus (AAV) was defective for transcription o
94 grade functionality into the capsid of adeno-associated virus (AAV), a vector that has shown promise
95 e are systematically investigated with adeno-associated virus (AAV), an anterograde viral tracer.
96 present two methods for radiolabeling adeno-associated virus (AAV), one of the most commonly used vi
97 ign, RAM can be packaged into a single adeno-associated virus (AAV), providing great versatility and
98 n the life cycle of a human virus, the adeno-associated virus (AAV), that causes no known disease but
99 Second, we package the library into adeno-associated virus (AAV), thereby allowing delivery to tar
101 Here, we describe a lineage-specific, adeno-associated virus (AAV)-derived endogenous viral element
102 inal neurons specifically by injecting adeno-associated virus (AAV)-expressing Cre-dependent DREADD (
106 ort a genome editing approach in which adeno-associated virus (AAV)-mediated CRISPR/Cas9 delivery to
107 C cardiac manifestations in mice after adeno-associated virus (AAV)-mediated gene delivery of mutant
112 We sought to develop an efficient adeno-associated virus (AAV)-mediated RNAi gene therapy for AL
113 the therapeutic potential of naked and adeno-associated virus (AAV)-packaged AONs in vitro and in viv
114 ut, and the right CST was treated with adeno-associated virus (AAV)-Sox11 or AAV-EBFP control, along
115 strophy, dystrophin restoration during adeno-associated virus (AAV)-U7-mediated exon-skipping therapy
116 intrahippocampal administration of the adeno-associated virus (AAV)-vectored anti-phospho-tau antibod
117 These aptayzmes efficiently regulated adeno-associated virus (AAV)-vectored transgene expression in
121 cacy of telomerase activation by using adeno-associated virus (AAV)9 gene therapy vectors carrying th
122 erformed intramuscular injection of an adeno-associated virus (AAV)9 vector expressing GAA (AAV9-hGAA
123 To address this, we developed a novel adeno-associated virus (AAV-GLP-1R) that utilizes short hairpi
128 hology, we investigated the effects of adeno-associated virus (AAV2/1)-mediated expression of Interle
133 ents challenges in packaging it within adeno-associated viruses (AAVs) for clinical applications.
135 neutralizing antibodies (NAbs) against adeno-associated viruses (AAVs) pose a major, unresolved chall
136 Despite the fact that a majority of adeno-associated viruses (AAVs) utilize sialic acid (SIA) for
138 neurons in both the MPP(+)-lesioned or adeno-associated virus alpha-synuclein rat models of Parkinson
139 s versus D2-MSNs using a Cre-inducible adeno-associated virus and Cre lines during cocaine conditione
140 specific molecular interaction between adeno-associated virus and its host cell, which can be rapidly
141 trehalose also inhibited production of cell-associated virus and partially blocked the reduction in
144 pG2 cells after HBV genome delivery by adeno-associated virus, and stable expression of NTCP in a ccH
146 y include the emergence of recombinant adeno-associated virus as the vector of choice, capsid enginee
148 tment of a mouse model of OPMD with an adeno-associated virus-based gene therapy combining complete k
151 Leading the way has been the use of adeno-associated virus-based strategies for factor IX gene tra
152 scular administration of a recombinant adeno-associated virus-based vector (rAAV vector) expressing t
153 ong other recently identified human-like bat-associated viruses because of its high sequence homology
158 neage-labeled hepatocytes by injecting adeno-associated virus containing thyroxine-binding globulin p
160 sequencing results confirmed that our adeno-associated virus-CRISPR/Cas9 strategy was very efficient
162 ng a combination of electrophysiology, adeno-associated virus-delivered fluorescent proteins, analysi
165 ession in the rostral striatum through adeno-associated virus effectively disrupted or restored dopam
168 le-mediated delivery of Cas9 mRNA with adeno-associated viruses encoding a sgRNA and a repair templat
172 nvestigated whether exosome-associated adeno-associated virus, (exo-AAV) enabled broad retinal target
173 naling in the substantia nigra through adeno-associated virus expressing a constitutively active type
175 system by stereotaxic injection of an adeno-associated virus expressing Cre recombinase (AAV-Cre) in
176 ress-susceptible or mice injected with adeno-associated virus expressing shRNA against Cldn5 caused i
178 n this study, we show that a ronivirus, gill-associated virus (GAV), encodes the 2'-O-MTase activity,
179 hat particular groups of drugs enhance adeno-associated virus gene delivery by unknown mechanisms.
181 l for FDA-approved drug enhancement of adeno-associated virus gene therapy, which could result in saf
186 -Fos using photoreceptor-specific AAV (adeno-associated virus)-hRK (human rhodopsin kinase)-sh_c-fos
188 ucts in mouse rod photoreceptors using adeno-associated virus in Xenopus laevis rod photoreceptors us
191 orter genes into the safe-harbor locus adeno-associated virus integration site 1 in human embryonic s
192 dition, introduction of 3xMyc-FXR1 via adeno-associated virus into mice leads to the redistribution o
193 used a DREADD, hM3Dq, administered via adeno-associated virus into the LC under a synthetic promoter,
194 the packaging capacity of recombinant adeno-associated virus is limited while tissue-specific delive
197 age, which is by far the most abundant human-associated virus known, comprising up to 90% of sequence
198 Systemic delivery of dCas9/gRNA by adeno-associated virus led to reductions in pathological RNA f
200 rs virus function, and we suggest that tumor-associated viruses may be more likely to contain DNA mix
203 n in the adult rat substantia nigra by adeno-associated virus-mediated delivery of a short hairpin RN
204 R6/2 Huntington's disease mouse model, adeno-associated virus-mediated delivery of CYP46A1 into the s
205 6A1 expression in the striatum, via an adeno-associated virus-mediated delivery of selective shCYP46A
206 malonic encephalopathy, liver-targeted adeno-associated virus-mediated ETHE1 gene transfer dramatical
207 we explored the effects of recombinant adeno-associated virus-mediated expression of Abeta38 and Abet
209 we have used anterograde tracing with adeno-associated virus-mediated expression of green fluorescen
210 g high over-expression of WT hAIPL1 by adeno-associated virus-mediated gene delivery, which was stabl
211 s and tested their efficacy in vivo by adeno-associated virus-mediated gene transfer to the brain of
214 Three approaches were compared: 1) adeno-associated virus-mediated in utero transfer of the ChR-2
227 stereotactic hippocampal injections of adeno-associated virus particles in APP/PS1 mice, localized pr
228 stereotactic hippocampal injections of adeno-associated virus particles in mutant hAPP Tg mouse brain
229 strategies based on SNTRVAP-displaying adeno-associated virus/phage (AAVP) particles in mice bearing
232 intranigral injections of recombinant adeno-associated virus pseudotype 2/5 to overexpress wildtype
236 efficient CRISPR directed recombinant Adeno-Associated Virus (rAAV) mediated gene targeting approach
237 gene therapy mediated by a recombinant adeno-associated virus (rAAV) vector expressing human G6Pase-a
238 receiving high doses of a recombinant adeno-associated virus (rAAV) vector expressing shRNAs (rAAV-s
239 he non-tumor-bearing (NT), recombinant adeno-associated virus (rAAV) vector-treated GSD-Ia mice (AAV-
240 or intrathecal injection, recombinant adeno-associated virus (rAAV) vectors can also infect sensory
241 microdystrophin genes and recombinant adeno-associated virus (rAAV) vectors is an attractive strateg
242 rapy in G6pt-/- mice using recombinant adeno-associated virus (rAAV) vectors, directed by either the
244 to investigate this using recombinant adeno-associated viruses (rAAV)-mediated expression of green f
247 on of DISC1 in the hippocampus with an adeno-associated virus reduced the levels of BACE1, soluble Ab
248 Hepatic arginase 1 gene therapy using adeno-associated virus rescued nearly all these abnormalities
249 n transgenesis was utilized to deliver adeno-associated virus serotype 1 (AAV1) encoding human mutant
251 nverted terminal repeats (ITRs) in the adeno-associated virus serotype 2 (AAV2) genome significantly
252 the virus particle, self-complementary adeno-associated virus serotype 2 carrying the mutated gene (s
253 dorsal amygdala Ce-region infusions of adeno-associated virus serotype 2 containing the CRF construct
254 ol of energy homeostasis, via specific adeno-associated virus serotype 2-mediated overexpression of I
255 intravenous dose of a codon-optimized adeno-associated virus serotype 5 (AAV5) vector encoding a B-d
256 ological doses of MIS, using either an adeno-associated virus serotype 9 (AAV9) gene therapy vector o
257 gene therapy for NPC1, we constructed adeno-associated virus serotype 9 (AAV9) vectors to deliver th
258 received a single dose of intravenous adeno-associated virus serotype 9 carrying SMN complementary D
259 nt inducer of premature senescence, by adeno-associated virus serotype 9 gene transfer, resulted in a
260 /Cas9-based cardiac gene editing using adeno-associated virus serotype 9 to deliver a single short gu
263 lications, we found that prior in vivo Adeno-associated virus serotype 9-mediated gene delivery of GJ
265 eart of 7-month-old SJ13 mice using an adeno-associated virus serotype-9 Cre recombinase vector (AAV.
267 ced by intraparenchymal delivery of an adeno-associated virus-short hairpin RNA construct was suffici
268 used mice with a mutation in Npas2 and adeno-associated virus-short hairpin RNA mediated knockdown of
269 c-specific overexpression of FGF16 via adeno-associated virus subtype 9 (AAV9) in the mutant hearts p
270 in the VTA, we delivered Cre-inducible adeno-associated virus that drives the expression of fluoresce
271 Here we describe a novel recombinant adeno-associated virus that restricts gene expression to GABAe
273 ograde tracing to label the inputs and adeno-associated virus to trace axonal projections, we identif
275 integrations of murine leukemia virus, adeno-associated virus, Tol2 transposons or Ac/Ds transposons,
276 pinal cord after dorsal root crush and adeno-associated virus transgene expression in dorsal root gan
278 pression using aerosolized delivery of adeno-associated virus type 1 (AAV1) in a large animal model o
280 ham-surgery-controlled trial assessing adeno-associated virus type 2 (AAV2)-neurturin injected into t
282 XCL10(-/-) mice, using the neurotropic adeno-associated virus type 8 (AAV8) vector, boosted the numbe
283 of RS1 via retina-specific delivery of adeno-associated virus type 8-RS1 (AAV8-RS1) vector rescues mo
284 tolerability of escalated doses of an adeno-associated virus vector (AAV) expressing a normal ND4 co
285 reus) and guide RNA constructs into an adeno-associated virus vector and systemically delivered them
286 e transduced NAcore astrocytes with an adeno-associated virus vector expressing hM3D designer recepto
287 scid gamma (NSG) mice by the use of an adeno-associated virus vector, followed by engraftment of huma
289 ow that expression of these genes from adeno-associated virus vectors in C57BL/6 mice is able to indu
290 g in a common mouse model of AD, using adeno-associated virus vectors normalized glutamate signaling
291 patients, and gene augmentation using adeno-associated virus vectors robustly sustained the rescue o
292 hin the packaging limit of recombinant adeno-associated virus vectors that have been shown to be safe
297 bited incomplete neutralization against cell-associated virus with T/F Envs, which was not observed w
298 that transcytosis of both cell-free and cell-associated viruses with diverse envelopes was significan
299 ors can package larger transgenes than adeno-associated viruses, yet lentiviral vectors remain largel
300 specific overexpression of human ZIP8 (adeno-associated virus-ZIP8 [AAV-ZIP8]) resulted in increased
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