ライフサイエンスコーパス: SMA

1 SMA is caused by low levels of the survival motor neuron

2 SMA is characterized by alpha-lower motor neuron loss an

3 SMA is characterized by loss of motor neurons, but the u

4 SMA is subdivided into four main types, with type I bein

5 SMA patients have deletions and other mutations of the s

6 SMA results from reduced levels of the ubiquitously expr

7 SMA therapeutics development efforts have focused on ide

8 SMA-affected individuals harbor low SMN expression from

9 SMA-causing missense mutations that block multimerizatio

10 rospective natural history study enrolled 26 SMA infants and 27 control infants aged <6 months.

11 r function in patients with type 2 or type 3 SMA over a period of 24 months.

12 ly confirmed type 2 or non-ambulatory type 3 SMA.

13 atients with type 2 or non-ambulatory type 3 SMA.

14 ucts and demonstrate the potential for ES-62 SMA-based DC therapy in inflammatory disease.

15 y synthesized as a small molecular acceptor (SMA) for nonfullerene polymer solar cells (PSCs).

16 We report a novel small molecule acceptor (SMA) named FTTB-PDI4 obtained via ring-fusion between th

17 ceptor (A-D-A)-type small molecule acceptor (SMA), and an indaceno[1,2-b:5,6-b']dithiophene-based SMA

18 ibes the preparation of styrene maleic acid (SMA) co-polymer to extract membrane proteins from prokar

19 The styrene-maleic acid (SMA) copolymer is rapidly gaining attention as a tool in

20 istochemical studies of smooth muscle actin (SMA), D2-40, CD34, and glucose transporter 1 (GLUT-1).

21 ility, apoptosis, alpha-smooth muscle actin (SMA), fibronectin (FN) production, and collagen remodeli

22 Active SMAs also resulted in a DC phenotype that exhibited redu

23 enhancing mean survival in severely affected SMA model mice by approximately 40%.

24 trons, in the spinal cord of mice 30 d after SMA induction, which was then rescued by a therapeutic A

25 nd -bundling protein, fully protects against SMA in SMN1-deleted individuals carrying 3-4 SMN2 copies

26 ABSTARCT: Shape memory alloys (SMAs) have the ability to show large recoverable shape c

27 functional stability of shape memory alloys (SMAs), especially for high temperature applications.

28 healing via promotion of expression of alpha SMA and TGF beta, neovascularization and re-epithelializ

29 pression of alpha-smooth muscle actin (alpha-SMA) and Collagen I were reduced as was the activity of

30 pression of alpha-smooth muscle actin (alpha-SMA) and collagen in fibrotic live tissues.

31 tion marker alpha-smooth muscle actin (alpha-SMA) in rat and mouse HSCs.

32 Alpha-smooth muscle actin (alpha-SMA) is a marker of activated fibroblasts and a poor pro

33 pression of alpha-smooth muscle actin (alpha-SMA), a marker of PSC activation.

34 ynthesis of alpha-smooth muscle actin (alpha-SMA), collagen 1 (COL1), and fibronectin (FN) in respons

35 neration of alpha-smooth muscle actin (alpha-SMA)-positive myofibroblasts that produced ECM proteins,

36 ration, and alpha-smooth muscle actin (alpha-SMA)-positive myofibroblasts.

37 in (FN) and alpha-smooth muscle actin (alpha-SMA).

38 mity to the alpha-smooth muscle actin (alpha-SMA+) area within mild fibrosis regions; while in severe

39 erentiated (alpha-smooth muscle actin [alpha-SMA](+)) fibrocytes were increased in asthmatic patients

40 and significantly reduces collagen and alpha-SMA expression in an animal model of liver fibrosis.

41 satinib also reduced proliferation and alpha-SMA expression in fibroblasts.

42 Cells positive for MMP-9, MMP-13, and alpha-SMA expression were present at the areas of epithelial i

43 eta-1-induced the expression of FN and alpha-SMA in human lung fibroblast.

44 MT, and the expression of vimentin and alpha-SMA in our diabetic cataract model in vitro.

45 umbers of circulating alpha-SMA(+) and alpha-SMA(+)CXCR4(+) fibrocytes were increased in asthmatic pa

46 liver IL-6, IL-17A, IL-17F, TGF-beta1, alpha-SMA, TGR5, NTCP, OATP1a1, and ileum ASBT and decreased l

47 on of EndMT markers (Slug, N-cadherin, alpha-SMA) in EC exposed to low shear stress.

48 In addition, numbers of circulating alpha-SMA(+) and alpha-SMA(+)CXCR4(+) fibrocytes were increase

49 creased infiltration of EP4 expressing alpha-SMA myofibroblasts, identifying a potential mechanism of

50 nd TGF-beta (Smad2/3, collagen type I, alpha-SMA) signaling, respectively.

51 , which correlated with an increase in alpha-SMA protein expression.

52 In contrast, a dramatic increase in alpha-SMA(+), EP4(+) double-positive cells were observed in EP

53 ated the TGF-beta1-induced increase in alpha-SMA, COL1, and FN expression.

54 Wnt/beta-catenin signaling, increasing alpha-SMA, COL1, and FN synthesis.

55 to the effects of hemoglobin inducing alpha-SMA expressions in cultured pericytes and brain slices v

56 n increase in the myofibroblast marker alpha-SMA, production and assembly of FN, and contraction of c

57 downregulated, while the expression of alpha-SMA and vimentin was upregulated in diabetic cataract ti

58 al cells promoted the proliferation of alpha-SMA(+) myofibroblasts in a paracrine manner.

59 oblasts as reflected by high levels of alpha-SMA, IL6, and MMP9.

60 at the TSS of transcript variant 2 of alpha-SMA, which correlated with an increase in alpha-SMA prot

61 re, by targeting the eNOS and pericyte alpha-SMA phenotype, our present data may shed new light on th

62 nhibition of FAK activation blocks the alpha-SMA and collagen expression, and inhibits the formation

63 FAK activation precedes the alpha-SMA expression in HSCs.

64 ctly attached to or separated from the alpha-SMA+ region.

65 ith SMN2 splicing modification to ameliorate SMA and demonstrates the promise of combinatorial ASOs f

66 tly, NCALD knockdown effectively ameliorates SMA-associated pathological defects across species, incl

67 tified plastin 3 (PLS3) overexpression as an SMA protective modifier in humans and showed that SMN de

68 In an SMA mouse model, m2R levels were increased and pharmacol

69 seq study that covers multiple tissues in an SMA mouse model.

70 drug-like PC-based small molecule analogues (SMAs) was synthesised.

71 ajority of cold-induced beige adipocytes and SMA-marked progenitors appeared essential for beiging.

72 stive, GLUT-1-negative endothelial cells and SMA-positive pericytes arranged in macro- or microlobule

73 At day 0, children with CM and SMA had greater values of C-reactive protein, ferritin,

74 Two SMA-based strains, SMA-Cre(ERT2) and SMA-rtTA, fate mapped into the majority of cold-induced

75 n conformational flexibility between LEN and SMA.

76 peutic monitoring of symptomatic newborn and SMA patients, respectively.

77 onfirmed dissociable roles of the preSMA and SMA in determining response costs.

78 CS intensity when conditioning PMv, rM1 and SMA at a 40-ms ISI, with larger effects after PMv condit

79 The only approved SMA treatment is an antisense oligonucleotide that targe

80 oncentration in the Seoul Metropolitan Area (SMA), South Korea, are puzzling.

81 pain, whereas the supplementary motor area (SMA) and pre-SMA are specifically associated with higher

82 l cortex (PFC) and supplementary motor area (SMA) during emotion regulation, although only change in

83 etermined that the supplementary motor area (SMA) plays an important role in the interlimb transfer o

84 rtex (rPMd) or the supplementary motor area (SMA) prior to the TS at various CS-TS inter-stimulus int

85 en right and left supplementary motor areas (SMA) was elevated after training.

86 aneurysm of the superior mesenteric artery (SMA) with a diameter of 2.2 cm was found incidentally on

87 neuromuscular deficits and diseases such as SMA, as well as for next generation prosthetics, utilizi

88 tor neuron diseases spinal muscular atrophy (SMA) and amyotrophic lateral sclerosis (ALS).

89 ebellar hypoplasia, spinal muscular atrophy (SMA) and central nervous system demyelination.

90 f childhood such as spinal muscular atrophy (SMA) and neuronal ceroid lipofuscinosis (NCLs).

91 Spinal muscular atrophy (SMA) is a common and often fatal neuromuscular disorder

92 underlying cause of spinal muscular atrophy (SMA) is a deficiency of the survival motor neuron (SMN)

93 Spinal muscular atrophy (SMA) is a devastating neuromuscular disorder characteriz

94 Spinal muscular atrophy (SMA) is a genetic disorder characterized by loss of moto

95 Spinal muscular atrophy (SMA) is a hereditary neurodegenerative disease with seve

96 Spinal muscular atrophy (SMA) is a major inherited cause of infant death worldwid

97 Spinal muscular atrophy (SMA) is a motoneuron disease caused by loss or mutation

98 Spinal muscular atrophy (SMA) is a neurodegenerative disease characterized by pro

99 Spinal muscular atrophy (SMA) is a neuromuscular disease caused by low levels of

100 Spinal muscular atrophy (SMA) is a neuromuscular disease caused by reduced expres

101 Spinal Muscular Atrophy (SMA) is a neuromuscular disorder caused by insufficient

102 Spinal muscular atrophy (SMA) is a progressive motor neuron disease causing loss

103 Spinal muscular atrophy (SMA) is a progressive neurodegenerative disease that is

104 Spinal muscular atrophy (SMA) is an autosomal-recessive disorder characterized by

105 Spinal muscular atrophy (SMA) is caused by deficiency of SMN protein, which is cr

106 Spinal muscular atrophy (SMA) is caused by deletions or mutations of Survival Mot

107 Spinal muscular atrophy (SMA) is caused by deletions or mutations of the Survival

108 Spinal muscular atrophy (SMA) is caused by depletion of the ubiquitously expresse

109 Spinal Muscular Atrophy (SMA) is caused by diminished Survival of Motor Neuron (S

110 Spinal muscular atrophy (SMA) is caused by homozygous mutations in human SMN1 Exp

111 Spinal muscular atrophy (SMA) is caused by low levels of survival motor neuron (S

112 motoneuron disease spinal muscular atrophy (SMA) is caused by low levels of the survival motor neuro

113 Spinal muscular atrophy (SMA) is caused by mutation or deletion of the survival m

114 NIFICANCE STATEMENT Spinal muscular atrophy (SMA) is caused by the loss of motor neurons, but astrocy

115 Spinal muscular atrophy (SMA) is caused by the low levels of survival motor neuro

116 Spinal muscular atrophy (SMA) is the leading genetic cause of infant death.

117 Infantile-onset spinal muscular atrophy (SMA) is the most common genetic cause of infant mortalit

118 y type 1 (CDM1) and spinal muscular atrophy (SMA) patients.

119 motor neurons from Spinal Muscular Atrophy (SMA) type 1 patient's.

120 Spinal muscular atrophy (SMA), a leading genetic disease of children and infants,

121 in a mouse model of spinal muscular atrophy (SMA), a reduction in proprioceptive synaptic drive leads

122 Spinal muscular atrophy (SMA), an autosomal recessive neuromuscular disease, is t

123 motoneuron disease spinal muscular atrophy (SMA), motor axons fail to form the normal extent of axon

124 Spinal muscular atrophy (SMA), the leading genetic cause of infant mortality, is

125 Spinal muscular atrophy (SMA), the leading genetic cause of infant mortality, pre

126 romuscular disorder spinal muscular atrophy (SMA), the most common inherited killer of infants, is ca

127 us SMN1 loss causes spinal muscular atrophy (SMA), the most common lethal genetic childhood motor neu

128 in a mouse model of spinal muscular atrophy (SMA).

129 tential therapy for spinal muscular atrophy (SMA).

130 d an indaceno[1,2-b:5,6-b']dithiophene-based SMA, namely IDTN, by end-capping with the naphthyl fused

131 Defects in contact interactions between SMA motoneurons and astrocytes impair synaptogenesis see

132 erhemispheric interactions between bilateral SMA play an instrumental role in CE and that the structu

133 fiber density in tracts connecting bilateral SMA were negatively correlated with and predictive of tr

134 cles of the hind-paw were vulnerable in both SMA and ALS, with a loss of neuronal innervation and shr

135 brane potential in motor neurons affected by SMA.

136 located patients (2:1 with stratification by SMA type and centre) to receive olesoxime (10 mg/kg per

137 s to remain with native lipid, surrounded by SMA.

138 ron markers at day 0 among study groups (CM, SMA, and CC groups) and at day 28 between children in ea

139 Four sulfone-containing SMAs termed 11a, 11e, 11i and 12b were found to reduce m

140 MA) activity tracked task-set control costs, SMA activity tracked response-set control costs, and bas

141 this for a high temperature ternary Ti-Pd-Cr SMA by achieving both a small DeltaT and high transforma

142 Our results are not limited to Ti-Pd-Cr SMAs but potentially provide a strategy for searching fo

143 in the peripheral nervous system by creating SMA mice selectively overexpressing SMN in myelinating S

144 onstrates modifier impact in three different SMA animal models, and suggests a potential combinatoria

145 protein, survive into adulthood and display SMA disease-relevant pathologies.

146 sed for future development of drugs for DM1, SMA, and other chronic diseases where GSK-3beta inhibiti

147 immunoglobulin light-chain variable domain, SMA, associated with AL amyloidosis, were investigated b

148 of SMA, NMR studies were performed for each SMA-like point mutant of LEN followed by in silico analy

149 ) were resistant to degeneration in endstage SMA mice, as well as in late symptomatic ALS mice.

150 mplications of this therapeutic landmark for SMA therapeutics and discuss how future developments wil

151 development of this series of molecules for SMA treatment.

152 rst precise molecularly targeted therapy for SMA (nusinersen), and a pivotal proof of principle that

153 linical potential of peptide-PMO therapy for SMA.

154 e is currently no FDA-approved treatment for SMA, early therapeutic efforts have focused on testing r

155 ntially provide a strategy for searching for SMAs with small thermal hysteresis.

156 ologous light-chain, LEN, which differs from SMA at eight positions but is non-amyloidogenic in vivo,

157 ndrial network in primary motor neurons from SMA mice, with no change in mitochondria density.

158 rget of SMN and show that motor neurons from SMA mouse models have reduced levels ofGAP43mRNA and pro

159 ntron retention in all examined tissues from SMA mice, and that U12-dependent intron retention is ind

160 er motoneurons, astrocytes or both were from SMA mice compared with those in WT motoneurons coculture

161 It is not known how SMA composition affects the solubilization behavior of S

162 fects are observed in the most severe Type I SMA patients and most of the widely used SMA mouse model

163 Importantly, SMA severity is correlated with decreased snRNP assembly

164 In SMA mice or after the blockade of proprioceptive synapti

165 In SMA mice, SMN-deficiency causes down-regulation of ZPR1

166 increase the level of SMN protein by >50% in SMA patient-derived fibroblasts at concentrations of <16

167 ved macrophages, were found to be altered in SMA spleens even in pre-symptomatic post-natal day 2 ani

168 Ns) are the most obviously affected cells in SMA, recent evidence suggest dysfunction in multiple cel

169 e specific defects in the motor circuitry in SMA is still unclear, but SMN has also been implicated i

170 s were identified between TIMPSI and CMAP in SMA infants.

171 d the spleen and immunological components in SMA mice.

172 ne whether the immune system is comprised in SMA, we analyzed the spleen and immunological components

173 Conversely, in SMA changes appeared concomitantly at the neuromuscular

174 Respiratory failure causes death in SMA but the underlying molecular mechanism is unknown.

175 we report that MCP1 levels are decreased in SMA mice and that replacement of deficient MCP1 increase

176 cones and rescues axon outgrowth defects in SMA neurons.

177 a critical role for mitochondrial defects in SMA pathogenesis and suggest a novel target for improvin

178 ential role for immunological development in SMA.

179 strategies to treat respiratory distress in SMA.

180 s a novel aspect of astrocyte dysfunction in SMA and indicates a possible approach for improving moto

181 m2R expression, and neuronal dysfunction in SMA.

182 ) SMNDelta7, provides a protective effect in SMA model mice and human motor neuron cell culture syste

183 ne of the analogs found to be efficacious in SMA mouse model.

184 , as NCALD knockdown improves endocytosis in SMA models and ameliorates pharmacologically induced end

185 analysis of survival to combined endpoint in SMA infants with 2 copies of SMN2 indicated a median age

186 logy, and impair essential liver function in SMA.

187 may explain disrupted Ca(2+) homeostasis in SMA and activation of Cdk5.

188 Hypovascularity in SMA mouse spinal cord was accompanied by significant fun

189 Astrocytes have been implicated in SMA as in in other neurodegenerative disorders.

190 es expressed in motor neurons is involved in SMA pathogenesis, but increasing evidence indicates that

191 junctions was restored to control levels in SMA-PLS3 mice.

192 on and neuromuscular junction maintenance in SMA.

193 contribute to the axonal defects observed in SMA.

194 ibute to the axonal degeneration observed in SMA.

195 h to treat CNS and peripheral pathologies in SMA.

196 liorated neuromuscular junction pathology in SMA mice.

197 ture contribute to motor neuron pathology in SMA.

198 no comprehensive study of liver pathology in SMA.

199 s is a major cellular mechanism perturbed in SMA and emphasize the power of protective modifiers for

200 1 and 53 have neuroprotective properties in SMA-derived cells.

201 action potential (CMAP) decreased rapidly in SMA infants, whereas MFS in all healthy infants rapidly

202 development and point to a role for RBPs in SMA.SIGNIFICANCE STATEMENT In zebrafish models of the mo

203 ved neurotrophic factor (GDNF) is reduced in SMA astrocytes.

204 Importantly, this activity is reduced in SMA patients, raising the possibility that the aetiology

205 ZPR1 is down regulated in SMA patients.

206 We propose that defects in intron removal in SMA promote DNA damage in part through the formation of

207 c in vivo, we find that multiple residues in SMA clustered around the N-terminus and CDR loops experi

208 DNF expression does not play a major role in SMA pathology as viral-mediated GDNF re-expression did n

209 trocyte mechanisms that could play a role in SMA pathology.

210 and astrocytes impair synaptogenesis seen in SMA pathology, possibly due to the disruption of the Eph

211 at miR-146a was significantly upregulated in SMA induced pluripotent stem cell (iPSC)-derived astrocy

212 f death or permanent invasive ventilation in SMA infants.

213 involved in their selective vulnerability in SMA.

214 ccessful isolation of membrane proteins into SMA lipid particles (SMALPs) allows the proteins to rema

215 Herein, we introduce a novel ca. 1.6 kDa SMA-based polymer with styrene:maleic acid moieties that

216 hite matter fibers connecting right and left SMA predicts the benefit that an individual derives from

217 a contributing factor in astrocyte-mediated SMA pathology.

218 We utilized a mild SMA model (C/C model) to examine the impact of low SMN o

219 llele C (C (+/+)) mouse recapitulates a mild SMA-like phenotype and offers an ideal system to monitor

220 Most SMA patients have SMN1 deletions, leaving SMN2 as sole S

221 ificant reduction in spleen size in multiple SMA mouse models and a pathological reduction in red pul

222 en SMN function and the distal neuromuscular SMA phenotype is an incorrectly spliced transcript or tr

223 The two new SMAs (IT-M and IT-DM) end-capped by methyl-modified dicy

224 ted to date for a large bandgap nonfullerene SMA.

225 By comparing the fused and nonfused SMAs, we show that the ring-fusion introduces several be

226 aising the possibility that the aetiology of SMA is linked to RNA metabolism.

227 oved understanding of the molecular basis of SMA, disease pathogenesis, natural history, and recognit

228 ition affects the solubilization behavior of SMA.

229 hat mRNA handling is a critical component of SMA.

230 INTERPRETATION: These data of SMA and control outcome measures delineates meaningful c

231 he majority of patients with milder forms of SMA are diagnosed at an older age.

232 Our findings uncover novel hallmarks of SMA disease progression and link SMN to general male inf

233 opment of the neurodegenerative hallmarks of SMA, implying a differential role of SMN in myeloid cell

234 ping the dynamic conformational landscape of SMA, NMR studies were performed for each SMA-like point

235 leotide (ASO)-based inducible mouse model of SMA to investigate the SMN-specific transcriptome change

236 r-specific modifier of a mild mouse model of SMA.

237 euron (SMN) protein level in mouse models of SMA.

238 n oral administration in two mouse models of SMA.

239 cells and in two transgenic mouse models of SMA.

240 his isoform of Agrin in the motor neurons of SMA model mice increases muscle fiber size, enhances the

241 ypoxia may contribute to the pathogenesis of SMA.

242 Liver is a key recipient of SMA therapies, and systemically delivered antisense trea

243 exon 7 inclusion for a potential therapy of SMA.

244 ent introns is mitigated by ASO treatment of SMA mice and that many transcriptional changes are rever

245 otential of these molecules for treatment of SMA, our work has wide-ranging implications in understan

246 complement other modalities for treatment of SMA.

247 optimization for the potential treatment of SMA.

248 Although many variants of SMA are commercially available, so far only SMA variants

249 It should be noted that properties of SMAs are highly temperature dependent.

250 of the important shape memory properties of SMAs.

251 on, and torsion) are used on bulk samples of SMAs to determine those properties.

252 SMA are commercially available, so far only SMA variants with a 2:1 and 3:1 styrene-to-maleic acid r

253 change in clinical trials in infantile-onset SMA.

254 All children with CM or SMA, as well as 35 CC, had zinc protoporphyrin (ZPP) con

255 tendency of the SMA, thus yielding a polymer:SMA blend with reasonably small domain size.

256 pendently of intensity, CS over pIFG and pre-SMA (but not over a control site) inhibited MEPs at an I

257 s the supplementary motor area (SMA) and pre-SMA are specifically associated with higher-level cognit

258 ifferential modulatory roles of pIFG and pre-SMA in M1 excitability.

259 (pIFG) and pre-supplementary motor area (pre-SMA) with M1 at rest.

260 including pre-supplementary motor area (pre-SMA), inferior frontal gyrus, caudate, and subthalamic n

261 n ventromedial PFC, insula, lateral PFC, pre-SMA, and dmPFC.

262 ) was administered over ipsilateral pIFG/pre-SMA sites before the TS at different CS-TS inter-stimulu

263 m and predictive models, mediated by the pre-SMA and its connectivity in frontostriatal circuits.

264 e STN on continue-evoked activity in the pre-SMA was predicted by interindividual differences in risk

265 In particular, the pIFG -but not the pre-SMA- exerts intensity-dependent modulatory influences ov

266 the dorsal area of the dACC and into the pre-SMA.

267 vitro, whereas miR-146a inhibition prevented SMA astrocyte-induced motor neuron loss.

268 or Neurocalcin delta (NCALD) as a protective SMA modifier in five asymptomatic SMN1-deleted individua

269 y identifies a previously unknown protective SMA modifier in humans, demonstrates modifier impact in

270 >250 days) and motoric abilities in a severe SMA mouse model.

271 oth the CNS and peripheral tissues in severe SMA mice following systemic administration.

272 ar, significant vascular depletion in severe SMA.

273 MN expression and improve survival of severe SMA mice.

274 ate here that Delta7 mice, a model of severe SMA, treated with a suboptimal dose of an SMN2 splicing

275 duced SMN levels, in a mouse model of severe SMA.

276 N deficiency in the spinal cord of SMNDelta7 SMA mice.

277 Two SMA-based strains, SMA-Cre(ERT2) and SMA-rtTA, fate mapped into the majorit

278 (unidirectional) constraint on a subordinate SMA-BG loop that determines response-selection, resultin

279 In contrast, we found that SMA astrocytes increased microRNA (miR) production and s

280 We show here that SMA astrocyte cultures derived from mouse spinal cord of

281 risk predictors were (1) R1 <1.0 mm for the SMA-margin in specific subgroups (upfront pancreatectomy

282 erm trend of surface PM concentration in the SMA declined in the 2000s, but since 2012 its concentrat

283 tion regulation, although only change in the SMA over time occurred in veterans with PTSD and not tho

284 otec, E-ventus BX((R))) was implanted in the SMA, covering the aneurysmal neck and overlapping the pr

285 uppress the self-aggregation tendency of the SMA, thus yielding a polymer:SMA blend with reasonably s

286 Since the seminal discovery of the SMA-causing gene in 1995, research has focused on the de

287 Disruption of the potential of the SMA-tracked progenitors to form beige adipocytes was acc

288 a, it can be reasonably anticipated that the SMA treatment landscape will transform significantly.

289 This SMA exhibits a relatively wide optical bandgap of 2.03 e

290 cy may thus be a new therapeutic approach to SMA.SIGNIFICANCE STATEMENT Spinal muscular atrophy (SMA)

291 N deficiency in astrocytes may contribute to SMA is unclear and what aspect of astrocyte activity is

292 xon skipping is the principal contributor to SMA severity, mechanisms governing stability of survival

293 al therapeutic strategy to efficiently treat SMA.

294 Two SMA-based strains, SMA-Cre(ERT2) and SMA-rtTA, fate mapp

295 ea of the terminal-group in these A-D-A-type SMAs is a promising approach not only for enhancing the

296 e I SMA patients and most of the widely used SMA mouse models, however, as effective therapeutics are

297 enty-nine children with CM, 77 children with SMA, and 83 CC who presented to Mulago Hospital, Kampala

298 We present a patient with SMA-like phenotype carrying a homozygous mutation in RBM

299 aningful clinical benefits for patients with SMA and, given its mode of action, might be used in comb

300 t with this, mice receiving DCs treated with SMAs exhibited significantly reduced severity of collage