ライフサイエンスコーパス: AAV vector

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

2 CI-MPR in muscle, was administered with the AAV vector.

3 ansgenes separately, or a single bicistronic AAV vector.

4 relative inaccessibility of BCs to standard AAV vectors.

5 and multiple gRNA expression cassettes with AAV vectors.

6 t for single-stranded and self-complementary AAV vectors.

7 decreased avidity favors systemic spread of AAV vectors.

8 aB in HeLa cells transduced with recombinant AAV vectors.

9 tocol for gene targeting in human cells with AAV vectors.

10 argeted integration of these double-stranded AAV vectors.

11 ignificant hurdle in clinical application of AAV vectors.

12 ization of cell clones containing integrated AAV vectors.

13 s have been developed to better characterize AAV vectors.

14 pecific memory CD8(+) T cells reactivated by AAV vectors.

15 and raise concerns over the clinical use of AAV vectors.

16 both high and low transgene expression from AAV vectors.

17 ne therapy clinical trials using recombinant AAV vectors.

18 ld be co-delivered to TG neurons by separate AAV vectors.

19 a substantial improvement over conventional AAV vectors.

20 a single functional adeno-associated virus (AAV) vector.

21 were delivered by an adeno-associated virus (AAV) vector.

22 hrough the use of an adeno-associated viral (AAV) vector.

23 igen delivered by an adeno-associated virus (AAV) vector.

24 livery, such as with adeno-associated viral (AAV) vectors.

25 r vectorization into adeno-associated virus (AAV) vectors.

26 tro), deliverable by adeno-associated virus (AAV) vectors.

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

28 uced higher transduction than their parental AAV vectors (2- to 9-fold over AAV2), with the highest o

29 th a human SMN-expressing self-complementary AAV vector - a vector that leads to earlier onset of gen

30 ravenous dose of the adeno-associated virus (AAV) vector, AAV-BR1-CAG-NEMO, delivering the Nemo gene

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

32 Although subretinal (SR) AAV vector administration can transfect retinal cells ef

33 ngly, the antibody response was prevented by AAV vector administration during the 12 wk of ERT, and t

34 in the liver was found after single-stranded AAV vector administration, regardless of the capsid sequ

35 overexpressed via an adeno-associated virus (AAV) vector after the onset of spontaneous recurrent sei

36 guide RNA expression cassette into a single AAV vector and targeted the cholesterol regulatory gene

37 e that were previously immunized against the AAV vector and the Cas9 cargo.

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

39 lt mouse brains via stereotaxic injection of AAV vectors and found that it also preferentially accumu

40 hould be considered for design of both safer AAV vectors and gene therapy studies.

41 outcome of liver-directed gene therapy using AAV vectors and showed in a proof-of-principle study how

42 ging, new approaches to engineer and improve AAV vectors and their genetic cargo are increasingly hel

43 ing of the determinants of immunogenicity of AAV vectors, and of potential associated toxicities, is

44 dministration of the adeno-associated virus (AAV)-vectored anti-phospho-tau antibody PHF1 to P301S ta

45 LG gene delivery by AAV vectors appears to be both safe and well tolerated.

46 The present work revealed that AAV vectors are able to delivery DNA to the LGs of mice.

47 Because AAV vectors are administered directly to the patient, th

48 Because AAV vectors are already used for liver-directed human ge

49 Furthermore, we show that single-stranded AAV vectors are better substrates for site-specific inte

50 AAV vectors are nonpathogenic and elicit minimal inflamm

51 Adeno-associated virus (AAV) vectors are attractive for gene delivery-based ther

52 Adeno-associated virus (AAV) vectors are being considered for in vivo applicatio

53 Adeno-associated virus (AAV) vectors are currently being evaluated for the treat

54 Adeno-associated virus (AAV) vectors are currently the leading candidates for vi

55 Adeno-associated virus (AAV) vectors are effective gene delivery vehicles mediat

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

57 Adeno-associated virus (AAV) vectors are ideal for performing gene repair due to

58 nsduction potential, adeno-associated virus (AAV) vectors are leading candidates for gene therapy in

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

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

61 s and opens novel perspectives on the use of AAV vectors as vaccines against emergent diseases.

62 from a set of other adeno-associated virus (AAV) vectors as a potent vector for the cochlear cell ta

63 imitations for using adeno-associated virus (AAV) vectors as gene transfer agents to LG.

64 ce by intraperitoneal injection of the PQBP1-AAV vector at E10 successfully rescued microcephaly with

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

66 adjuvant; in contrast, administration of the AAV vector before the GAA challenge prevented the antibo

67 Polyploid AAV vectors can be generated from any AAV serotype, whet

68 However, some AAV vectors can be transported along neuronal pathways a

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

70 Adeno-associated virus (AAV) vectors can stably express HIV-1 broadly neutralizi

71 ators to dissect the immune responses to the AAV vector capsid and to the transgene product, and to d

72 orchid donor testes were exposed in vitro to AAV vectors carrying a GFP transgene and transplanted to

73 To overcome this obstacle, we constructed AAV vectors carrying the channelrhodopsin-2 (ChR2) gene

74 a self-complementary adeno-associated viral (AAV) vector carrying human RP2-coding sequence and demon

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

76 eletion of mTOR with adeno-associated viral (AAV) vector carrying the Cre recombinase in the hippocam

77 ere amplified from human DNA, cloned into an AAV vector cassette upstream of the green fluorescent pr

78 Thus, we did not find evidence that AAV vectors cause insertional activation of oncogenes an

79 ed a single-stranded adeno-associated viral (AAV) vector consisting of a bioengineered capsid, liver-

80 Since most commonly used recombinant AAV vectors contain a single-stranded DNA (ssDNA), which

81 served in these mice after injection with an AAV vector containing a lacZ gene fragment, and precise

82 AAV-DJ is an artificial chimeric AAV vector containing hybrid capsid sequences from three

83 inner retina after intravitreal injection of AAV vectors containing five distinct promoters.

84 gene therapy with an adeno-associated virus (AAV) vector containing a liver-specific promoter elevate

85 passionate use of an adeno-associated virus (AAV) vector containing the human AADC gene (AAV2-hAADC)

86 ion of a recombinant adeno-associated virus (AAV) vector containing the RPE65 gene (AAV2-hRPE65v2) in

87 Adeno-associated virus (AAV) vectors containing single-stranded DNA efficiently

88 Here, we used adeno-associated virus (AAV) vectors containing the astrocyte-specific Gfa2 prom

89 s study, we injected adeno-associated virus (AAV) vectors containing the human CLN2 cDNA into the bra

90 IIa (mFVIIa) from an adeno-associated viral (AAV) vector corrected abnormal hemostatic parameters in

91 Thus, CpG-depleted AAV vectors could improve outcome of clinical trials of

92 with stereotaxic injection of 5-HT2CR shRNA AAV vector decreased vocalizations and anxiety- and depr

93 sgenic mice using an adeno-associated virus (AAV) vector, decreased parenchymal Abeta amyloid deposit

94 strophic mdx(4cv) mice using single and dual AAV vector delivery of a muscle-specific Cas9 cassette t

95 The combination of an AAV vector delivery system and exploitation of the endog

96 By using adeno-associated viral (AAV) vector delivery in vivo, we achieved long-term expr

97 report that systemic adeno-associated virus (AAV) vector delivery of zinc finger nucleases (ZFNs) and

98 sgene cassette using adeno-associated virus (AAV) vector delivery.

99 Thus, the new HSV/AAV vector demonstrated unique advantages in safe and ef

100 Tumors in mice treated with AAV vectors did not have significantly different amounts

101 In addition, we demonstrated that the AAV vector dose, enhancer/promoter selection, and the ti

102 ets currently is the adeno-associated viral (AAV) vector due to its desirable safety profile and stro

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

104 Injection of a double-stranded AAV vector encoding IL-2 driven by a mouse insulin promo

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

106 AAV vectors encoding firefly luciferase were administere

107 As proof of concept, delivery of AAV vectors encoding for hexokinase or vascular endothel

108 adapted an intravascular delivery system of AAV vectors encoding the FIX transgene to skeletal muscl

109 inal injection of an adeno-associated virus (AAV) vector encoding ARES, luciferase expression can be

110 ly manipulated by an adeno-associated virus (AAV) vector encoding BMP4 delivered by a clinically appl

111 We used an adeno-associated viral (AAV) vector encoding green fluorescent protein under an

112 Introduction of an adeno-associated viral (AAV) vector encoding IL-4 into the hippocampus resulted

113 gene therapy with an adeno-associated viral (AAV) vector encoding REP1 (AAV.REP1) in patients with th

114 ) with CRE-dependent adeno-associated viral (AAV) vector encoding the engineered Gi/o-coupled human m

115 administration of an adeno-associated virus (AAV) vector encoding the human DOK7 gene resulted in an

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

117 Recombinant adeno-associated virus (AAV) vectors encoding the growth factors were injected t

118 long-term effect of adeno-associated viral (AAV) vector-encoding vascular endothelial growth factor

119 In six pigs, adeno-associated viral (AAV) vector-encoding VEGF (AAV-VEGF) gene was injected a

120 we demonstrate that adeno-associated viral (AAV) vectors, especially serotypes 8 and 9, mediated eff

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

122 rcinoma (HCC) after neonatal injection of an AAV vector expressing b-glucuronidase.

123 eral infusions of a retrogradely transported AAV vector expressing Cre recombinase (Retro-Cre-GFP) in

124 Four weeks after disease induction, AAV vector expressing the vIL-10 gene under control of a

125 arate cohort was injected with CRE-dependent AAV vectors expressing diphtheria toxin (DTA) to selecti

126 f-complementary adenovirus-associated virus (AAV) vector expressing a codon-optimized human factor IX

127 y to the VMH with an adeno-associated viral (AAV) vector expressing a short hairpin RNA for AMPKalpha

128 6 was depleted using adeno-associated viral (AAV) vector expressing CD36 short hairpin RNA (shRNA) in

129 s of a Cre-dependent adeno-associated viral (AAV) vector expressing enhanced halorhodopsin 3.0 fused

130 enous infusion of an adeno-associated viral (AAV) vector expressing factor IX.

131 iated by AAV-GPE, an adeno-associated virus (AAV) vector expressing G6Pase-alpha directed by the huma

132 In this study, an adeno-associated virus (AAV) vector expressing human SMN (AAV8-hSMN) was injecte

133 ted injections of an adeno-associated virus (AAV) vector expressing short hairpin RNA (shRNA) to knoc

134 se was achieved with adeno-associated viral (AAV) vectors expressing Cas9 or guide RNAs (gRNAs).

135 to hepatocytes using adeno-associated virus (AAV) vectors expressing hepatic transcription factors.

136 athway in rats using adeno-associated virus (AAV) vectors expressing the astrocyte-specific promoter

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

138 he potential of CREATE to produce customized AAV vectors for biomedical applications.

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

140 proaches to the molecular evolution of novel AAV vectors for human gene therapy applications.

141 nd their application to characterize current AAV vectors for preclinical and clinical use.

142 ut also provide a general approach to tailor AAV vectors for systemic or hepatic gene transfer by ree

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

144 eering and payload design aimed at tailoring AAV vectors for transduction and treatment of cancer cel

145 n gene therapy using adeno-associated viral (AAV) vector for hemophilia B (HB) showed that the risk o

146 we review the use of adeno-associated virus (AAV) vectors for delivery of HIV bNAbs and antibody-like

147 We investigated adeno-associated virus (AAV) vectors for gene delivery to the TG after intraderm

148 and effective use of adeno-associated viral (AAV) vectors for gene therapy.

149 f seven serotypes of adeno-associated virus (AAV) vectors for genetic manipulation of primary culture

150 ed gene therapy with adeno-associated viral (AAV) vectors for hemophilia.

151 Use of adeno-associated viral (AAV) vectors for liver-directed gene therapy has shown c

152 The adeno-associated virus (AAV) vector gene delivery system has shown promise in se

153 With this in mind, adeno-associated virus (AAV) vector gene delivery was used to localize IL-2 expr

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

155 g second-strand synthesis in single-stranded AAV vector genomes and to facilitate robust transgene ex

156 efficiency, significantly higher numbers of AAV vector genomes were successfully delivered to the nu

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

158 Gene targeting with adeno-associated virus (AAV) vectors has been demonstrated in multiple human cel

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

160 Gene transfer using adeno-associated virus (AAV) vectors has great potential for treating human dise

161 AAV vectors have achieved positive results in a number o

162 For cancer applications, AAV vectors have been harnessed for delivery of an exten

163 me receptor.IMPORTANCE Over the past decade, AAV vectors have emerged as leading gene delivery tools

164 Adeno-associated virus (AAV) vectors have been most successful; to target the ou

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

166 Although adeno-associated viral (AAV) vectors have been successfully used in hepatic gene

167 in gene therapy with adeno-associated virus (AAV) vectors have been the object of almost two decades

168 Adeno-associated virus (AAV) vectors have been used successfully in clinical tri

169 Adeno-associated virus (AAV) vectors have been widely adopted for use in gene th

170 Adeno-associated virus (AAV) vectors have made great progress in their use for g

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

172 Adeno-associated virus (AAV) vectors have the potential to promote long-term gen

173 therapies utilizing adeno-associated viral (AAV) vectors hold great promise for treating Duchenne mu

174 ibodies (mAbs) using adeno-associated virus (AAV) vectors holds promise for the prevention and treatm

175 However, as with other AAV vectors, host anti-capsid immune responses are a det

176 Delivery of a single AAV vector in the lung generated loss-of-function mutati

177 can be avoided by enhancing the efficacy of AAV vectors in hepatocytes.

178 greatly improved transduction efficiency of AAV vectors in human and mouse hepatocytes independent o

179 cations for the potential use of these novel AAV vectors in human gene therapy.

180 gnificant implications in the optimal use of AAV vectors in human gene therapy.

181 ection site following subretinally delivered AAV vectors in normal dogs.

182 full spectrum of transduction patterns from AAV vectors in vivo, will be foundational to current and

183 A expression with an adeno-associated virus (AAV) vector in GAA-knockout (KO) mice.

184 n be transduced with adeno-associated virus (AAV) vectors in vivo, in a manner that would be useful f

185 nger genetic elements to be packaged into an AAV vector including tissue-specific promoters, multiple

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

187 both single-stranded and self-complementary AAV vectors indicate that the genomes are largely packag

188 ssion of HMGB1 using adeno-associated virus (AAV) vectors induced inflammation in the hearts of both

189 smid transfection and 4- to 6-fold lower for AAV vector infection), but they still represented a sign

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

191 novel high-throughput method for identifying AAV vector integration sites was developed and used to c

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

193 Remarkably, loading AAV vectors into pSi microparticles increases gene deliv

194 We found that a direct microinjection of AAV vectors into the vagal nodose ganglia in vivo leads

195 This new HSV/AAV vector is designed in a way that little or no Rep wo

196 nactive, efficient transgene expression from AAV vectors is dependent upon viral second-strand DNA sy

197 The eGFP encoded by AAV vectors is robustly transported to both the central

198 luation of nNOS binding mini-dystrophin dual AAV vectors is warranted in dystrophic dogs and eventual

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

200 et of gene expression compared with standard AAV vectors - led to improved efficacy of gene therapy,

201 ted in humans and additional modification to AAV vectors may be required for further study in order t

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

203 These data support AAV vector-mediated expression of a comparable truncated

204 Thus, AAV vector-mediated gene therapy induced a tolerance to

205 ance between tolerance and immunogenicity in AAV vector-mediated gene transfer are not fully understo

206 VP16, an NF-kappaB activator, augmented AAV vector-mediated transgene expression up to 25-fold.

207 ional silencing upon adeno-associated viral (AAV) vector-mediated expression in photoreceptors.

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

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

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

211 ibe a novel study of adeno-associated virus (AAV) vector-mediated gene therapy that induced immune to

212 nt tissue target for adeno-associated viral (AAV) vector-mediated gene transfer of the factor IX (FIX

213 f IFN-beta following adeno-associated virus (AAV) vector-mediated gene transfer resulted in significa

214 Adeno-associated virus (AAV) vector-mediated transfer of a normal cDNA can corre

215 AAV vectors must reach the nucleus in order to deliver t

216 We demonstrated that AAV vectors of serotypes 1, 7, 8, and 9 trafficked from

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

218 limiting delivery by adeno-associated virus [AAV] vectors), off-target editing, or complex protospace

219 No significant apoptosis related to AAV vectors or IDUA was observed under any conditions in

220 In this study, a very low number of AAV vector particles was administered before initiation

221 Biodistribution analyses suggested AAV vectors persisted only in the trauma-induced corneas

222 mice was fully rescued by treatment with the AAV-vectored PHF1 antibody.

223 ibrillary tangles following a single dose of AAV-vectored PHF1 compared with mice treated with an AAV

224 The self-complementary AAV vector preparation appears to contain particles with

225 AAV vectors produce stable transgene expression and elic

226 atum, we injected an adeno-associated virus (AAV) vector producing a short hairpin RNA (AAV.sh.p11).

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

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

229 poxia-regulated, retinal glial cell-specific AAV vector provides a platform for gene therapy within r

230 nistered recombinant adeno-associated viral (AAV) vectors requires crossing the blood-brain barrier (

231 jection of 2 x 10(11) vg/kg of the hFVIIcoop AAV vector resulted in therapeutic levels of hFVII expre

232 imilarly, coadministration of rapamycin with AAV vectors resulted in markedly enhanced expression of

233 mic and intravitreal injection of engineered AAV vectors resulted in RdCVF and RdCVFL expression in t

234 iated by recombinant adeno-associated virus (AAV) vectors resulted in significant antiapoptotic activ

235 as well as to a novel, structurally distinct AAV vector, rh32.33, in an in vitro transduction inhibit

236 of this association and the implications for AAV vector safety.

237 positive for the presence of the transferred AAV vector sequence.

238 To evaluate systemic toxicity, we measured AAV vector sequestration in the liver using qPCR, and fo

239 AAV vector serotype DJ efficiently transduced CDKL5-muta

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

241 arrow reconstitution of mice treated with an AAV vector several weeks earlier.

242 F-kappaB inhibitors before transduction with AAV vectors should lead to a dampened immune response, w

243 ons of HBV delivered by a self-complementary AAV vector showed better antiviral effects than single s

244 ophilia B (HB) using adeno-associated viral (AAV) vectors showed that the safety of a given strategy

245 hic factor, using an adeno-associated virus (AAV) vector significantly increased the neuritin level a

246 y, questions have arisen about the safety of AAV vectors, specifically, whether integration of vector

247 Rhesus macaques inoculated with an AAV vector stably expressed 17-77 mug ml(-1) of fully fu

248 c biology of AAV, the history of progress in AAV vector technology, and some of the clinical and rese

249 ith both plasmid and adeno-associated virus (AAV) vector templates.

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

251 These studies have led to the development of AAV vectors that are capable of high-efficiency transduc

252 in mdx and mdx4cv mice using a pair of dual AAV vectors that expressed a 6 kb nNOS-binding mini-dyst

253 ts may lend insight into the design of novel AAV vectors that have an enhanced nuclear entry capabili

254 he development of an adeno-associated viral (AAV) vector that rescues rhodopsin mislocalization, main

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

256 the effect of HBV on adeno-associated viral (AAV) vectors, the most frequently applied gene transfer

257 ific integration than are self-complementary AAV vectors; the absence of DNAPKcs did not affect the t

258 ly cross the blood-brain barrier, we used an AAV vector to deliver antibody directly to the hippocamp

259 ese studies show that the use of recombinant AAV vector to deliver genes is a promising approach for

260 y is the first to use a recombinant chimeric AAV vector to knockout a gene in porcine fibroblasts for

261 This review describes the applications of AAV vectors to cancer models and presents developments i

262 Using AAV vectors to deliver antibodies like PHF1 directly to

263 A related approach uses AAV vectors to edit specific regions of the DMD gene usi

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

265 Additionally, we developed novel systemic AAV vectors to facilitate morphological reconstruction o

266 The goal was to investigate the ability of AAV vectors to induce long-term, safe delivery of transg

267 velopment can be exploited to redirect novel AAV vectors to specific cell types in the brain.

268 Airway-based delivery of AAV vectors to the pulmonary arteries was feasible, effi

269 ving vector efficacy may improve delivery of AAV vectors to their therapeutic targets.

270 We have used an adeno-associated viral (AAV) vector to deliver the genes encoding an anti-phosph

271 further injected an adeno-associated viral (AAV) vector to express IVS-AAA in the brain of a knock-i

272 , we created several adeno-associated virus (AAV) vectors to deliver genes that combat oxidation.

273 When using adeno-associated viral (AAV) vectors to express luciferases in the brain, we fou

274 n this study, we use adeno-associated viral (AAV) vectors to increase Perm1 expression in skeletal mu

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

276 and guide RNAs using adeno-associated viral (AAV) vectors to target single (Mecp2) as well as multipl

277 ach provides design strategies to expand the AAV vector toolkit.

278 potential strategies to redirect recombinant AAV vectors toward more productive trafficking pathways

279 AAV vectors transduced liver and kidney in GSD Ia and st

280 Our data strongly suggest that AAV vector-transduced cells are rarely eliminated by AAV

281 sented here provide the first description of AAV vectors transducing neurons following delivery at th

282 r, AAV receptors contribute significantly to AAV vector transduction efficiency and tropism.

283 following a corneal intrastromal injection, AAV vector transduction kinetics, using a chimeric AAV c

284 d function following adeno-associated virus (AAV) vector transduction of MPS1 patient fibroblasts.

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

286 fficiently regulated adeno-associated virus (AAV)-vectored transgene expression in cultured mammalian

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

288 None of the dogs (n = 14) receiving the AAV vector under transient IS developed inhibitory antib

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

290 The AAV vector was tested in vivo by subretinal injections i

291 e-directed expression of canine FIX-R338L by AAV vectors was carried out in HB dogs.

292 MRI-guided technology for administration of AAV vectors we have developed and now employ in current

293 Using an adeno-associated viral (AAV) vector, we expressed the regeneration-promoting cel

294 anial and intrathecal injections of the same AAV vector were combined.

295 led to the development of novel recombinant AAV vectors which are more efficient in allowing increas

296 WT and Tlr9-deficient mice that received an AAV vector with an immunogenic capsid, AAVrh32.33.

297 ation for rational structural engineering of AAV vectors with improved therapeutic efficacy.

298 ion to date from a parenterally administered AAV vector, with broad implications for the future of mu

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

300 er-specific expression of human GAA with the AAV vector would induce immune tolerance and enhance the