ライフサイエンスコーパス: Friedreich

1 Friedreich ataxia (FA) is a neurodegenerative and cardio

2 Friedreich ataxia (FA) is a progressive genetic neurodeg

3 Friedreich ataxia (FA) is a progressive neurodegenerativ

4 Friedreich ataxia (FA) is an autosomal recessive disease

5 Friedreich ataxia (FA) is the most common ataxia and res

6 Friedreich Ataxia (FRDA) is a chronic neurodegenerative

7 Friedreich ataxia (FRDA) is a frequent autosomal recessi

8 Friedreich ataxia (FRDA) is a neurodegenerative disease

9 Friedreich ataxia (FRDA) is a neurodegenerative disorder

10 Friedreich ataxia (FRDA) is a neurodegenerative disorder

11 Friedreich ataxia (FRDA) is a rare multisystem, life-lim

12 Friedreich ataxia (FRDA) is an autosomal recessive degen

13 Friedreich ataxia (FRDA) is an autosomal recessive degen

14 Friedreich ataxia (FRDA) is an autosomal recessive neuro

15 Friedreich ataxia (FRDA) is an autosomal recessive neuro

16 Friedreich ataxia (FRDA) is an inherited neurodegenerati

17 Friedreich ataxia (FRDA) is caused by a homozygous GAA r

18 Friedreich ataxia (FRDA) is caused by an expanded GAA tr

19 Friedreich ataxia (FRDA) is caused by hyperexpansion of

20 Friedreich ataxia (FRDA) is caused by the reduced expres

21 Friedreich ataxia (FRDA) is primarily caused by an unsta

22 Friedreich ataxia (FRDA) is the most common genetic sens

23 Friedreich ataxia (FRDA) is the most common inherited at

24 Friedreich ataxia (FRDA) is typically caused by homozygo

25 Friedreich ataxia (FRDA) patients are homozygous for exp

26 Friedreich ataxia (FRDA), an autosomal recessive, neurod

27 Friedreich ataxia (FRDA), the most common hereditary ata

28 Friedreich ataxia accounts for approximately 75% of Euro

29 Friedreich ataxia is a genetic disease caused by deficie

30 Friedreich ataxia is a severe autosomal-recessive diseas

31 Friedreich ataxia is an autosomal recessive neurodegener

32 Friedreich ataxia is an early-onset multisystemic diseas

33 Friedreich ataxia is an inherited neurodegenerative dise

34 Friedreich ataxia is caused by an expanded (GAA*TTC)n se

35 Friedreich ataxia is caused by an expanded (GAA.TTC)n se

36 Friedreich ataxia is caused by expansion of a GAA triple

37 Friedreich ataxia is caused by mutations in the frataxin

38 Friedreich ataxia is caused by reduced activity of frata

39 Friedreich ataxia is caused by the expansion of a polymo

40 Friedreich ataxia is commonly caused by large expansions

41 Friedreich ataxia may be one of the most thoroughly stud

42 Friedreich ataxia patients are homozygous for expanded G

43 Friedreich ataxia patients are typically homozygous for

44 Friedreich ataxia results from frataxin insufficiency ca

45 Friedreich ataxia, a neurodegenerative disorder resultin

46 Friedreich ataxia, myotonic dystrophy type 1 and 3 forms

47 Friedreich ataxia, the most common inherited ataxia, is

48 Friedreich ataxia, the most prevalent inherited ataxia,

49 Friedreich's ataxia (FA) is a debilitating, multisystemi

50 Friedreich's ataxia (FA) is a devastating, multi-systemi

51 Friedreich's ataxia (FA) is a leading form of hereditary

52 Friedreich's ataxia (FA) is a progressive, multisystem,

53 Friedreich's ataxia (FA) is an autosomal recessive disea

54 Friedreich's ataxia (FA) is an inherited progressive neu

55 Friedreich's ataxia (FA) is the most frequently inherite

56 Friedreich's ataxia (FRDA) and ataxia with oculomotor ap

57 Friedreich's ataxia (FRDA) is a common hereditary degene

58 Friedreich's ataxia (FRDA) is a devastating, multisystem

59 Friedreich's ataxia (FRDA) is a hereditary neurodegenera

60 Friedreich's ataxia (FRDA) is a human hereditary disease

61 Friedreich's ataxia (FRDA) is a neurodegenerative diseas

62 Friedreich's ataxia (FRDA) is a neurodegenerative diseas

63 Friedreich's ataxia (FRDA) is a progressive disease affe

64 Friedreich's ataxia (FRDA) is a progressive neurodegener

65 Friedreich's ataxia (FRDA) is a severe neurodegenerative

66 Friedreich's ataxia (FRDA) is an autosomal recessive deg

67 Friedreich's ataxia (FRDA) is an autosomal recessive dis

68 Friedreich's ataxia (FRDA) is an autosomal recessive neu

69 Friedreich's ataxia (FRDA) is an autosomal recessive neu

70 Friedreich's ataxia (FRDA) is an autosomal recessive neu

71 Friedreich's ataxia (FRDA) is an autosomal-recessive neu

72 Friedreich's ataxia (FRDA) is caused by biallelic expans

73 Friedreich's ataxia (FRDA) is caused by large GAA expans

74 Friedreich's ataxia (FRDA) is caused by point mutations

75 Friedreich's ataxia (FRDA) is the most common inherited

76 Friedreich's ataxia (FRDA) is the most common inherited

77 Friedreich's ataxia (FRDA) is the result of mutations in

78 Friedreich's ataxia (FRDA), an autosomal recessive cardi

79 Friedreich's ataxia (FRDA), the most common inherited at

80 Friedreich's ataxia (FRDA), the most common inherited at

81 Friedreich's ataxia (GAA)n repeats of various lengths we

82 Friedreich's ataxia is a devastating neurological diseas

83 Friedreich's Ataxia is a genetic disease caused by expan

84 Friedreich's ataxia is a neurodegenerative disease cause

85 Friedreich's ataxia is a neurodegenerative disorder caus

86 Friedreich's ataxia is a progressive degenerative disord

87 Friedreich's ataxia is a rare autosomal recessive neurod

88 Friedreich's ataxia is an incurable genetic disorder cau

89 Friedreich's ataxia is associated with a deficiency in f

90 Friedreich's ataxia is caused by a triplet repeat expans

91 Friedreich's ataxia is caused by expansion mutations in

92 Friedreich's ataxia is caused by the massive expansion o

93 Friedreich's ataxia patients are homozygous for expanded

94 Friedreich's ataxia, an autosomal cardio- and neurodegen

95 Friedreich's ataxia, an autosomal recessive neurodegener

96 a diagnosis by Nikolaus Friedreich in 1863, Friedreich's ataxia (FA) is an autosomal recessive progr

97 ension (-14+/-6%), Fabry disease (-12+/-5%), Friedreich ataxia (-16+/-2%), or control subjects (-17+/

98 he etiology of myotonic dystrophy type 1 and Friedreich's ataxia, respectively.

99 ia type 3, spinocerebellar ataxia type 6 and Friedreich's ataxia are common hereditary ataxias.

100 reduced in spinocerebellar ataxia type 6 and Friedreich's ataxia compared to matched controls (P-valu

101 comparing spinocerebellar ataxia type 6 and Friedreich's ataxia to matched controls (P < 0.01, boots

102 pinocerebellar ataxias 1, 2, 3, 6 and 7, and Friedreich's ataxia, 132 sporadic idiopathic and 33 clin

103 oth real data sets (cystic fibrosis data and Friedreich ataxia data) and simulated data sets.

104 es neural and cardiac cell degeneration, and Friedreich's ataxia.

105 yndrome 2 (HDL2), familial prion disease and Friedreich's ataxia.

106 clinical trials in Huntington's disease and Friedreich's ataxia.

107 human disorders, such as Menkes disease and Friedreich's ataxia.

108 ed arterial hypertension, Fabry disease, and Friedreich ataxia (n=25 per group) were investigated; 25

109 TTC triplet repeats (responsible for DM1 and Friedreich's ataxia, respectively) can expand by genetic

110 (DM1), myotonic dystrophy type 2 (DM2), and Friedreich's ataxia (FRDA).

111 t expansions found in myotonic dystrophy and Friedreich's ataxia confer variegation of expression on

112 g fragile X syndrome, myotonic dystrophy and Friedreich's ataxia.

113 1 (SCA1), Machado-Joseph disease (MJD), and Friedreich ataxia.

114 < 0.05) in two Friedreich's mouse models and Friedreich's lymphocytes.

115 Fragile X mental retardation and Friedreich's ataxia were among the first pathogenic trin

116 otonic dystrophy, the fragile X syndrome and Friedreich's ataxia.

117 A, fragile X syndrome, Hunter syndrome, and Friedreich's ataxia.

118 strophy, Huntington's disease, fragile X and Friedreich's ataxia.

119 ses associated with oxidative stress such as Friedreich ataxia, spongiform encephalopathies, and Alzh

120 cluster biogenesis in mitochondria, such as Friedreich ataxia.

121 -S cluster assembly lead to diseases such as Friedreich's ataxia.

122 luded standardized neurological assessments (Friedreich Ataxia Rating Scale [FARS], International Coo

123 tive disorders such as mitochondrial ataxia, Friedreich ataxia, spinocerebellar ataxia type 2, ataxia

124 the most common autosomal recessive ataxia, Friedreich ataxia (FA).

125 genesis of the most common recessive ataxia, Friedreich ataxia.

126 Fe-S cluster biogenesis has extended beyond Friedreich ataxia to include a sideroblastic anemia with

127 long with common phenotypic traits shared by Friedreich's ataxia and FXTAS carriers (e.g. gait ataxia

128 with autosomal dominant congenital cataract, Friedreich's ataxia, and X-linked sideroblastic anemia w

129 reduce the expression of frataxin and cause Friedreich's ataxia (FRDA), an autosomal recessive neuro

130 Mutations in Frataxin (FXN) cause Friedreich's ataxia (FRDA), a recessive neurodegenerativ

131 peats in intron 1 of the frataxin gene cause Friedreich's ataxia (FRDA) by reducing frataxin mRNA lev

132 irst intron of the X25 (frataxin) gene cause Friedreich's ataxia, the most common inherited ataxia.

133 unclear where and how, and deficiency causes Friedreich ataxia.

134 of the mitochondrial protein frataxin causes Friedreich's ataxia (FRDA); the mechanism by which this

135 t in the first intron of the FXN gene causes Friedreich ataxia by reducing frataxin expression.

136 the first intron of the frataxin gene causes Friedreich's ataxia (FRDA).

137 s in the first intron of the FXN gene causes Friedreich's ataxia.

138 Low expression of frataxin in humans causes Friedreich's ataxia, an autosomal recessive neurodegener

139 deficiency of this protein in humans causes Friedreich's ataxia, while its complete absence in yeast

140 egulate the FXN gene silencing, which causes Friedreich's ataxia.

141 oteins include mutations of frataxin causing Friedreich's ataxia, PINK1, DJ1 causing Parkinson's dise

142 lled 592 patients with genetically confirmed Friedreich's ataxia between Sept 15, 2010, and April 30,

143 enrolled patients with genetically confirmed Friedreich's ataxia from 11 European study sites in Aust

144 ohort of patients with genetically confirmed Friedreich's ataxia.

145 a Ireland, Association Suisse de l'Ataxie de Friedreich, Associazione Italiana per le Sindromi Atassi

146 human subjects caused by the genetic disease Friedreich's ataxia results in decreased mitochondrial f

147 AA repeats responsible for the human disease Friedreich's ataxia.

148 tein linked to the neurodegenerative disease Friedreich ataxia, has recently been proposed as an iron

149 d to the incurable neurodegenerative disease Friedreich's ataxia (FRDA).

150 orrelated with the neurodegenerative disease Friedreich's ataxia and results in the inactivation of F

151 The progressive neurodegenerative disease Friedreich's ataxia is caused by a decreased level of ex

152 development of the neurodegenerative disease Friedreich's ataxia.

153 otein frataxin (FXN) causes the rare disease Friedreich's ataxia (FA), for which there is no successf

154 nd reduced levels lead to the human disease, Friedreich's ataxia (FRDA).

155 data on cystic fibrosis, Huntington disease, Friedreich ataxia, and progressive myoclonus epilepsy.

156 implicated in the neurodegenerative disease, Friedreich's ataxia.

157 se models for the neurodegenerative diseases Friedreich ataxia and Huntington disease.

158 However, the human diseases, Friedreich ataxia, glutaredoxin 5-deficient sideroblasti

159 The neurodegenerative disorder Friedreich's ataxia (FRDA) is caused by mutations in fra

160 loss-of-function neurodegenerative disorder Friedreich's ataxia (FRDA).

161 tosomal recessive neurodegenerative disorder Friedreich's ataxia.

162 re responsible for the neurological disorder Friedreich ataxia (FA).

163 in a hereditary neurodegenerative disorder, Friedreich's ataxia.

164 l sclerosis, nucleotide expansion disorders (Friedreich ataxia and fragile X syndrome), and cancer.

165 s for a prospective, international, European Friedreich's ataxia database registry.

166 onal Institute for Health Research, European Friedreich's Ataxia Consortium for Translational Studies

167 The European Friedreich's Ataxia Consortium for Translational Studies

168 Within the European Friedreich's Ataxia Consortium for Translational Studies

169 aining to activation of frataxin expression (Friedreich's ataxia) and production of active survival m

170 n = 12, age range 41-76 years, five female), Friedreich's ataxia (n = 12, age range 21-55 years, seve

171 9 was recently 'excluded' as a candidate for Friedreich's ataxia following the identification of an e

172 Frataxin deficiency, responsible for Friedreich's ataxia (FRDA), is crucial for cell survival

173 e are the principal mutation responsible for Friedreich's ataxia (FRDA).

174 AA)(n) repeat expansions are responsible for Friedreich's ataxia as well as late-onset cerebellar ata

175 The gene responsible for Friedreich's ataxia, a disease characterized by neurodeg

176 ity of borderline alleles confers a risk for Friedreich ataxia, and the range of pathogenic alleles i

177 evant to the development of therapeutics for Friedreich's ataxia.

178 modifying, and neuroprotective treatment for Friedreich's ataxia.

179 Humans with frataxin deficiency have Friedreich's ataxia, a neurodegenerative disorder charac

180 In Friedreich's ataxia (FRDA) patients, diminished frataxin

181 In Friedreich's ataxia (FRDA), expanded GAA repeats in intr

182 In Friedreich's ataxia patients there was a significant inv

183 o explain the reduction in mRNA abundance in Friedreich's ataxia based on intermolecular triplex form

184 ss disease-modifying therapeutic advances in Friedreich ataxia, highlighting the most promising candi

185 ata indicate that expanded GAA-TR alleles in Friedreich ataxia are highly mutable and have a natural

186 triggering neuro- and cardiodegeneration in Friedreich's ataxia.

187 function of this unusual DNA conformation in Friedreich's ataxia.

188 pathologic process leading to cell damage in Friedreich's ataxia.

189 tolerability, and efficacy of deferiprone in Friedreich ataxia (FRDA).

190 inant cause of transcriptional deficiency in Friedreich ataxia.

191 ability to ameliorate frataxin deficiency in Friedreich's ataxia is warranted.

192 Frataxin deficiency in Friedreich's ataxia results from transcriptional downreg

193 mitochondrial protein which is deficient in Friedreich's ataxia, a hereditary neurodegenerative dise

194 and frataxin (FXN), the protein deficient in Friedreich's ataxia.

195 f frataxin, the protein which is depleted in Friedreich ataxia.

196 , analogous to disease-causing expansions in Friedreich ataxia, including two that are in introns of

197 isms of reduced frataxin (FXN) expression in Friedreich's ataxia (FRDA) are linked to epigenetic modi

198 repeats that silence frataxin expression in Friedreich's ataxia, a terminal neurodegenerative diseas

199 incomplete shift of IRP1 to its ISC form in Friedreich ataxia (FRDA) fibroblasts, associated with de

200 e homolog of the human protein implicated in Friedreich ataxia, is involved in iron homeostasis.

201 a GAA/TTC DNA triplex has been implicated in Friedreich's ataxia.

202 We show that GAA instability in Friedreich's Ataxia is a DNA-directed mutation caused by

203 m underlying reduced frataxin mRNA levels in Friedreich Ataxia.

204 ure is the most common cause of mortality in Friedreich's ataxia (FRDA), a mitochondrial disease char

205 The genetic mutation in Friedreich ataxia (FRDA) is a hyperexpansion of the trip

206 The most common mutation in Friedreich ataxia is an expanded (GAA*TTC)n sequence, wh

207 rate that the GAA triplet repeat mutation in Friedreich ataxia is destabilized, frequently undergoing

208 tology shows marked atrophy of the nuclei in Friedreich's ataxia and spinocerebellar ataxia type 3.

209 n the other hand, revealed that pathology in Friedreich's ataxia and spinocerebellar ataxia type 3 is

210 spinocerebellar ataxia type 6, preserved in Friedreich's ataxia, and mildy reduced in spinocerebella

211 Human frataxin (fxn) is severely reduced in Friedreich ataxia (FRDA), a frequent autosomal recessive

212 ch corresponds to the expanded GAA repeat in Friedreich ataxia, as well as for ATT, CCT and GTT repea

213 repeat sequences, such as (GAA)n repeats in Friedreich's ataxia, (CTG)n repeats in myotonic dystroph

214 Defects in frataxin result in Friedreich ataxia, a genetic disease characterized by ea

215 gene causes an mRNA deficit that results in Friedreich ataxia (FRDA).

216 s in the first intron of FXN gene results in Friedreich's ataxia.

217 Epigenetic silencing in Friedreich ataxia (FRDA), induced by an expanded GAA tri

218 Thus epigenetic promoter silencing in Friedreich ataxia is reversible, and the results implica

219 cations for the design of clinical trials in Friedreich's ataxia, for which SARA might be the most su

220 differentiated somatic cells might influence Friedreich's ataxia pathogenesis.

221 h conditions that are recessively inherited, Friedreich ataxia and RFC1-associated cerebellar ataxia,

222 netically confirmed FA and baseline modified Friedreich's Ataxia Rating Scale (mFARS) scores between

223 In most Friedreich ataxia patients, a large GAA-repeat expansion

224 tures reminiscent of mitochondrial myopathy, Friedreich ataxia, and 3-hydroxy-3-methylglutaryl-CoA ly

225 First established as a diagnosis by Nikolaus Friedreich in 1863, Friedreich's ataxia (FA) is an autos

226 The DNA abnormality found in 98% of Friedreich's ataxia (FRDA) patients is the unstable hype

227 ical approach for the clinical assessment of Friedreich ataxia (FA) cardiomyopathy (FA-CM).

228 tiple tissues obtained from six autopsies of Friedreich's ataxia patients.

229 assembly process and the molecular basis of Friedreich's ataxia.

230 in the homozygous state in atypical cases of Friedreich's ataxia, such as older age of onset, preserv

231 Frataxin deficiency is the primary cause of Friedreich ataxia (FRDA), an autosomal recessive cardiod

232 can lead to gene inactivation, the cause of Friedreich's ataxia disease in humans.

233 n a parent does not exclude the diagnosis of Friedreich's ataxia in the offspring, and tests for the

234 ty-six patients with a clinical diagnosis of Friedreich's ataxia were investigated for the GAA trinuc

235 eat tracts are involved in the etiologies of Friedreich ataxia, fragile X syndrome, and myotonic dyst

236 ra-mitochondrial frataxin in the etiology of Friedreich's ataxia, also have important implications fo

237 tract as possibly related to the etiology of Friedreich's ataxia.

238 ataxin protein as related to the etiology of Friedreich's ataxia.

239 ansion, its role in the GAA.TTC expansion of Friedreich ataxia (FRDA) is less clear.

240 )n found in intron 1 of the frataxin gene of Friedreich's ataxia patients.

241 egistry investigating the natural history of Friedreich's ataxia.

242 The vast majority of Friedreich ataxia patients are homozygous for large GAA

243 to influence the clinical manifestations of Friedreich's ataxia (FRDA), an autosomal recessive neuro

244 s, consistent with a multi-step mechanism of Friedreich's ataxia pathophysiology, and suggesting alte

245 n healthy volunteers and in a mouse model of Friedreich ataxia versus wild-type mice (~50% reduction

246 ataxin overexpression in a cellular model of Friedreich's ataxia, a neurodegenerative disease caused

247 factor (SCF) in a humanized murine model of Friedreich's ataxia.

248 However, studies in models of Friedreich ataxia, a neurodegenerative and cardiodegener

249 re tightly associated with the occurrence of Friedreich's ataxia.

250 function have a role in the pathogenesis of Friedreich's ataxia, Wilson's disease and hereditary spa

251 s thought to underlie the pathophysiology of Friedreich ataxia and may occur at the expense of cytoso

252 tically contribute to the pathophysiology of Friedreich ataxia.

253 ensurate with the observed low prevalence of Friedreich ataxia in Mestizos.

254 The onset and progress of Friedreich's ataxia (FRDA) is associated with the geneti

255 g1/MTP1, Sfxn1 and DCYTB: Ongoing studies of Friedreich's ataxia, sideroblastic anemia, aceruloplasmi

256 The common clinical symptoms of Friedreich's ataxia (FRDA) include ataxia, muscle weakne

257 sease-associated repeats except for those of Friedreich's ataxia.

258 iquinone showing promise in the treatment of Friedreich's Ataxia, reacts at the flavin site.

259 or the design of upcoming clinical trials of Friedreich's ataxia.

260 modulate the effects of oxidative stress on Friedreich's ataxia patients and, more in general, on ot

261 for causing the neurodegenerative pathology Friedreich's ataxia.

262 3) (prevalence, 3.1 per 100,000 population), Friedreich ataxia (prevalence, 1.0 per 100,000 populatio

263 xia and peripheral neuropathy that resembles Friedreich's ataxia.

264 eurodegenerative diseases (i.e. Alzheimer's, Friedreich's ataxia and Parkinson's diseases).

265 ion, including expansions analogous to short Friedreich ataxia mutations.

266 tutes a new and useful model system to study Friedreich ataxia.

267 ng those associated with fragile X syndrome, Friedreich's ataxia, and Huntington's disease, and corre

268 This indicates that Friedreich ataxia in Mexican Mestizos is due to genetic

269 The Friedreich's ataxia cohort was subdivided into three gro

270 Disease severity was assessed by the Friedreich's Ataxia Rating Scale (FARS).

271 To characterize the Friedreich's ataxia intermediates, we generated massive

272 Our discovery that plasmids containing the Friedreich's ataxia (FRDA) expanded GAA.TTC sequence, wh

273 This region contains the Friedreich's Ataxia gene, raising the possibility that H

274 The effects were most pronounced for the Friedreich ataxia and the fragile X triplet repeat seque

275 the formation of non-B-DNA structures in the Friedreich ataxia-associated (GAA)n*(TTC)n repeats from

276 activities of daily living (ADL) part of the Friedreich's Ataxia Rating Scale and EQ-5D.

277 ISCU interacts with the Friedreich ataxia gene product frataxin in iron-sulfur c

278 xpression also inversely correlated with the Friedreich Ataxia Rating Scale score, an indicator of di

279 ession of the Frataxin gene (FXN) leading to Friedreich's ataxia (FRDA).

280 as elevated over 1.35-fold (P < 0.05) in two Friedreich's mouse models and Friedreich's lymphocytes.

281 The commonest genetic ataxias were Friedreich's ataxia (22%), SCA6 (14%), EA2 (13%), SPG7 (

282 nded GAA*TTC repeat sequence associated with Friedreich's ataxia (FRDA) adopts non-B DNA structures,

283 al, and behavioural deficits associated with Friedreich's ataxia.

284 ters but whose mutations are associated with Friedreich's ataxia.

285 In families with Friedreich's Ataxia, the only recessive trinucleotide di

286 Most individuals with Friedreich ataxia (FRDA) are homozygous for an expanded

287 ugust 2023, we assessed 169 individuals with Friedreich ataxia and 95 controls.

288 Compared to controls, individuals with Friedreich ataxia had lower volume of dentate nucleus an

289 emale patients (aged 18 years or older) with Friedreich's ataxia were given single doses (phase 1) an

290 [(18)F]BCPP-EF binding in participants with Friedreich ataxia was lower than that in healthy volunte

291 oncentrations are increased in patients with Friedreich ataxia, which supports the hypothesis that it

292 There were 383 patients with Friedreich's ataxia (FRDA), 205 patients with SCA and 16

293 characterize the myocardium in patients with Friedreich's ataxia (FRDA), and the relationship between

294 hy of the cerebellar nuclei in patients with Friedreich's ataxia and spinocerebellar ataxia type 3.

295 Patients with Friedreich's ataxia as well as those with intrinsic cere

296 Nov 21, 2013, we enrolled 605 patients with Friedreich's ataxia.

297 y of high-dose nicotinamide in patients with Friedreich's ataxia.

298 ression and treatment effects in people with Friedreich ataxia.

299 l deficit and frataxin levels in people with Friedreich ataxia.

300 rs in Htt.Q111 Huntington's disease and YG8s Friedreich's ataxia mice resulted in efficient editing i