ライフサイエンスコーパス: PTC

1 PTC annotates PubMed (29 million abstracts) and the PMC

2 PTC is synchronized with PubMed and PubMed Central, with

3 PTC suppression during translation offers a promising ap

4 PTC suppression is mediated by the base pairing of a nea

5 PTC suppression therapy utilizes small molecules that su

6 PTC-to-ATC transformation was also observed in primary B

7 PTCs are a frequent cause of human genetic diseases, and

8 in Wilms tumors and has been identified in 2 PTCs.

9 ed rep1 protein by 23.1% (PTC124) and 17.2% (PTC-414) and restored biochemical function as confirmed

10 r than 2.5 layers assessed in a sample of 25 PTCs around 1 year after transplantation is indicative o

11 on assays (98 +/- 2% [PTC124] and 68 +/- 5% [PTC-414]).

12 In all 1,704 PTCs, microPTC (mPTC) with maximum diameter less than or

13 identity of the amino acid(s) inserted at a PTC during this process in mammalian cells, or how the s

14 s that suppress translation termination at a PTC to restore synthesis of a full-length protein.

15 rmits pairing of a near-cognate complex at a PTC.

16 gene or introducing a transgene containing a PTC to trigger a GCR.

17 with genetic disorders by either creating a PTC in the mutated gene or introducing a transgene conta

18 d causes alternative splicing to introduce a PTC.

19 e GCR is dependent on both the presence of a PTC and the nucleotide sequence of the transgene mRNA, w

20 processing lead to selective retention of a PTC-containing intron in EIF2B5.

21 ansition from NMD complexes that recognize a PTC to those that promote mRNA decay.

22 ring of a near-cognate aminoacyl-tRNA with a PTC and subsequently, the amino acid becomes incorporate

23 do-mRNAs (alternatively spliced mRNAs with a PTC) into protein encoding molecules, or induce exon ski

24 d that betaAR activity during, but not after PTC training initiates the activation of two plasticity-

25 ome the dominant form taking up 56.5% of all PTCs in 2013 while only 43.1% in 2008.

26 ty of ataluren (PTC124) and its novel analog PTC-414: (1) the chm(ru848) zebrafish, the only nonsense

27 medullary thyroid carcinoma (9/12, 75%) and PTC (14/30, 47%).

28 ionship between patient age at diagnosis and PTC-specific mortality with respect to BRAF status in 2,

29 ntiation characteristics of Thyroid MSCs and PTC MSCs were comparable with bone marrow MSCs.

30 0, R01-NS062869), Ionis Pharmaceuticals, and PTC Therapeutics.

31 comprehensive study on the use of PTC124 and PTC-414 as successful nonsense suppression agents for th

32 chr5:1,295,228C>T and chr5:1,295,250C>T) and PTC-specific mortality in 1051 patients (764 women and 2

33 ation, for a mean number of BM layers around PTC and in serial biopsies.

34 On ART, PTCs had significantly lower levels of residual plasma v

35 )-infected rhesus macaques (RMs), defined as PTCs, who started ART 8 weeks postinfection, continued A

36 Greater improvement at PTC also occurred for grass allergen peptide 8x6Q2W vers

37 nt improvement from baseline in mean TRSS at PTC was observed in the group receiving one 6-nmol intra

38 Adolescents treated at PTCs were more likely to be injured by a blunt than pene

39 adolescents was lower among those treated at PTCs, compared with those treated at ATCs and MTCs.

40 diate the insertion of near-cognate tRNAs at PTCs.

41 We report here that besides PTC events, translation kinetics depend on steric constr

42 still unclear how NMD discriminates between PTCs and normal stop codons.

43 ing a small molecule inhibitor against BMI1, PTC-028, we were able to demonstrate complete ablation o

44 ved in the NMD mechanism which degrades both PTC-bearing mutant transcripts and also many physiologic

45 PTC-containing mRNAs, increased eIF4E-bound PTC-containing mRNA levels, and subsequent eIF4E-depende

46 In conclusion, BVE-PTC progression could be contained via p53-dependent OIS

47 In contrast, BVE-PTC transplants continued to grow when transplanted into

48 nsformation was also observed in primary BVE-PTC tumors.

49 progression of Braf(V600E)-induced PTC (BVE-PTC) under normal TSH, we transplanted BVE-PTC tumors su

50 E-PTC) under normal TSH, we transplanted BVE-PTC tumors subcutaneously into nude and TPO-Braf(WT) mic

51 erns and associated patient deaths caused by PTC.

52 erns and associated patient deaths caused by PTC.

53 that some variant CFTR proteins generated by PTC suppression exhibit reduced maturation and activity,

54 d to treat multiple human diseases caused by PTCs.

55 in affords a porphyrin tubular organic cage, PTC-1(2H).

56 ssive histology of papillary thyroid cancer (PTC) impacts overall survival (OS).

57 Papillary thyroid cancer (PTC) is the most common type of endocrine malignancy.

58 s) associated with papillary thyroid cancer (PTC) risk.

59 S-like family with papillary thyroid cancer (PTC), applying a combined linkage-based and whole-genome

60 stratification of papillary thyroid cancer (PTC), but whether this is generally applicable, particul

61 af(V600E) -induced papillary thyroid cancer (PTC).

62 genic thyroids and papillary thyroid cancer (PTC).

63 ons can coexist in papillary thyroid cancer (PTC).

64 ons can coexist in papillary thyroid cancer (PTC).

65 ode negative (cN0) papillary thyroid cancer (PTC).

66 TPO-Cre) leads to papillary thyroid cancers (PTC) that rapidly progress to poorly differentiated thyr

67 roximately 40% of papillary thyroid cancers (PTC).

68 MNG, schwannomas, papillary thyroid cancers (PTCs), and Wilms tumors.

69 embrane (BM) around peritubular capillaries (PTC) can be used in a cohort of patients with de novo do

70 thelial staining in peritubular capillaries (PTCs) was substantially decreased with a de novo express

71 lar carcinoma (FTC) and papillary carcinoma (PTC) thyroid tissues.

72 Papillary thyroid carcinoma (PTC) is the most common histotype of thyroid carcinoma.

73 Papillary thyroid carcinoma (PTC) remained to be the most common type counting 86.4%

74 Papillary thyroid carcinoma (PTC), the most frequent thyroid cancer, is characterized

75 ed with risk of papillary thyroid carcinoma (PTC).

76 ns detected in papillary thyroid carcinomas (PTC).

77 Papillary thyroid carcinomas (PTCs) account for 90% of human thyroid cancer cases, whi

78 ter reagents under phase-transfer catalysis (PTC).

79 e.g. K2CO3) and/or phase transfer catalysts (PTC) (e.g. kryptofix 2.2.2) associated with fluorine-18

80 ack of complete peptidyl transferase center (PTC) active site mutational analyses to inform design.

81 turation of the peptidyl transferase center (PTC) and the nascent polypeptide exit tunnel (NPET), and

82 constitute the peptidyl transferase center (PTC) and those that connect PTC with the GTPase-associat

83 at suppress the peptidyl transferase center (PTC) catalytic activity stimulated by eukaryotic release

84 A away from the peptidyl transferase center (PTC) is functionally significant.

85 that target the peptidyl transferase center (PTC) on the large subunit of the ribosome.

86 function of the peptidyl transferase center (PTC).

87 ormation at the peptidyl transferase center (PTC).

88 hildren treated at pediatric trauma centers (PTCs) compared with those treated at adult trauma center

89 PubTator Central (PTC) provides automated annotations from state-of-the-ar

90 the mechanism of peptidyltransferase centre (PTC) completion that explains how integration of the las

91 of tetracycline-induced pseudotumor cerebri (PTC-T) and those of idiopathic intracranial hypertension

92 s 2 to 4 of a 4-day posttreatment challenge (PTC) in the EEU after the grass pollen season.

93 cores (TRSSs) after posttreatment challenge (PTC) to rye grass in an environmental exposure unit afte

94 e the association of center characteristics (PTC, ATC, or MTC) on mortality among patients aged 15 to

95 tients treated from 2004 to 2015 for classic PTC (cPTC) or AVPTCs were identified from the National C

96 cN0 PTC patients treated either with TT or TT + pCND had sim

97 Sixty patients with cN0 PTC were randomized to a total thyroidectomy (TT) or a T

98 ants comprise a premature termination codon (PTC) and are therefore potential targets of nonsense-med

99 hey introduce a premature termination codon (PTC) and prevent the formation of full-length protein.

100 stallation of a premature termination codon (PTC) from a frameshift-inducing INDEL that elicits nonse

101 NA containing a premature termination codon (PTC) in a VCS-dependent manner.

102 f a premature translation termination codon (PTC) in an atypical sequence context.

103 esirable when a premature termination codon (PTC) is found in a critical gene.

104 or at least one premature termination codon (PTC) mutation in COL7A1, and previous studies have shown

105 position of the premature termination codon (PTC) on the CFTR protein function.

106 protein with a premature termination codon (PTC) predicted to elicit degradation via nonsense-mediat

107 mRNA bearing a premature termination codon (PTC) promptly triggers a GCR that involves Upf3a and com

108 at introduces a premature termination codon (PTC) that prevents synthesis of the full-length peptide

109 usion creates a premature termination codon (PTC), that leads to a 65kDa truncated protein isoform th

110 tions contain a premature termination codon (PTC), thus progranulin haploinsufficiency has been propo

111 s and exogenous Premature Termination Codon (PTC)-containing mRNA isoforms and its effects are dose-,

112 in an in-frame premature termination codon (PTC).

113 resulting in a premature termination codon (PTC).

114 As containing a premature termination codon (PTC).

115 anscripts with premature termination codons (PTC).

116 he presence of premature termination codons (PTC).

117 uced readthrough over premature stop codons (PTCs) is a potentially attractive therapy for genetic di

118 nscripts that contain premature stop codons (PTCs) to mitigate their potentially harmful consequences

119 In-frame premature termination codons (PTCs) account for approximately 11% of all disease-assoc

120 pts containing premature termination codons (PTCs) are not always degraded efficiently and can genera

121 NAs containing premature termination codons (PTCs) are rapidly degraded through nonsense-mediated mRN

122 Premature termination codons (PTCs) are responsible for 10-15% of all inherited diseas

123 ng premature translation termination codons (PTCs) serves to protect cells from accumulating non-func

124 des mRNAs with premature termination codons (PTCs).

125 ssociated with premature termination codons (PTCs).

126 ive exons with premature termination codons (PTCs).

127 mRNAs bearing premature termination codons (PTCs).

128 ntroduction of premature termination codons (PTCs).

129 mounts of mutant proteins from NMD-competent PTC-containing constructs was not affected by inhibition

130 ontaining mRNAs expressed from NMD-competent PTC-containing constructs were as stable as their PTC-fr

131 NTs exist within a progenitor toxin complex (PTC), which protects the toxin and facilitates its absor

132 forms a specific "pre-termination complex" (PTC) with RNAP and elongation factors NusA and NusG, whi

133 During Pavlovian threat (fear) conditioning (PTC), sensory and neuromodulatory inputs converge on pos

134 nsferase center (PTC) and those that connect PTC with the GTPase-associated center and with mitoribos

135 duals, defined as posttreatment controllers (PTCs).

136 Plant tissue cultures (PTCs) provide researchers with unique materials that acc

137 eillance mechanism that detects and destroys PTC-containing transcripts, with UPF1 being the central

138 ion of Braf(V600E) (TPO-Braf(V600E)) develop PTC rapidly with high levels of serum thyroid-stimulatin

139 There are other patients who develop PTC-T, discontinue the antibiotic, and later develop IIH

140 e appear to be nonobese patients who develop PTC-T, discontinue the antibiotic, and never develop PTC

141 ur center and identified those who developed PTC-T after treatment with a tetracycline-class antibiot

142 ild-type Cyp24a1 (BVE(Cyp24a1-wt)) developed PTC at 5 weeks of age.

143 Eight of 12 different PTC alleles responded to treatment and produced full len

144 o infer therapeutic strategies for different PTC mutations at large.

145 near-cognate aminoacyl-tRNA selection during PTC suppression.

146 A PRS based on the 10 established PTC SNPs showed a stronger predictive power compared wit

147 Finally, PTCs showed immunologic benefits of viral control after

148 Kaplan-Meier analyses revealed a flat PTC-specific patient survival curve with neither mutatio

149 odulates aversive memory formation following PTC through two molecularly and temporally distinct sign

150 at betaAR activity during, but not following PTC sets in motion cascading molecular events for the ac

151 rates were significantly different (4.0% for PTC-T vs 16.5% for IIH, P <.001).

152 ates were significantly different (43.8% for PTC-T vs 79.2% for IIH, P < .001).

153 arboring neither mutation, HRs (95% CIs) for PTC-specific mortality were 3.08 (0.87-10.84) for BRAF V

154 The mean illness duration was shorter for PTC-T (18.3 weeks vs 62.9 weeks for IIH, P <.0001).

155 age cut point in current staging systems for PTC and argue for considering a revision in how we antic

156 The mean age at diagnosis was younger for PTC-T (19.8 years vs 28.1 years for IIH, P < .001).

157 oglycosides in a cell culture system on four PTCs responsible for FTD or a related neurodegenerative

158 RNA) which can effectively suppress in-frame PTCs and faithfully encode their cognate amino acid.

159 clear p27, a CDK2 inhibitor, in samples from PTC.

160 ging variability of regenerants derived from PTCs may have both genetic and epigenetic origins, and m

161 he treatment of patients with RDEB harboring PTC mutation in COL7A1.

162 The underlying mechanisms of how PTC mutations in MYBPC3 lead to the onset and progressio

163 In PTC samples from patients, upregulation of TGF-beta, p27

164 value of assessing the mean number of BM in PTC for early prediction of progression to chronic antib

165 f(V600E)gene is supposed to be the driver in PTC development, down-regulation of expression should co

166 the prognostic power of this genetic duet in PTC-specific mortality.

167 the prognostic power of this genetic duet in PTC-specific mortality.

168 1 TYPE A1, TENASCIN, and SOD3 expression in PTC MSCs compared to Thyroid MSCs, suggesting the presen

169 use of patient age as a high-risk factor in PTC and call for differentiation between patients with B

170 tance of heritable low-penetrance markers in PTC.

171 nce that Cyp24a1 functions as an oncogene in PTC, where its overexpression activates multiple signali

172 We demonstrate the full text results in PTC significantly increase biomedical concept coverage a

173 ng-observed age-associated mortality risk in PTC is dependent on BRAF status; age is a strong, contin

174 portance of rigorous control of serum TSH in PTC patients.

175 we found COL1A1 and LOX to be upregulated in PTC and expressed at highest levels in PDTC and anaplast

176 siveness of PTC, but its prognostic value in PTC-related mortality remains to be specifically establi

177 o expression of alpha-smooth muscle actin in PTCs.

178 hile it remained stable or even decreased in PTCs.

179 romoter mutations were rare and subclonal in PTCs, they were clonal and highly prevalent in advanced

180 ly used antibiotic in humans that can induce PTC readthrough and suppress nonsense mutations at high

181 igate the progression of Braf(V600E)-induced PTC (BVE-PTC) under normal TSH, we transplanted BVE-PTC

182 s of interaction with the ribosome influence PTC read-through efficiency.

183 Before ART initiation, PTCs had higher CD4 T cell counts, lower plasma viremia,

184 components of pharmaceutical gentamicin lack PTC readthrough activity but the minor component gentami

185 me-wide association studies of lipid levels (PTC=0.004, PHDL-C=0.008 and Ptriglycerides=0.00003) and

186 For most PTCs, impaired secretion/function produced by readthroug

187 iPSC-CMs carrying MYBPC3 PTC mutations displayed aberrant calcium signaling and m

188 generated from HCM patients harboring MYBPC3 PTC mutations (p.R943x; p.R1073P_Fsx4) using genome edit

189 e pathogenesis of HCM associated with MYBPC3 PTC mutations by utilizing human isogenic induced plurip

190 The new PTC web interface allows users to build full text docume

191 n rapamycin-treatment as compared to the non-PTC isoform.

192 onstructs, we show that a fraction ( 30%) of PTC-containing mRNAs expressed from NMD-competent PTC-co

193 ated to be involved in the aggressiveness of PTC, but its prognostic value in PTC-related mortality r

194 etion, which induced polysome association of PTC-containing mRNAs, increased eIF4E-bound PTC-containi

195 lack of apoptosis and metastatic behavior of PTC.

196 We identified 52 cases of PTC-T and 302 cases of IIH.

197 strate a simple 4-genotype classification of PTC, particularly CPTC, with a disease-specific mortalit

198 strate a simple 4-genotype classification of PTC, particularly CPTC, with a disease-specific mortalit

199 Rapamycin-treatment also causes depletion of PTC-containing mRNA isoforms from polyribosomes, undersc

200 by mutant SRSF2, restoring the expression of PTC-containing transcript.

201 ore, microporous supramolecular framework of PTC-1(2H) is able to promote the heterogeneous photo-oxi

202 The heritability of PTC is high compared to other cancers, but its underlyin

203 n reveals much prolonged triplet lifetime of PTC-1(2H) relative to monomer reference, illustrating th

204 age to risk stratification and management of PTC.

205 he DMD locus are affected by the presence of PTC, hinting at a possible epigenetic mechanism responsi

206 in DMD expression levels in the presence of PTC.

207 d of SMG9 do not appear to have reduction of PTC-containing transcripts but do display global transcr

208 iant conferred a significantly lower risk of PTC.

209 rovides a consistent and effective source of PTC readthrough activity to study the potential of nonse

210 Targeting of PTC-containing transcripts is mediated by the nonsense-m

211 ecurrent MB cells upon re-transplantation of PTC-028-treated cells into secondary recipient mouse bra

212 ly patients with the conventional variant of PTC were analyzed.

213 Aggressive variants of PTC (AVPTC) are associated with invasive features.

214 About 45% of PTCs exhibit activating BRAF(V600E) mutations.

215 miRNA profiling of PTCs, MNG, schwannomas, and Wilms tumors revealed a comm

216 Although the prognosis of PTCs is excellent, 5% to 10% of tumors display an unfavo

217 o induce eukaryotic ribosomal readthrough of PTCs to produce a full-length protein.

218 at in addition to stimulating readthrough of PTCs, aminoglycosides stimulate readthrough of normal te

219 ombined genetic effects of these variants on PTC risk by using summarized GWAS results to build polyg

220 treatment with either PTC124 (42 +/- 5%) or PTC-414 (36 +/- 11%), although an increase in REP1 prote

221 PTC124 or PTC-414 treatment of chm(ru848) embryos led to a approxi

222 with the remainder at MTCs (7572 [25.6%]) or PTCs (1639 [5.5%]).

223 Other PTCs were edited, but less efficiently.

224 anism in which the formation of a persistent PTC is a prerequisite for termination.

225 caregivers, investigational site personnel, PTC Therapeutics employees, and all other study personne

226 urea compounds, such as phenylthiocarbamide (PTC) and 6-n-propylthiouracil (PROP), is inherited.

227 The ability to taste phenylthiocarbamide (PTC) and 6-n-propylthiouracil (PROP) is a polymorphic tr

228 Xenopus oocytes and mice in vivo, producing PTC repair in multiple genes, including disease causing

229 venue for familial FTD caused by progranulin PTC mutations.

230 disorders, yet small molecules that promote PTC read-through have yielded mixed performance in clini

231 at aminoglycosides are able to overcome RDEB PTC mutations by inducing "read-through" and incorporati

232 C2 specifically and synergistically regulate PTC endocytosis and transport processes.

233 s expressing ACE-tRNA at levels which repair PTC indicate that there are limited interactions with tr

234 vels of intestinal CD4 T cells were similar, PTCs had higher frequencies of Th17 cells.

235 However, some PTC-containing mRNAs evade NMD, and might generate mutan

236 Several agents are known to suppress PTCs but are poorly efficacious or toxic.

237 on a continuous influx of newly synthesized PTC-containing mRNAs, indicating that truncated mutant p

238 Of 497 TCGA PTC individuals, 138 (27%) were found to carry this germ

239 We conclude that PTC-T represents a spectrum of disease in susceptible in

240 These results suggest that PTC suppression in combination with CFTR modulators may

241 Our findings suggest that PTC-containing mRNAs are potent and regulatable sources

242 The PTC-containing SRSF6 transcript exhibits a shorter half-

243 crosstalk between the NMD machinery and the PTC-bound ribosome, a central mechanistic step of RNA su

244 h" and incorporation of an amino acid at the PTC site.

245 ss depends on the rate of translation at the PTC.

246 unction complexes (EJCs) downstream from the PTC through RNA-mediated molecular interactions.

247 Thus, it is crucial to determine how the PTC variation level can be controlled.

248 We map the principal interactions in the PTC and demonstrate their critical role in termination.

249 rrect positioning of their CCA-ends into the PTC thus making peptide bond formation impossible.

250 16 P-site loop to fully accommodate into the PTC where it competes with Nmd3 for an overlapping bindi

251 ay intermediates originate downstream of the PTC and harbor 80S ribosomes that migrate into the mRNA

252 mined by both RNA sequence downstream of the PTC and the inactivating mutation within the active site

253 to investigate the positional effect of the PTC because of its well-understood structure-function re

254 In theory, the location of the PTC in a gene determines the alternative mechanisms of t

255 , we have revealed the mode of action of the PTC inhibitor madumycin II, an alanine-containing strept

256 nature of the PTC, we found that >85% of the PTC nucleotides possess mutational flexibility.

257 In eukaryotes, final assembly of the PTC occurs in the cytoplasm by insertion of the ribosoma

258 catalytically efficient organization of the PTC, highlighting the importance of proteins in the RNA-

259 Despite the highly-conserved nature of the PTC, we found that >85% of the PTC nucleotides possess m

260 o the nascent polypeptide at the site of the PTC.

261 ted to a catalytically inactive state of the PTC.

262 eral tolerance to epitopes downstream of the PTC.

263 rstanding of BoNTs and TeNT, focusing on the PTC, receptor recognition, new BoNT-like toxins, and the

264 n factors NusA and NusG, which stabilize the PTC.

265 variable and major gentamicins suppress the PTC readthrough activity of B1.

266 arget these transcripts and read-through the PTC, leading to the production of a full length function

267 ssible single nucleotide mutation within the PTC-ring, A-loop and P-loop, 180 total point mutations.

268 ontaining constructs were as stable as their PTC-free counterparts in a steady state.

269 These PTC-containing mRNAs were monosome-enriched and rarely c

270 -CFTR revealed heterogenous effects of these PTCs on CFTR function.

271 d polygenic risk score (PRS) models in three PTC study groups from Ohio (1,544 patients and 1,593 con

272 The characterization of three PTC mutations, E60X-, G542X- and W1282X-CFTR revealed he

273 Pharmacological studies of these three PTCs with various CFTR modulators suggest position-depen

274 x was tested for its ability to read-through PTC mutations in cells derived from patients with RDEB.

275 tic measure of the contrast between tissues: PTC.

276 onjunctivitis symptom score from baseline to PTC) occurred across days 2 to 4 with grass allergen pep

277 g gene FOXE1 underlies the predisposition to PTC triggered by rs965513.

278 1L4A is a mechanism in the predisposition to PTC.

279 anisms of PTMC initiation and progression to PTC, further translating into targeted tumor prevention

280 rough, namely Gln, Lys, or Tyr at UAA or UAG PTCs and Trp, Arg, or Cys at UGA PTCs.

281 UAA or UAG PTCs and Trp, Arg, or Cys at UGA PTCs.

282 osphine borane compounds was performed under PTC to obtain C60-amino acid or dipeptide derivatives in

283 minutes) from GPS1 baseline to the follow-up PTC calculated across all time points over days 2 to 4 f

284 Using PTC-containing human genomic beta-globin constructs, we

285 uet were seen when only conventional-variant PTC (CPTC) was analyzed (HR, 54.46; 95% CI, 12.26-241.82

286 ine transcarbamylase gene containing various PTC-inducing non-sense mutations is able to generate and

287 classifiers, including benign thyroid versus PTC and benign thyroid versus FTC, were built and valida

288 membrane conductance regulator (CFTR) W1282X PTC (a UGA codon) in the context of its three upstream a

289 in DMD patient and animal model muscles when PTC are present.

290 into methods that accurately predict whether PTC-containing mRNAs are degraded or not.

291 ophysiological studies of W1282X-CFTR, whose PTC is closer to the C-terminus of CFTR, suggest the pre

292 Diplopia was more common with PTC-T (40.4% vs 20.1% for IIH, P = .001).

293 opic lymph node involvement in patients with PTC is common, but the optimal management is unclear.

294 Results A total of 31,802 patients with PTC were included.

295 ull-length endogenous protein, patients with PTC-inducing non-sense mutations may still present T cel

296 ow we anticipate prognosis for patients with PTC.

297 variant at the 5q22 locus, rs73227498, with PTC predisposition.

298 tio, 6.68; 95% CI, 2.03-21.99) compared with PTCs but was not different between level I and II center

299 ficial for the treatment of CF patients with PTCs.

300 (324/469) of hemophilia B (HB) patients with PTCs.