ライフサイエンスコーパス: ASO

1 ASO facilitated cell proliferation, metabolism, and angi

2 ASO reduction of ApoC-III had no effect on VLDL secretio

3 ASO treatment achieved specific reduction of Ube3a-ATS a

4 ASO treatment starting after birth led to a significant

5 ASO when applied in normothermic kidney machine perfusio

6 ASO-T3 enhances white fat browning, decreases genes for

7 ASOs are a useful class of therapeutic agents with broad

8 ASOs are ideal candidates for the treatment of neurodege

9 ASOs bind the mid-domain of Hsp90 protein.

10 ASOs modified with phosphorothioate (PS) linkages enter

11 ASOs primarily target fat and liver with poor penetrance

12 The Mcl-1 ASOs also induced mitochondrial hyperpolarization and a

13 Moreover, administration of the exon 11 ASO reduced lamin A expression in wild-type mice and pro

14 artile range): ASO 2.5mg/kg: 1.9 (1.3; 3.2), ASO 25mg/kg: 2.8 (0.7; 5.0), mismatch oligonucleotide (M

15 Additionally, ASO-based MDM4 targeting reduced diffuse large B cell ly

16 tically applicable approach, we administered ASOs targeting ataxin-2 to the central nervous system of

17 and found that protein binding could affect ASO potency.

18 evels in patient myotubular cell lines after ASO treatment.

19 Agt ASO resulted in significant reduction in plasma, liver,

20 Agt-ASO suppressed cell proliferation in both cystic and non

21 Kidneys from Agt-ASO-treated mice were not as enlarged and showed reduced

22 However, Agt-ASO did not reduce cell proliferation in liver, which in

23 These data suggest that Agt-ASO effectively attenuates intrarenal RAS and therefore

24 iferation in liver, which indicates that Agt-ASO targets cell signaling pathways that specifically su

25 ene knockout, the mice were treated with Agt-ASO (66 mg/kg/wk), lisinopril (100 mg/kg/d), PBS (contro

26 ter controlled with lisinopril than with Agt-ASO.

27 ee and colleagues developed and evaluated an ASO-dependent method for treating certain molecularly de

28 in 2-5 nucleotides at each end (wing) of an ASO.

29 Here, we show that an ASO targeting MALAT1 RNA, delivered by transuterine micr

30 These studies report for the first time an ASO and RNAi delivery system based upon protein toxin ar

31 Apolipoprotein B (ApoB)-ASO is an FDA approved drug for treating familial hyperc

32 NNMT-ASO and ApoB-ASO are chemically conjugated with T3 using a non-cleava

33 Both NNMT-ASO-T3 (NAT3) and ApoB-ASO-T3 (AAT3) enhance thyroid hormone receptor activity.

34 nts LDL cholesterol-lowering effects of ApoB-ASO.

35 SMN-AS1 ASOs delivered together with SMN2 splice-switching oligo

36 s can profoundly affect interactions between ASOs and intracellular proteins in ways that are only be

37 Nusinersen is a steric block ASO that binds the SMN2 messenger RNA and promotes exon

38 s showed better preserved morphology in both ASO groups than saline- and MM-treated kidneys (median a

39 Both ASOs and siRNAs are being explored to knock-down the tra

40 showed that in vivo inhibition of miR-182 by ASO improved kidney function and morphology after AKI.

41 ion of U12-dependent introns is mitigated by ASO treatment of SMA mice and that many transcriptional

42 he downstream cleavage products generated by ASOs or siRNA targeting mRNAs.

43 t, downstream cleavage products generated by ASOs targeting nuclear long non-coding RNA Malat 1 and p

44 Exon skipping by ASOs is gaining traction as a therapeutic strategy, and

45 beta-Catenin ASO altered hepatic lipid composition in high-fat-fed mi

46 beta-Catenin ASO improved hepatic insulin sensitivity and increased i

47 nhances the activity of PS/LNA or PS/(S)-cEt ASOs, and imply that altering protein binding of ASOs us

48 not reach a solid 10(-4), whereas classical ASO-PCR is time-consuming and labor intensive.

49 nd demonstrates the promise of combinatorial ASOs for the treatment of neurogenetic disorders.

50 A mixed-modality approach combining ASOs and RNAi reagents improved knockdown efficacy, part

51 vitro or in vivo uptake of GalNAc-conjugated ASO and that the major subunit, ASGR1, plays a small but

52 ationships of triantennary GalNAc conjugated ASOs for enhancing potency via ASGR mediated delivery to

53 1 GalNAc conjugated ASOs showed 10-fold reduced ASGR binding affinity relati

54 of N-acetylgalactosamine (GalNAc)-conjugated ASOs for Asialoglycoprotein Receptor (ASGR)-mediated upt

55 nalization and activity of GalNAc-conjugated ASOs and their parents in endogenous ASGR-expressing cel

56 in white adipose tissue relative to control ASO-treated mice.

57 Compared to current ASOs, these multimers and multi-targeting oligonucleotid

58 A similarly delivered ASO targeting a causal splice site mutation for Usher sy

59 need to address the challenges of delivering ASO therapies to the CNS, with appropriate efficiency an

60 h indicate that rates for RNase H1-dependent ASO-mediated degradation of the targeted RNAs are differ

61 lNAc conjugated single stranded and duplexed ASOs were studied.

62 nces susceptibility to degradation by either ASOs or RNAi, nuclear lncRNAs (MALAT1 and NEAT1), cytopl

63 potential of disarmed Atx to deliver either ASOs or siRNA.

64 M1, ANXA2, VARS and PC4, appeared to enhance ASO activities, likely through mechanisms related to sub

65 ncreased internalization rates also enhanced ASO potency for reducing expression of the non-coding RN

66 PS-ASOs in late endosomes (LEs) and enhances ASO activity.

67 Two established ASOs are tested.

68 implying that these proteins may facilitate ASO release from the endocytic pathway.

69 activities increased significantly following ASO treatment in patient myotubes.

70 Further investigation into the basis for ASO-FUS binding illustrated the importance of ASO backbo

71 of various nucleic acid binding domains for ASO depend on chemical modifications and further demonst

72 most likely by competition with RNase H1 for ASO/RNA duplex binding.

73 degradation, however kinetic parameters for ASO-mediated targeting and subsequent cleavage and degra

74 eduction of PMP22 mRNA in skin biopsies from ASO-treated rats is a suitable biomarker for evaluating

75 FXI-ASO (ISIS 416858) is a second-generation antisense oligo

76 hroplasty to receive one of two doses of FXI-ASO (200 mg or 300 mg) or 40 mg of enoxaparin once daily

77 We compared the efficacy and safety of FXI-ASO with those of enoxaparin in patients undergoing tota

78 In order to form a complex with LFn-GAL4, ASOs were engineered to contain a double-stranded region

79 The in vitro toxicity of both PA:LFn-GAL4:ASO and PA:LFn-PKR:siRNA complexes was low (IC50>100 mug

80 The PA:LFn-GAL4:ASO complexes had transfection efficiency approximately

81 PA:LFn-GAL4:ASO transfection of non- or terminally-differentiated TH

82 GR binding affinity relative to three GalNAc ASOs but only 2-fold reduced activity in mice.

83 GalNAc-ASO conjugates exhibited excellent potencies (ED50 0.5-2

84 GalNAc-ASO conjugates thus represent a viable approach for enha

85 iver ASGR for the effective uptake of GalNAc-ASO conjugates, suggesting broad opportunities to exploi

86 pared with the parent single-stranded gapmer ASO.

87 g of fully phosphorothioated (PS)-LNA gapmer ASOs designed against the BACH1 transcript.

88 One second generation ASO, Kynamro, was recently approved by the FDA for the t

89 In both cellular compartments RNase H1 ASOs essentially double the endogenous rates of clearanc

90 lls and were able to recapitulate hepatocyte ASO uptake and activity in cells engineered to heterolog

91 al modifications and further demonstrate how ASO-protein interactions influence the localization of A

92 Together, these studies identify ASO-mediated reduction of prelamin A as a potential stra

93 However, the ApoC-III ASO did not lower TG levels in mice lacking both LDLR an

94 ApoC-III ASO treatment lowered plasma TGs in mice lacking lipopro

95 DLR and LRP1 were also required for ApoC-III ASO-induced reduction of plasma TGs in mice fed a high-f

96 Importantly, ASOs targeting ACP1 mRNA significantly increased the lev

97 Advances in ASO chemistry, biological understanding, and clinical de

98 ar ASO levels, suggesting potential roles in ASO nuclear accumulation.

99 and the position of modified nucleotides in ASOs affected translation of a pORF.

100 This dramatically increased ASO-binding landscape together with relatively high pote

101 other hand, Ku70 and Ku80 proteins inhibited ASO activity, most likely by competition with RNase H1 f

102 Interestingly, ASO treatment starting at the onset of amyloid depositio

103 Interestingly, ASO-29 treatment at P1, P5 or P15 resulted in sufficient

104 eport the identification of 56 intracellular ASO-binding proteins using multi-step affinity selection

105 Recently we identified many intracellular ASO-binding proteins and found that protein binding coul

106 H1 is necessary for the activity of DNA-like ASOs.

107 intolerance for further DMPK loss may limit ASO therapy, especially since mice with Dmpk gene deleti

108 own in mice treated with non-hepatotoxic LNA ASOs, while the levels of many unintended transcripts we

109 ficacious hepatotoxic or non-hepatotoxic LNA ASOs.

110 reduced in mice treated with hepatotoxic LNA ASOs.

111 NA transcripts commonly reduced by toxic LNA ASOs were generally not strongly associated with any par

112 uction in the level of VARS altered lysosome/ASO localization patterns, implying that these proteins

113 s an exciting therapeutic target, and Malat1 ASOs represent a potential therapy for inhibiting breast

114 models, antisense oligonucleotide-mediated (ASO-mediated) skipping of exon 6 decreased MDM4 abundanc

115 cies upon treatment with chemically modified ASOs targeting 5' UTR inhibitory regions in the mRNAs en

116 Here we used a class of modified ASOs that bind to mRNA sequences in upstream open readin

117 ke of unconjugated phosphorothioate modified ASOs in the liver as evidenced by the loss of activity o

118 Nicotinamide N-methyltransferase (NNMT)-ASO prevents diet-induced obesity in mice.

119 NNMT-ASO and ApoB-ASO are chemically conjugated with T3 using

120 Both NNMT-ASO-T3 (NAT3) and ApoB-ASO-T3 (AAT3) enhance thyroid hor

121 Depletion of La and NPM1 reduced nuclear ASO levels, suggesting potential roles in ASO nuclear ac

122 igene system to determine the time course of ASO activity in the cell.

123 ded C9ORF72 and establish the feasibility of ASO-mediated therapy.

124 SO-FUS binding illustrated the importance of ASO backbone and hydrophobic 2' sugar modifications and

125 Notably, initiation of ASO treatment after disease onset restored myelination,

126 expression after treatment with 200 pmol of ASO and demonstrated versatility.

127 receptor (ASGR) has improved the potency of ASO drugs approximately 30-fold in the clinic (1).

128 t a viable approach for enhancing potency of ASO drugs in the clinic without adding significant compl

129 ular compartments is limiting to the rate of ASO activity.

130 yzed the structure-activity-relationships of ASO-protein interactions and found 2'-modifications sign

131 valent to Nucleofection(R) over a variety of ASO concentrations (24h post-transfection) and during a

132 ncrease in ER stress in the first 3 weeks of ASO treatment, followed by development of ER autophagy a

133 reduction caused significant accumulation of ASOs in early endosomes (EEs) and reduced localization i

134 The activities of ASOs in enhancing translation were sequence and position

135 eterozygous knockouts, the administration of ASOs reduced Dmpk expression in cardiac and skeletal mus

136 in mind regarding the human applicability of ASOs, we anticipate ongoing in vivo research and clinica

137 , and imply that altering protein binding of ASOs using different chemical modifications can improve

138 The conjugation of ASOs to a receptor ligand can dramatically increase thei

139 ne back-fusion for the cytosolic delivery of ASOs and siRNA, which would account for the lack of toxi

140 Transuterine delivery of ASOs is an innovative platform for developing fetal ther

141 We conclude that intra-amniotic delivery of ASOs is well tolerated and produces a sustained effect o

142 o research and clinical trial development of ASOs for the treatment of neurodegenerative diseases.

143 n interactions influence the localization of ASOs.

144 l studies are demonstrating the potential of ASOs as a source of drugs to treat neurological disease.

145 ally affects the pharmacologic properties of ASOs.

146 This suggests that for a certain set of ASOs containing high affinity modifications such as LNA,

147 These results support the use of ASOs as a potential treatment for CMT1A and elucidate po

148 Objective: To review the use of ASOs for the treatment of neurological disorders.

149 on as a therapeutic strategy, and the use of ASOs is now being applied to bypass mutations and genera

150 Work is progressing on the use of ASOs to activate the normally silent paternal copy of th

151 The use of ASOs to alter gene-splicing to treat spinal muscular atr

152 0 to 2016 for articles describing the use of ASOs to treat disease, with specific attention to neurol

153 ally administered antisense oligonucleotide (ASO) inhibited miR-182 in the kidneys up to 96 hours.

154 treatment with an antisense oligonucleotide (ASO) targeted to the site of the mutation.

155 tified an exon 11 antisense oligonucleotide (ASO) that increased lamin C production at the expense of

156 red a novel Gen 2 antisense oligonucleotide (ASO) that inhibits angiotensinogen (Agt) synthesis to li

157 onents persist in antisense oligonucleotide (ASO) therapeutics because it has been feasible neither t

158 Antisense oligonucleotide (ASO) therapeutics show tremendous promise for the treatm

159 oxyethyl chimeric antisense oligonucleotide (ASO) to decrease hepatic and adipose expression of beta-

160 We utilize an antisense oligonucleotide (ASO) to reduce apoE expression in the brains of APP/PS1-

161 that therapeutic antisense oligonucleotide (ASO) treatment can effectively target the central nervou

162 We used an antisense oligonucleotide (ASO)-based inducible mouse model of SMA to investigate t

163 ly reported on an antisense oligonucleotide (ASO-29) that dramatically improves auditory function and

164 ptor genes (allele-specific oligonucleotide [ASO]-PCR) are claimed to meet these criteria, but classi

165 vivo potency of antisense oligonucleotides (ASO) has been significantly increased by reducing their

166 eted delivery of antisense oligonucleotides (ASO) to hepatocytes via the asialoglycoprotein receptor

167 the synthesis of antisense-oligonucleotides (ASO) and thyroid hormone T3 conjugates for obesity treat

168 inhibition using antisense oligonucleotides (ASOs) and combine this approach with an RTC to effective

169 of mRNAs include antisense oligonucleotides (ASOs) and RNA interference (RNAi).

170 Antisense oligonucleotides (ASOs) and RNA-interference approaches are emerging as at

171 e limited use of antisense oligonucleotides (ASOs) and siRNA as therapeutics.

172 Antisense oligonucleotides (ASOs) are an established tool for the therapeutic modula

173 ase H1-dependent antisense oligonucleotides (ASOs) are chemically modified to enhance pharmacological

174 Antisense oligonucleotides (ASOs) are most commonly designed to reduce targeted RNA

175 Antisense oligonucleotides (ASOs) are often used to downregulate gene expression or

176 Antisense oligonucleotides (ASOs) are small sequences of DNA able to target RNA tran

177 Antisense oligonucleotides (ASOs) are versatile tools that can regulate multiple ste

178 ly SMN-restoring antisense oligonucleotides (ASOs) at the age of onset can extend survival and rescue

179 High affinity antisense oligonucleotides (ASOs) containing bicylic modifications (BNA) such as loc

180 expression with antisense oligonucleotides (ASOs) could provide a new treatment strategy for disease

181 mically modified antisense oligonucleotides (ASOs) designed to mediate site-specific cleavage of RNA

182 Antisense oligonucleotides (ASOs) designed to serve as substrates for RNase H1 were

183 re, we show that antisense oligonucleotides (ASOs) effectively suppress PMP22 mRNA in affected nerves

184 DNA-based antisense oligonucleotides (ASOs) elicit cleavage of the targeted RNA by the endorib

185 g mice with apoB antisense oligonucleotides (ASOs) for 6 weeks decreased VLDL secretion and plasma ch

186 Antisense oligonucleotides (ASOs) for ApoC-III reduce plasma TGs in primates and mic

187 te (PS)-modified antisense oligonucleotides (ASOs) have been extensively investigated over the past t

188 TTR levels with antisense oligonucleotides (ASOs) improves glucose metabolism and insulin sensitivit

189 of Malat1 using antisense oligonucleotides (ASOs) in the MMTV (mouse mammary tumor virus)-PyMT mouse

190 of SMN-AS1 with antisense oligonucleotides (ASOs) increases SMN expression in patient-derived cells,

191 delivery of Dnm2 antisense oligonucleotides (ASOs) into Mtm1KO mice efficiently reduces DNM2 protein

192 Antisense oligonucleotides (ASOs) modified with phosphorothioate (PS) linkages and d

193 We evaluated antisense oligonucleotides (ASOs) targeting Angptl3 messenger RNA (mRNA) for effects

194 ose injection of antisense oligonucleotides (ASOs) that target repeat-containing RNAs but preserve le

195 n be enhanced by antisense oligonucleotides (ASOs) that target upstream open reading frames.

196 clude the use of antisense oligonucleotides (ASOs) to alter splicing or knock-down expression of a tr

197 on skipping uses antisense oligonucleotides (ASOs) to alter transcript splicing for the purpose of re

198 s DMPK-targeting antisense oligonucleotides (ASOs) to reduce levels of toxic RNA.

199 ssfully utilized antisense oligonucleotides (ASOs) to reduce PMP22 and ameliorated neuropathy in both

200 Antisense oligonucleotides (ASOs) with phosphorothioate (PS) linkages are broadly us

201 roRNAs (miRNAs), antisense oligonucleotides (ASOs), aptamers, synthetic mRNAs and CRISPR-Cas9, have g

202 splice-switching antisense oligonucleotides (ASOs), we increased the synthesis of Mcl-1S, which induc

203 by screening 152 antisense oligonucleotides (ASOs).

204 g Ube3a-ATS with antisense oligonucleotides (ASOs).

205 tested proteins had no significant effect on ASO activity; however, some proteins, including La/SSB,

206 of liver dysfunction observed in the parent ASO at a similar silencing effect.

207 the uptake of unconjugated phosphorothioate ASOs into hepatocytes.

208 In HeLa cells, at 200 pmol ASO (with PA:LFN-GAL4), 5.4 +/- 2.0% Synt5 expression wa

209 no loss of antisense activity above 30 pmol ASO was evident.

210 models and human clinical trials have proven ASOs both safe and effective.

211 esterolemia and over 35 second generation PS ASOs are at various stages of clinical development.

212 ught to bind DNA, do bind and internalize PS ASOs.

213 uptake and intracellular distribution of PS ASOs are mediated by protein interactions.

214 RE and 190-HARE), we have determined that PS ASOs bind with high affinity and these receptors are res

215 ntified, and intracellular sites in which PS ASOs are active, or inactive, cataloged.

216 the transport of PS-ASOs to LEs, as ANXA2/PS-ASO co-localization was observed inside LEs.

217 tion of Alix also substantially decreased PS-ASO activities without affecting total PS-ASO uptake.

218 nti-LBPA antibody significantly decreased PS-ASO activities without affecting total PS-ASO uptake.

219 reduced localization in LEs and decreased PS-ASO activity.

220 r results indicate that ANXA2 facilitates PS-ASO trafficking from early to late endosomes where it ma

221 Importantly, the kinetics of PS-ASO activity upon free uptake show that target mRNA redu

222 ugh ANXA2 appears not to affect levels of PS-ASO internalization, ANXA2 reduction caused significant

223 This study was focused on the details of PS-ASO trafficking through endocytic pathways.

224 hich are supported by LBPA, contribute to PS-ASO intracellular release from LEs.

225 endosomes where it may also contribute to PS-ASO release.

226 PS-ASO activities without affecting total PS-ASO uptake.

227 PS-ASO activities without affecting total PS-ASO uptake.

228 e show that co-localization of ANXA2 with PS-ASO is not dependent on their direct interactions or med

229 PS-ASOs are known to be internalized via a number of endocy

230 PS-ASOs can enter cells without additional modification or

231 PS-ASOs containing more hydrophobic 2'-modifications exhibi

232 PS-ASOs exited early endosomes (EEs) rapidly after internal

233 RNA reduction occurs at least 4 hrs after PS-ASOs exit from EEs and is coincident with release from L

234 Although PS-ASOs function in both the cytoplasm and nucleus, localiz

235 We found that Hsp90 protein binds PS-ASOs containing locked-nucleic-acid (LNA) or constrained

236 Inside LEs, PS-ASOs and LBPA were co-localized in punctate, dot-like st

237 Chemically modified PS-ASOs can mediate efficient target reduction by site-spec

238 ic acid (LBPA) is required for release of PS-ASOs from LEs.

239 stead, ANXA2 accompanies the transport of PS-ASOs to LEs, as ANXA2/PS-ASO co-localization was observe

240 on of Hsp90 protein decreased activity of PS-ASOs with 5'-LNA or 5'-cEt wings, but not with 5'-MOE wi

241 ns can improve therapeutic performance of PS-ASOs.

242 The mechanisms by which PS-ASOs are released from membrane-enclosed endocytotic org

243 and limited co-localization of LBPA with PS-ASOs at ILVs inside LEs.

244 that annexin A2 (ANXA2) co-localizes with PS-ASOs in late endosomes (LEs) and enhances ASO activity.

245 ers, nor to synthesize stereochemically pure ASOs.

246 ury (mg/dL; median and interquartile range): ASO 2.5mg/kg: 1.9 (1.3; 3.2), ASO 25mg/kg: 2.8 (0.7; 5.0

247 Strikingly, Usher mice receiving ASO-29 treatment have normal or elevated vestibular resp

248 The resulting ASO conjugates were evaluated in ASGR binding assays, in

249 rminal region of FUS is sufficient to retain ASOs in cellular foci.

250 The same ASO also reduced the expression of progerin, the mutant

251 terquartile range of overall injury scores): ASO both concentrations 1 (1, 1), saline 3 (3, 3) and MM

252 l (100 mg/kg/d), PBS (control), or scrambled ASO (66 mg/kg/wk) for 10 wk, followed by tissue collecti

253 those seeded by the aggregation of specific ASO-binding proteins such as FUS/TLS (FUS) and PSF/SFPQ

254 riplet stereochemical code in the stereopure ASOs, 3'-SpSpRp, that promotes target RNA cleavage by RN

255 e durable response in mice than stereorandom ASOs.

256 and three GalNAc conjugated single stranded ASOs bind the ASGR with the strongest affinity and displ

257 These findings support ASOs as a promising approach for treating some human neu

258 Synthetic ASOs can recognize cellular RNA and control gene express

259 Moreover, systemic ASO injection into severely affected mice leads to rever

260 ed that a murine-specific ApoC-III-targeting ASO reduces fasting TG levels through a mechanism that i

261 post-maturity knockdown using Dmpk-targeting ASOs in mice with heterozygous deletion.

262 els for type II/III SMA, we demonstrate that ASO therapy can be effective even when administered afte

263 We hypothesize that ASO-T3 conjugates may knock down target genes and enrich

264 We have shown that ASO treatment diminished aberrant splicing and increased

265 The current study suggests that ASO treatment may serve as a viable approach to correcti

266 We hypothesize that ASOs administered to the amniotic cavity will gain entry

267 Here, we report that ASOs with specific backbone and sugar modifications can

268 The ASO-mediated increase in protein expression was sequence

269 Estimates of the time required for the ASO to enter and traverse the cell, scan the target mRNA

270 , e.g. (S)-cEt or LNA, in the 5'-wing of the ASO.

271 We have shown that the ASO gapmers can interact with the Ago-2 PAZ domain and c

272 The ASOs appear to improve the recruitment of translation in

273 were also achieved by administration of the ASOs late after onset, independent of the restoration of

274 ion, which was then rescued by a therapeutic ASO.

275 le synthetic process that yields therapeutic ASOs having high stereochemical and chemical purity.

276 Therefore, ASO and hormone/drug conjugation may provide a novel str

277 Thus, ASO-mediated DNM2 knockdown can efficiently correct musc

278 evaluating target engagement in response to ASO therapy.

279 from six distinct scaffolds and attached to ASOs.

280 ailable starting materials and conjugated to ASOs using a solution phase conjugation strategy.

281 TTR-ASO treatment decreased LDL cholesterol in high-fat diet

282 TTR-ASO treatment of mice with genetic or diet-induced obesi

283 % in high-fat diet-fed mice treated with TTR-ASOs, demonstrating improved insulin sensitivity.

284 tivity of GalNAc conjugated and unconjugated ASOs in ASGR knockout mice.

285 can affect protein binding and understanding ASO-protein interactions is important for better drug de

286 Upon ASO treatment, levels of SDHB in patient myotubular cell

287 Using ASO treatment, we increased the amount of proteins expre

288 e chosen for more extensive evaluation using ASOs targeting SRB-1, A1AT, FXI, TTR, and ApoC III mRNAs

289 tein can also be selectively increased using ASOs designed to hybridize to other translation inhibito

290 support the feasibility and safety of using ASOs for post-transcriptional silencing of DMPK in muscl

291 cRNAs were more effectively suppressed using ASOs, cytoplasmic lncRNAs were more effectively suppress

292 putative cytoplasmic mechanism through which ASO gapmers silence their targets when transfected or de

293 horough understanding of mechanisms by which ASOs are internalized in cells and their intracellular t

294 esting a therapeutic benefit to balance with ASO-29 treatment at P15 despite the profound vestibular

295 Notably, systemic treatment of mice with ASO resulted in an approximately 80% protein increase of

296 In contrast, treatment of mice with ASO-29 treatment at P15 was minimally effective at rescu

297 expression networks that were restored with ASO treatment, we also identified potential disease biom

298 NA surveillance prior to treating cells with ASOs or siRNA and analyzed cleavage products by RACE.

299 VARS and ANXA2 co-localized with ASOs in endocytic organelles, and reduction in the level

300 ited ALS (caused by a mutation in Sod1) with ASOs against Sod1 has been shown to substantially slow d