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2 Rats received intra-NAc administration of an adeno-associated vector that overexpressed CRF (AAV2/5-C
7 ine dogs with hemophilia A were treated with adeno-associated viral (AAV) gene therapy and followed f
8 e potential utility of a novel combinatorial adeno-associated viral (AAV) gene therapy by expressing
11 r fused to apolipoprotein A-1 by means of an adeno-associated viral (AAV) system in the mouse model o
12 ally precise neural inactivation, we used an adeno-associated viral (AAV) vector carrying the channel
13 many nervous system targets currently is the adeno-associated viral (AAV) vector due to its desirable
14 ms were subsequently cloned into recombinant adeno-associated viral (AAV) vector genome and high-titr
15 crossed with the P23H knock-in mouse and by adeno-associated viral (AAV) vector-mediated gene augmen
16 ut of tyrosine hydroxylase was achieved with adeno-associated viral (AAV) vectors expressing Cas9 or
18 ion by systemically administered recombinant adeno-associated viral (AAV) vectors requires crossing t
23 lar gene therapy post-disease onset using an adeno-associated viral 6 (AAV6) vector carrying a functi
24 to genome editing success to date, including adeno-associated viral and lentiviral vectors for gene t
25 iarrhythmic efficacy of TASK-1 inhibition by adeno-associated viral anti-TASK-1-siRNA (small interfer
30 her excitatory or inhibitory IC neurons, and adeno-associated viral tracings to label excitatory effe
33 LTP, we used in vivo genetic silencing by an adeno-associated viral vector (AAV2) short hairpin RNA a
36 cortical neuron cultures transduced with an adeno-associated viral vector driving the GCaMP6f report
37 ety concerns after subretinal delivery of an adeno-associated viral vector encoding codon-optimized h
38 genic retinal detachment for the delivery of adeno-associated viral vector encoding Rab Escort Protei
42 ear of Ush1c c.216G>A mice using a synthetic adeno-associated viral vector, Anc80L65, shown to transd
43 ion and delivery of this microRNA through an adeno-associated viral vector, treated animals showed ma
44 of human clotting factors IX and X following adeno-associated viral vector-mediated intrauterine gene
45 we have explored the therapeutic efficacy of adeno-associated viral vector-mediated l-DOPA delivery t
47 3 signaling axis, we used local injection of adeno-associated viral vectors (AAVs) encoding ligand-sp
50 ek-old mdx and wild-type (C57BL10) mice with adeno-associated viral vectors encoding either MuSK or r
52 d gene replacement therapies using synthetic adeno-associated viral vectors to deliver the coding seq
55 ts in 34 control and 24 22qDS model mice via adeno-associated viral vectors, driven by excitatory neu
56 iver viral envelopes) and self-complementary adeno-associated viral vectors, serotype-9 (scAAV-9) in
60 bly of 3 types of icosahedral viral capsids: Adeno Associated Virus serotype 2 (AAV2) and Minute Viru
63 ession in 1-month old mdx:utr (-/-) mice via adeno-associated virus (AAV) 9-mediated RNA interference
65 the VACV CRISPR antivirals was tested using adeno-associated virus (AAV) as a packaging vector for b
70 ice at 6 wks were injected unilaterally with adeno-associated virus (AAV) containing either NT3 or GF
71 g RNP delivery with naturally recombinogenic adeno-associated virus (AAV) donor vectors enables site-
72 roteostasis in AMD, we delivered recombinant adeno-associated virus (AAV) encoding Abeta42 and Abeta4
74 uman alpha-synuclein (alpha-syn) fibrils and adeno-associated virus (AAV) expressing human wild-type
75 ats received nodose ganglia injections of an adeno-associated virus (AAV) expressing short hairpin RN
76 PVT involvement in conflict, we injected an adeno-associated virus (AAV) expressing the genetically
77 p an effective long-term therapy for NIU, an adeno-associated virus (AAV) gene therapy approach was u
78 fective therapy for corneal vascularization, adeno-associated virus (AAV) gene therapy, exploiting a
79 al cord via intrathecal administration of an adeno-associated virus (AAV) gene transfer vector signif
81 PR/Cas9 systems that can be packaged into an adeno-associated virus (AAV) hold great promise for gene
88 ating protein (AAP) is a recently discovered adeno-associated virus (AAV) protein that promotes capsi
89 nervous system, we systemically delivered an adeno-associated virus (AAV) serotype 9 carrying the hum
90 ght to determine the efficacy of intrathymic adeno-associated virus (AAV) serotypes to transduce thym
92 placement of SDS-PAGE for purity analysis of adeno-associated virus (AAV) therapeutic products of dif
93 show in adult animals successfully delivers adeno-associated virus (AAV) throughout the cervical, th
94 previously reported compassionate use of an adeno-associated virus (AAV) vector containing the human
96 ed bilateral alBST-targeted injections of an adeno-associated virus (AAV) vector expressing short hai
98 dministered a single intravenous dose of the adeno-associated virus (AAV) vector, AAV-BR1-CAG-NEMO, d
101 e a critical pre-clinical model for studying adeno-associated virus (AAV) vector-mediated gene therap
105 ants identified Anc80L65 from a set of other adeno-associated virus (AAV) vectors as a potent vector
106 that intramuscular or systemic injections of adeno-associated virus (AAV) vectors encoding nuclease-d
109 avoid these issues, and such approaches with adeno-associated virus (AAV) vectors have been shown to
113 anti-HIV monoclonal antibodies (mAbs) using adeno-associated virus (AAV) vectors holds promise for t
118 n mouse are systematically investigated with adeno-associated virus (AAV), an anterograde viral trace
119 ere we present two methods for radiolabeling adeno-associated virus (AAV), one of the most commonly u
120 a similar efficacy by gene augmentation via adeno-associated virus (AAV), providing a proof-of-conce
121 acrRNA)-independent CRISPR-Cpf1 systems with adeno-associated virus (AAV), we were able to build a st
124 lian MG proliferation, along with reports of adeno-associated virus (AAV)-based MG reprogramming and
125 HSV in a well-established mouse model, using adeno-associated virus (AAV)-delivered meganucleases, as
126 iant ribozymes enabled in vivo regulation of adeno-associated virus (AAV)-delivered transgenes, allow
129 ficiency on atherosclerosis using a model of adeno-associated virus (AAV)-induced hypercholesterolemi
130 on as an inflammatory disease model and used adeno-associated virus (AAV)-mediated Ac45 RNA interfere
131 ine for HIV is a crucial public health need; adeno-associated virus (AAV)-mediated antibody gene deli
132 we report a genome editing approach in which adeno-associated virus (AAV)-mediated CRISPR/Cas9 delive
133 ment of a mouse model of SARS-CoV-2 based on adeno-associated virus (AAV)-mediated expression of hACE
136 Primary adult rat ventricular myocytes, adeno-associated virus (AAV)-mediated gene delivery in m
139 Here, we describe the development of an adeno-associated virus (AAV)-mediated gene therapy to tr
140 To address this challenge, we first used adeno-associated virus (AAV)-mediated gene transfer to p
145 enic mice that receive systemic injection of adeno-associated virus (AAV)2/1-carrying truncated gRNAs
146 D1(+/L144F) MNs, and intrathecal delivery of adeno-associated virus (AAV)9-mir-17~92 improves motor d
148 dy(2j)/dy(2j) mouse model of MDC1A using an adeno-associated virus (AAV9) carrying a catalytically i
149 gene was delivered to the brains of mice by adeno-associated virus (AAV9) via stereotactic injection
152 The recent market approvals of recombinant adeno-associated virus (rAAV) gene therapies in Europe a
153 trolled release of a therapeutic recombinant adeno-associated virus (rAAV) vector overexpressing the
154 tional microdystrophin genes and recombinant adeno-associated virus (rAAV) vectors is an attractive s
155 Transplantation of PCs pretransfected with adeno-associated virus 1-vascular endothelial growth fac
156 che for either virus to replicate.IMPORTANCE Adeno-associated virus 2 (AAV2) differs from most other
160 ntrols, were given injections of recombinant adeno-associated virus 8 vector that expressed the prima
161 d (fl/fl) Shc mice on FFD were injected with adeno-associated virus 8-thyroxine-binding globulin-Cre-
163 n-human primate model of HIV infection, with adeno-associated virus 9 (AAV9)-CRISPR/Cas9 gene editing
164 /iodide symporter) to noninvasively quantify adeno-associated virus 9 (AAV9)-mediated gene expression
166 Here, we demonstrate that a recombinant adeno-associated virus 9 (rAAV9) is highly effective for
168 ic mice were injected with M7.8L packaged in adeno-associated virus 9 at 3 days of age and 60 days of
169 phy, we performed intramuscular injection of adeno-associated virus 9 carrying the TIPE2 gene in mdx
170 1-1-93 and treated with a sCAR-Fc expressing adeno-associated virus 9 vector 1, 3, and 7 days after C
171 exclusively in cardiomyocytes by using AAV9 (adeno-associated virus 9) to deliver multiplexed single
173 ior descending coronary artery ligation, and adeno-associated virus 9-mediated in vivo overexpression
176 hindered by large size (limiting delivery by adeno-associated virus [AAV] vectors), off-target editin
177 l of SCLC by delivering an adenovirus (or an adeno-associated virus [AAV]) that expresses Cre recombi
178 bout 75 Snord115 genes were packaged into an adeno-associated virus and administered to a mouse model
179 D1-MSNs versus D2-MSNs using a Cre-inducible adeno-associated virus and Cre lines during cocaine cond
182 versely, overexpression of VZVsncRNA13 using adeno-associated virus consistently increased VZV spread
185 tment of the gyrencephalic brain by systemic adeno-associated virus delivery in human neurological di
187 iabetes and two models of type 2 diabetes by adeno-associated virus delivery of renin (ReninAAV).
188 reated with a single intrathecal infusion of adeno-associated virus encoding a microRNA targeting SOD
190 hydrodynamic injection or transduction with adeno-associated virus encoding the HBV genome (AAV-HBV)
192 of stress-susceptible or mice injected with adeno-associated virus expressing shRNA against Cldn5 ca
193 es; disruption of anchoring in vivo using an adeno-associated virus gene therapy vector inhibited car
197 In addition, introduction of 3xMyc-FXR1 via adeno-associated virus into mice leads to the redistribu
198 of CRISPRa, we injected CRISPRa-recombinant adeno-associated virus into the hypothalamus, which led
199 e injection of an engineered virus, often an adeno-associated virus or herpes simplex virus, among ma
200 s9 mRNA, compared with cells transduced with adeno-associated virus or lentivirus expressing SaCas9.
202 gets by performing intraspinal injections of adeno-associated virus serotype 2 (AAV2)-BDNF vector.
203 elation to recent clinical translation in an adeno-associated virus serotype 2-mediated human aromati
205 single intravenous dose of a codon-optimized adeno-associated virus serotype 5 (AAV5) vector encoding
209 23a was expressed using a self-complementary adeno-associated virus serotype 9 (scAAV9) viral vector
215 CRISPR/Cas9-based cardiac gene editing using adeno-associated virus serotype 9 to deliver a single sh
217 ic implications, we found that prior in vivo Adeno-associated virus serotype 9-mediated gene delivery
220 Pak2 activation by genetic overexpression or adeno-associated virus serotype-9-based gene delivery wa
221 Moreover, IRE-1 activator, Quercetin, and adeno-associated virus serotype-9-delivered XBP-1s were
222 Cbeta was genetically targeted using a novel adeno-associated virus shuttle vector to deliver microRN
223 ticular administration of miR-204-expressing adeno-associated virus significantly decelerates OA prog
224 g within the VTA, we delivered Cre-inducible adeno-associated virus that drives the expression of flu
225 er a single intravenous administration of an adeno-associated virus that encodes CRISPR (AAV-CRISPR).
226 ice (NTS-HIF-1alpha(-/-) ) by microinjecting adeno-associated virus that expressed Cre-recombinase in
227 injected into NAc variants of a new designer adeno-associated virus that permits robust retrograde ac
228 from a transgene or after infection with an adeno-associated virus that transferred an overlength HB
229 in mice, using Cre recombinase delivered by adeno-associated virus to knockout endogenous hepatic In
231 of HCC in patients with hepatitis B virus or adeno-associated virus type 2 infection might involve ac
232 patients infected with hepatitis B virus or adeno-associated virus type 2, due to integration of the
233 fferent transgenic Cre mice or injected with adeno-associated virus type 8 (AAV8) Cre and fed diets t
234 cus aureus) and guide RNA constructs into an adeno-associated virus vector and systemically delivered
235 SG -( K(b) D(b)) (null) ( IA(null)) mice via adeno-associated virus vector increased human CD45(+) ce
237 F21 by administration of an FGF21-expressing adeno-associated virus vector recapitulated these compli
239 erapy approach for Alzheimer's disease using adeno-associated virus vector-based knockdown of CD33 re
240 receptor retargeting.IMPORTANCE Recombinant adeno-associated virus vectors (rAAVs), based on AAV8 an
243 atory elements in the context of recombinant adeno-associated virus vectors has enabled cell-type-res
244 gnaling in a common mouse model of AD, using adeno-associated virus vectors normalized glutamate sign
245 human patients, and gene augmentation using adeno-associated virus vectors robustly sustained the re
247 nd loss of KLF5 function approaches and AAV (adeno-associated virus)-mediated Klf5 delivery in mice s
248 wild-type mice via stereotaxic injection of adeno-associated virus, we found that adult cerebellar n
250 was also increased in CPVT mice, and further adeno-associated virus-9-induced overexpression of SN at
252 e treatment of a mouse model of OPMD with an adeno-associated virus-based gene therapy combining comp
254 d deep sequencing results confirmed that our adeno-associated virus-CRISPR/Cas9 strategy was very eff
255 by a single-dose systemic administration of adeno-associated virus-delivered CRISPR-Cas9 components
257 noted similar results in mice infected with adeno-associated virus-expressing small hairpin RNAs tar
258 gulation and that induction of NRF2 using an adeno-associated virus-mediated approach in hepatocytes
259 of spared axons, using projection-specific, adeno-associated virus-mediated chemogenetic neuronal si
260 imary striatal cell culture model of HD, and adeno-associated virus-mediated Elk-1 overexpression all
262 ene deletion in the NAc in adult mice, using adeno-associated virus-mediated expression of cre recomb
263 tem, we intravitreally delivered recombinant adeno-associated virus-mediated expression of soluble Fm
266 sue of the JCI, Wang et al. demonstrate that adeno-associated virus-mediated overexpression of TGF-be
270 R induced by intraparenchymal delivery of an adeno-associated virus-short hairpin RNA construct was s
275 replication, we constructed two recombinant adeno-associated-virus 2 (rAAV2) vectors encoding CIP fu
278 ockout (L-G6pc(-/-) ) mice were treated with adeno-associated viruses (AAVs) 2 or 8 directed against
283 es of CRISPR-associated protein 9 (Cas9) and adeno-associated viruses (AAVs) evades adaptive immune r
285 in defined cell types, recombinase-dependent adeno-associated viruses (AAVs) have become the tool of
286 fficient AAV genome transcription.IMPORTANCE Adeno-associated viruses (AAVs) have proven to be effect
287 rity and tight junction integrity.IMPORTANCE Adeno-associated viruses (AAVs) have recently emerged at
289 sting neutralizing antibodies (NAbs) against adeno-associated viruses (AAVs) pose a major, unresolved
290 ified here by stamping cultured neurons with adeno-associated viruses (AAVs), is completed in a few m
293 ved by transducing the BSCs with recombinant adeno-associated viruses (rAAVs) that express alpha-synu
294 o synaptic damage in vivo, we have generated adeno-associated viruses AAV-Tat and AAV-Exo-Tat viruses
295 eta receptor 2 [sTGFbetaR2]) delivered using adeno-associated viruses and explored their ability to m
299 of tauopathy utilizing neonatal delivery of adeno-associated viruses expressing wild-type (WT) or mu