ライフサイエンスコーパス: AAV

1 AAV are used to express in vivo mAKAPbeta-derived RSK3 a

2 AAV serotypes were injected intrathymically into wild-ty

3 AAV vector serotype DJ efficiently transduced CDKL5-muta

4 AAV vectors typically use a B-domain-deleted FVIII trans

5 AAV-Cre-mediated SIRT1 knockdown in the medial prefronta

6 AAV-mediated delivery of TGF-beta1 rescued degenerating

7 AAV-mediated, conditional Rit2 knockdown in DAergic neur

8 AAV-RSPO1-Fc-transduced Apc(Min/+) mice also developed f

9 AAV-Slc25a46 treatment was able to rescue the premature

10 AAVs coding for glutaredoxin-1 (Glrx) shRNA successfully

11 AAVs have a complex and unique pathogenesis, with eviden

12 peripheral T cells harboring approximately 1 AAV genome per cell persisted for more than 40 weeks, an

13 stingly neither AAV-M3 alone nor AAV-IL-10 + AAV-M3 extend survival beyond that of the AAV-IL-10 alon

14 Accordingly, global Jnk1 ablation, AAV-mediated Dusp8 overexpression in the mediobasal hypo

15 erved among the pipelines when low abundance AAVs are concerned, likely due to differences in their N

16 e the sensitivity in detecting low abundance AAVs, with frequencies between 1~20%, is less a concern

17 at the AAV small hairpin RNA targeting Ac45 (AAV-sh-Ac45) impaired cellular acidification, extracellu

18 , our studies are among the first to acheive AAV-mediated, conditional and inducible DAergic knockdow

19 NHEJ knock-in of BDDF8 at Alb introns after AAV-mediated delivery of editing components.

20 in the induction of humoral immunity against AAV serotypes.

21 strategy modulates the IgG response against AAV cargos.

22 ly modulate the host immune response against AAV-mediated gene therapy and influence the course of se

23 uld explore higher doses of AAV, alternative AAV serotypes and gene expression cassettes, or other br

24 We subsequently created an AAV vector for Cre-dependent gRNA expression as well as

25 address both of these issues we designed an AAV vector that uses mutant "cross-over insensitive" rec

26 validated a novel biosensor technology in an AAV system by using an NFkappaB response element and rev

27 To do this, we injected an AAV into the PB region to deliver a Cre-dependent antero

28 and is compact enough to be packaged into an AAV for genome editing.

29 Following systemic administration of an AAV-uUtro to neonatal dystrophin-deficient mdx mice, his

30 as efficiently delivered to MCC cells via an AAV vector.

31 Male Vglut2-Cre mice were injected with an AAV-DIO-ChR2-mCherry reporter in CA1 hippocampus.

32 er, white adipose tissues, and heart with an AAV-shRNA (Grb14-shRNA) improves glucose homeostasis in

33 In vivo co-treatment with AAV-miRzip-21 and AAV-miR-7 in mice bearing malignant brain tumors resulte

34 ain and loss of KLF5 function approaches and AAV (adeno-associated virus)-mediated Klf5 delivery in m

35 ore significantly than that by AAV-sh-C1 and AAV-sh-CtsK Unbiased genome-wide transcriptome sequencin

36 immunogenic in humans, as healthy human and AAV patient sera contain anti-6PGD and anti-6PGD(391-410

37 escues the defective ATP2C1 KO phenotype and AAV transduction in vitro Conversely, the calcium ionoph

38 nerated adeno-associated viruses AAV-Tat and AAV-Exo-Tat viruses.

39 r cell persisted for more than 40 weeks, and AAV vector integration was detected.

40 enous Ig, IdeS administration decreased anti-AAV antibodies and enabled efficient liver gene transfer

41 ere, we studied if IdeS could eliminate anti-AAV antibodies in the context of gene therapy.

42 dy has described the high prevalence of anti-AAV neutralizing antibodies among domestic cats in Switz

43 Strategies aimed at overcoming anti-AAV antibodies are being studied(7), which often involve

44 Finally, IdeS reduced anti-AAV antibody levels from human plasma samples in vitro,

45 samples and by analyzing their properties as AAV/HBoV1 gene transfer vectors.

46 hypothesis that critical properties, such as AAV capsid antibody evasion and tropism, can be coevolve

47 their specificity dramatically decreased at AAV frequencies <2%, suggesting that 2% threshold may be

48 in the murine brain using BAPTA-AM augments AAV gene expression in vivo Taking these data together,

49 Next to the gold-standard iodixanol-based AAV vector production, we recently published a protocol

50 Building upon our Cre-recombination-based AAV targeted evolution (CREATE) platform, we developed M

51 IdeS treatment before AAV vector infusion was safe and resulted in enhanced li

52 AAVR binds between AAV's spikes on a plateau that is conserved, except in o

53 in, which disrupts calcium gradients, blocks AAV transduction.

54 Suppression of early-stage neuronal death by AAV-YAPdeltaC reduces the later-stage extracellular Abet

55 ation of Sema3G levels in the hippocampus by AAV transfection.

56 deno-associated viruses (AAVs) is limited by AAV packaging capacity, which precludes the use of full-

57 first evidence that gene therapy mediated by AAV vectors can be used for treating CDKL5 disorder.

58 tion of injured RGCs and optic nerve (ON) by AAV-mediated CRISPR/Cas9 inhibition of genes that are cr

59 K secretion more significantly than that by AAV-sh-C1 and AAV-sh-CtsK Unbiased genome-wide transcrip

60 ities, limitations, and progress in clinical AAV gene therapy.

61 demonstrated that gene therapy that combines AAV-mSncg promoter with clustered regularly interspaced

62 Finally, combining AAV-ISL2-shRNA with temozolomide suppressed oligodendrog

63 Complementary AAV-based gene therapies constitute rationally-designed

64 rt hairpin RNAs targeting GHSR (or a control AAV) for RNAi-mediated VAN-specific GHSR knockdown.

65 pecific cortical neurons using Cre-dependent AAV vectors and for mapping inputs to such neurons using

66 advances in developing clinically desirable AAV capsids, optimizing genome designs and harnessing re

67 f ATP2C1 decreases transduction by different AAV serotypes.

68 optimized therapeutic cassette in different AAV systems provides long-term correction of copper meta

69 t the clinical development of liver-directed AAV gene therapy for hemophilia A, while emphasizing the

70 We adopted a dual AAV approach using two different recombinant vectors, on

71 Here, we report the application of dual AAVs for the delivery of split cytosine and adenine base

72 Optimized dual AAVs enable in vivo base editing at therapeutically rele

73 ers, the Cre/lox system, and genome editing, AAVs represent a practical, rapid, and economical altern

74 for priming the capsid to support efficient AAV genome transcription.IMPORTANCE Adeno-associated vir

75 ness this structural information to engineer AAV capsid libraries through saturation mutagenesis of d

76 t inform the design of functionally enriched AAV libraries and accelerate therapeutic candidate ident

77 To expand their utility, we evolved AAV capsids to efficiently transduce specific cell types

78 ic pathogen-free (SPF) cats for pre-existing AAV-binding antibodies against the 11 serotypes.

79 amma2 subunit reduction in the VTA following AAV-Cre-GFP infusion in floxed Gabrg2 mice.

80 Our results provide proof of concept for AAV-delivered mAbs to produce a "functional cure." Howev

81 ery in adult animals is highly effective for AAV-mediated gene delivery throughout the spinal cord an

82 , have identified host factors essential for AAV cell entry, but no genome-wide screens that address

83 uction, we recently published a protocol for AAV production based on chloroform-precipitation, which

84 The receptor for AAV (AAVR; also named KIAA0319L) was recently identified

85 c repressor can be an effective strategy for AAV-based CRISPR therapies, as this pathway serves as a

86 ta indicate that CD33 is a viable target for AAV-based knockdown strategies to reduce AD pathology.

87 ells display both full-length and fragmented AAV genomes at Cas9 on-target sites.

88 Furthermore, AAV-sh-Ac45 significantly reduced osteoclast formation a

89 no-associated virus encoding the HBV genome (AAV-HBV).

90 is an unmet need to comprehensively improve AAV tissue tropism, transduction efficiency, and antibod

91 is an unmet need to comprehensively improve AAV vector properties.

92 nd concomitantly provides avenues to improve AAV/HBoV1 gene transfer vectors.

93 ase 1 [SPCA1]) encoded by the ATP2C1 gene in AAV infection.

94 Although any tissue can be involved in AAV, the upper and lower respiratory tract and kidneys a

95 porting threshold for ensured specificity in AAV calling and reporting.

96 fficacy of remission-induction treatments in AAV with severe renal involvement.

97 Vector engineering can increase AAV transduction efficiency (by optimizing the transgene

98 ng demonstrates that both HSV and individual AAV serotypes are non-randomly distributed among neurona

99 understanding of host factors that influence AAV trafficking and transduction is still evolving.

100 understanding of host factors that influence AAV transduction in different cell types is still evolvi

101 of how the host cell environment influences AAV transduction is still evolving.

102 ATP2C1 KO does not appear to inhibit AAV binding, cellular uptake, or nuclear entry; however,

103 Intravenously injected AAV-Tat or AAV-Exo-Tat mainly infects liver and heart.

104 avioral control during conflict, we injected AAV expressing the inhibitory hM4Di DREADD and determine

105 pression related significantly to integrated AAV in the absence of clinical toxicity, supporting its

106 Interestingly, AAV-sh-Ac45 impaired mature cathepsin K secretion more s

107 Intrathymic AAV-transduced progenitors promote a rapid restoration o

108 show that, although antibodies binding known AAV serotypes (AAV1 to AAV11) are prevalent in cats livi

109 ane-associated accessory protein that limits AAV production through competitive exclusion.

110 vating protein (AAP) is required for maximum AAV production and has multiple roles in capsid assembly

111 Moreover, AAV-mediated therapy successfully prevented clinical neu

112 ositive AAV (PR3-AAV), MPO-positive AAV (MPO-AAV) and, for EGPA, by the presence or absence of ANCA (

113 Here, we solve the structures of a natural AAV isolate complexed with antibodies using cryo-electro

114 Interestingly neither AAV-M3 alone nor AAV-IL-10 + AAV-M3 extend survival beyo

115 Slc25a46) and treated them with neurotrophic AAV-PHP.B vector carrying the mouse Slc25a46 coding sequ

116 ese antibodies do not necessarily neutralize AAV infectivity.

117 esence of a helper adenovirus to yield a new AAV variant that then serves as a template for evolving

118 ing opportunities for the development of new AAV vectors for gene therapy.

119 Interestingly neither AAV-M3 alone nor AAV-IL-10 + AAV-M3 extend survival beyond that of the AA

120 generating and systemically delivering novel AAV variants was not previously available.

121 library design strategies to generate novel AAV capsid variants.

122 tion, we observed a defect in the ability of AAV capsids to undergo conformational changes and suppor

123 terminant that restricts the availability of AAV glycan attachment factors on the cell surface by mai

124 ll, our studies show the structural basis of AAV Rep's structural flexibility required to fulfill its

125 iled knowledge of the fundamental biology of AAV as well as cooperative international research and cl

126 indings provide insights into the biology of AAV entry with high-resolution details, providing opport

127 ntified, and the precise characterization of AAV-AAVR recognition is in immediate demand.

128 Thus, localized ocular gene delivery of AAV-HLA-G1/5 may reduce the off-target risks and establi

129 Our results showed that local delivery of AAV-sh-Ac45 in periapical tissues in bacterium-induced i

130 T cell transduction through the detection of AAV genomes and transgene mRNA, and show that intracellu

131 uture studies should explore higher doses of AAV, alternative AAV serotypes and gene expression casse

132 g early pathogenesis, safety and efficacy of AAV-intrauterine gene transfer (IUGT) requires assessmen

133 These findings establish the feasibility of AAV-mediated in vivo gene delivery to immune cells which

134 ths was associated with a lower incidence of AAV relapse compared with standard maintenance therapy.

135 10 ug; n = 4), or intramuscular injection of AAV (10 ug; n = 4).

136 in vivo stereotaxic hippocampal injection of AAV-encoded expression, we identified specific and delay

137 One limitation of AAV gene delivery is preexisting neutralizing antibodies

138 expression kinetics, and biologic markers of AAV transduction for up to 3 years.

139 h indicates that the genetic modification of AAV vectors may further facilitate the success of AAV ge

140 ht a substantial difference in the nature of AAV-binding antibodies in cats living in geographically

141 This study shows the potential of AAV-CRISPR for permanent genome corrections and highligh

142 The process of AAV capsid assembly remains poorly understood.

143 ll as optimize the large-scale production of AAV.

144 an influence the CNS transduction profile of AAV capsids.IMPORTANCE Understanding how viruses cross t

145 The integration profile of AAV-465lambda in cultured cells display both full-length

146 ee-dimensional (3D) structural properties of AAV capsids.

147 s harnessing the 3D structural properties of AAV capsids.

148 t in-house production of small quantities of AAV vector without the need for specialized equipment.

149 ectors may further facilitate the success of AAV gene therapy.

150 linical trial work focused on these types of AAV treatments for Duchenne MD, various limb girdle MDs,

151 maintaining cellular calcium homeostasis on AAV transduction.

152 estimate the impact of residue mutations on AAV capsid protein-protein interactions and thus predict

153 Nme2Cas9 combines all-in-one AAV compatibility, exceptional editing accuracy within c

154 All-in-one AAV delivery of Nme2Cas9 with a guide RNA targeting Pcsk

155 coevolve multiple, desirable properties onto AAV by harnessing 3D structural information.

156 therapeutic efficacy of the AAV-IFN-alpha or AAV-IFN-alpha fused to apolipoprotein A-1 vectors in exp

157 Vehicle control or AAV viruses (1 x 10(12 )GC/mouse in 200 mul PBS) were in

158 Intravenously injected AAV-Tat or AAV-Exo-Tat mainly infects liver and heart.

159 omise in vivo As recently attained for other AAV biologics, e.g., Luxturna and Zolgensma, based on AA

160 as PR3-positive AAV (PR3-AAV), MPO-positive AAV (MPO-AAV) and, for EGPA, by the presence or absence

161 mes may be better classified as PR3-positive AAV (PR3-AAV), MPO-positive AAV (MPO-AAV) and, for EGPA,

162 e better classified as PR3-positive AAV (PR3-AAV), MPO-positive AAV (MPO-AAV) and, for EGPA, by the p

163 for a more economical and efficient purified AAV preparation.

164 plored here, called BRAVE (barcoded rational AAV vector evolution), enables efficient selection of en

165 TANCE Clinical gene therapy with recombinant AAV vectors has largely relied on natural capsid isolate

166 ty.IMPORTANCE Biologics based on recombinant AAVs (rAAVs) are increasingly becoming attractive human

167 identify cellular host factors that restrict AAV infection in hepatocyte cultures.

168 to the BLA in adult male mice using a retro-AAV construct and assessed their necessity in aversive a

169 icacy of the treatment, an optimized shorter AAV vector was generated, in which four out of six metal

170 Similarly, AAV delivery of the CRISPR antivirals resulted in a redu

171 milar results can be obtained using a single AAV vector incorporating both the `silence' and `replace

172 We further expand our single-AAV platform to pre-implanted zygotes for streamlined ge

173 Recently, tissue-specific AAV capsids (e.g. PHP.eB) have been shown to enhance bra

174 In addition, a recently developed synthetic AAV vector, AAVAnc80, carrying the miniATP7B gene was si

175 mine the infection patterns of two synthetic AAVs (AAV2.7m8 and AAV8BP2) in the mouse inner ear.

176 uch as many mitochondrial diseases, and that AAV-Slc25a46 could be a novel approach for treating SLC2

177 Taken together, our results indicated that AAV-sh-Ac45 simultaneously inhibits osteoclast-mediated

178 Here we show that AAV-delivered meganucleases, but not CRISPR/Cas9, mediat

179 This proof-of-concept study showed that AAV-mediated delivery of a CaMKII peptide inhibitor to t

180 Overall, our study shows that AAV-PHP.B's neurotrophic properties are plausible for tr

181 ator of axonal regeneration and suggest that AAV-mediated delivery of constitutively active Pfn1, tog

182 The AAV-binding antibodies showed broad reactivities with ot

183 pate broad immune cross-reactivity among the AAV serotypes.

184 fulfill its multifunctional role during the AAV life cycle.

185 ls can potentially be applied to improve the AAV gene transfer efficiency.

186 rafficking and conformational changes in the AAV capsid that support efficient genome transcription.

187 stigated the role of cellular calcium in the AAV infectious pathway.

188 , which exceed the packaging capacity of the AAV when combined.

189 prophylactic and therapeutic efficacy of the AAV-IFN-alpha or AAV-IFN-alpha fused to apolipoprotein A

190 + AAV-M3 extend survival beyond that of the AAV-IL-10 alone cohort.

191 ved from a protein, of known function on the AAV capsid surface, and a unique molecular barcode in th

192 We demonstrated that the AAV small hairpin RNA targeting Ac45 (AAV-sh-Ac45) impai

193 Thus, the AAV-hACE2 mouse model enables rapid deployment for in-de

194 Herein, we use the AAV-PHP.eB to deliver an instability-prone Mecp2 (iMecp2

195 Using the AAV-PHP.S capsid to target the lysosomal enzyme glucocer

196 The three AAV subgroups, namely granulomatosis with polyangiitis (

197 both R6/2 and YAC128 HD mouse models through AAV-mediated ectopic expression of NeuroD1 and Dlx2 tran

198 small batches of high quality and high titer AAV vectors for their experimental needs.

199 ution to overcome pre-existing antibodies to AAV-based gene therapy.

200 ity in cats is not necessarily inhibitory to AAV and highlight a substantial difference in the nature

201 discussion of technical concerns specific to AAV-mediated reprogramming experiments that must be addr

202 To provide the sensitivity required to track AAVs injected at picomolar levels, a unique multichelato

203 ed, delivery of meganucleases using a triple AAV serotype combination results in the greatest decreas

204 human primates, a natural host for wild-type AAV.

205 To understand the mechanisms underlying AAV Rep function, we investigated the cryo-EM and X-ray

206 rom six treated dogs identified 1,741 unique AAV integration events in genomic DNA and expanded cell

207 work and many promising ongoing and upcoming AAV clinical trials.

208 liorate the effects of such factors, we used AAV vectors to express isoforms of the antiinflammatory

209 d a hepatocyte specific PRMT1 knockout using AAV mediated Cre delivery in mice fed either alcohol or

210 ing PKR in C9orf72 BAC transgenic mice using AAV-PKR-K296R or the Food and Drug Administration (FDA)-

211 ablation of KORs from dopamine neurons using AAV-TH-cre in KOR(loxP) mice prevented pain-induced aver

212 more examples of virologic suppression using AAV delivery of a cocktail of four mAbs in a 12-monkey s

213 a resource, we have generated and validated AAV-PA-Cre 3.0 as well as two mouse lines that can condi

214 All pipelines detected amino acid variants (AAVs) at full range of frequencies (1~100%) and demonstr

215 re-existing humoral immunity against various AAV serotypes in cats is still limited.

216 mic antibody (ANCA)-associated vasculitides (AAVs) are a group of disorders involving severe, systemi

217 of patients with ANCA-associated vasculitis (AAV) and severe renal involvement is not established.

218 cytoplasmic antibody-associated vasculitis (AAV).

219 Adeno-associated viral vectors (AAVs) achieve stable therapeutic expression without long

220 r delivery of green fluorescence protein via AAV vector serotype PHP.B in adult wild-type male mice t

221 AAV9 vectors expressing canine factor VIII (AAV-cFVIII) corrected the FVIII deficiency to 1.9-11.3%

222 Adeno-associated viral (AAV) delivery of constitutively active Pfn1 to rodents p

223 A were treated with adeno-associated viral (AAV) gene therapy and followed for up to 10 years.

224 3 weeks after adeno-associated viral (AAV) infusion, mice were exposed to 330 mug of Mn (MnCl(

225 n A-1 by means of an adeno-associated viral (AAV) system in the mouse model of myelin oligodendrocyte

226 tivation, we used an adeno-associated viral (AAV) vector carrying the channelrhodopsin-2 gene under t

227 ned into recombinant adeno-associated viral (AAV) vector genome and high-titre viral vectors were pro

228 nock-in mouse and by adeno-associated viral (AAV) vector-mediated gene augmentation of ERdj5 in P23H-

229 complementation with adeno-associated viral (AAV) vectors is one strategy to treat RP.

230 lentiviral (LV) and adeno-associated viral (AAV) vectors that preferentially express anti-sense miR

231 se findings, we used adeno-associated viral (AAV) vectors to overexpress MIF in the spinal cord of mu

232 sed in cerebellum via adenoassociated virus (AAV) viral transfection in mice, the mutant BK(G354S) ch

233 rationally designed adeno-associated virus (AAV) 6 capsid that demonstrates efficiency in lung epith

234 Using an adeno-associated virus (AAV) approach, coupled with genome editing, we validated

235 als was tested using adeno-associated virus (AAV) as a packaging vector for both SaCas9 and sgRNA.

236 ive, we developed an adeno-associated virus (AAV) based "Provector" whose cellular transduction can b

237 Adeno-associated virus (AAV) capsid modification enables the generation of recom

238 Adeno-associated virus (AAV) exhibits anterograde transneuronal transport, howev

239 pha-syn) fibrils and adeno-associated virus (AAV) expressing human wild-type alpha-syn unilaterally i

240 lia injections of an adeno-associated virus (AAV) expressing short hairpin RNAs targeting GHSR (or a

241 lict, we injected an adeno-associated virus (AAV) expressing the genetically encoded Ca(2+) indicator

242 be packaged into an adeno-associated virus (AAV) hold great promise for gene therapy.

243 Adeno-associated virus (AAV) is a leading vector for virus-based gene therapy.

244 Adeno-associated virus (AAV) is a promising gene therapy vector because of its e

245 Adeno-associated virus (AAV) is a promising vector for gene therapy, but its bro

246 The adeno-associated virus (AAV) non-structural Rep proteins catalyze all the DNA tr

247 mically delivered an adeno-associated virus (AAV) serotype 9 carrying the human GBA gene under contro

248 icacy of intrathymic adeno-associated virus (AAV) serotypes to transduce thymocyte subsets and correc

249 uccessfully delivers adeno-associated virus (AAV) throughout the cervical, thoracic and lumbar spinal

250 The adeno-associated virus (AAV) vector effectively transduced in the cerebrum, cere

251 l methods to discern adeno-associated virus (AAV) vector transduction patterns are based on high, sta

252 ular injection of an adeno-associated virus (AAV) vector-based system encoding an artificial microRNA

253 Adeno-associated virus (AAV) vector-mediated gene delivery was recently approved

254 l model for studying adeno-associated virus (AAV) vector-mediated gene therapies.

255 lizing antibodies to adeno-associated virus (AAV) vectors are highly prevalent in humans(1,2), and bl

256 Adeno-associated virus (AAV) vectors are preeminent in emerging clinical gene th

257 Adeno-associated virus (AAV) vectors are the leading platform for gene delivery

258 stemic injections of adeno-associated virus (AAV) vectors encoding nuclease-dead Cas9 and a single-gu

259 Gene therapy with adeno-associated virus (AAV) vectors has demonstrated appropriate tropism for ta

260 such approaches with adeno-associated virus (AAV) vectors have been shown to be safe and efficacious

261 Adeno-associated virus (AAV) vectors have shown promising results in preclinical

262 HiUGE employs adeno-associated virus (AAV) vectors of autonomous insertional sequences (payloa

263 the human body using adeno-associated virus (AAV) vectors.

264 ene augmentation via adeno-associated virus (AAV), providing a proof-of-concept for curing the vast m

265 Adeno-associated virus (AAV)-based gene therapies can restore endogenous factor

266 d mouse model, using adeno-associated virus (AAV)-delivered meganucleases, as a potentially curative

267 n vivo regulation of adeno-associated virus (AAV)-delivered transgenes, allowing dose-dependent and u

268 und that delivery of Adeno-associated virus (AAV)-expressing miR760 in the cerebellum reduces ATXN1 l

269 -transgenic and adenovirus-associated virus (AAV)-HBV mouse models.

270 sease model and used adeno-associated virus (AAV)-mediated Ac45 RNA interference knockdown to study t

271 SARS-CoV-2 based on adeno-associated virus (AAV)-mediated expression of hACE2.

272 und that recombinant adeno-associated virus (AAV)-mediated expression of IL-10, alone, or in combinat

273 entricular myocytes, adeno-associated virus (AAV)-mediated gene delivery in mice, and human tissue sa

274 Adeno Associated Virus (AAV)-mediated gene expression in the brain is widely app

275 Adeno-associated virus (AAV)-mediated gene therapy is under investigation as a t

276 lenge, we first used adeno-associated virus (AAV)-mediated gene transfer to perform a low-throughput

277 Adeno-associated virus (AAV)-mediated SaCas9-KKH delivery prevented deafness in

278 tte are nested in an adeno-associated virus (AAV).

279 ther parvovirus, the adeno-associated virus (AAV).

280 ystemic injection of adeno-associated virus (AAV)2/1-carrying truncated gRNAs targeting Myd88 and the

281 , we have generated adeno-associated viruses AAV-Tat and AAV-Exo-Tat viruses.

282 Adeno-associated viruses (AAV) are composed of nonenveloped, icosahedral protein s

283 Adeno-associated viruses (AAV) are helper-dependent parvoviruses that have been de

284 were treated with adeno-associated viruses (AAVs) 2 or 8 directed against short hairpin ChREBP to no

285 Adeno-associated viruses (AAVs) are dependoparvoviruses that have proven useful fo

286 delivery systems, adeno-associated viruses (AAVs) are relatively safe and demonstrate high gene tran

287 Adeno-associated viruses (AAVs) are typically single-stranded deoxyribonucleic aci

288 otein 9 (Cas9) and adeno-associated viruses (AAVs) evades adaptive immune responses and enables effec

289 Adeno-associated viruses (AAVs) from clade E are often used as vectors in gene del

290 ription.IMPORTANCE Adeno-associated viruses (AAVs) have proven to be effective gene transfer vectors.

291 tegrity.IMPORTANCE Adeno-associated viruses (AAVs) have recently emerged at the forefront as gene the

292 Delivery via adeno-associated viruses (AAVs) is limited by AAV packaging capacity, which preclu

293 tured neurons with adeno-associated viruses (AAVs), is completed in a few minutes and allows stable t

294 wever, major obstacles remain for widespread AAV utilization, such as impractical purification strate

295 r, the ability to transduce lymphocytes with AAV in vivo after systemic delivery has not been careful

296 y of MPO- or PR3-ANCA-positive patients with AAV (MPA and GPA) and severe kidney disease (eGFR <30 ml

297 YC or RTX for the treatment of patients with AAV and severe kidney disease are balanced.

298 nt pneumococcal vaccination in patients with AAV, thus offering potential clinical benefit.

299 he genomic consequences of transduction with AAV vectors encoding CRISPR-Cas nucleases is still being

300 In vivo co-treatment with AAV-miRzip-21 and AAV-miR-7 in mice bearing malignant br