ライフサイエンスコーパス: thalassemia

1 eta(0)-thalassemia, and hemoglobin S/beta(+)-thalassemia).

2 n those mice does not improve anemia of beta-thalassemia.

3 es to iron overload in a mouse model of beta-thalassemia.

4 patients with hereditary hemochromatosis or thalassemia.

5 ctor suppressing hepcidin production in beta-thalassemia.

6 hemoglobin (Hb) S heterozygotes, and alpha(+)thalassemia.

7 are a frequent finding in patients with beta-thalassemia.

8 tis) for the removal of cardiac iron in beta-thalassemia.

9 nthesis to treat sickle cell anemia and beta thalassemia.

10 e of an autocrine amplification loop in beta-thalassemia.

11 ythroblasts and sera from subjects with beta-thalassemia.

12 cal severity of sickle cell disease and beta-thalassemia.

13 d transfusion independence for patients with thalassemia.

14 odifier of hemoglobin disorders such as beta-thalassemia.

15 and ineffective erythropoiesis, such as beta-thalassemia.

16 such as hereditary hemochromatosis and beta-thalassemia.

17 and patients with polycythemia vera or beta-thalassemia.

18 ss iron accumulation in mouse models of beta-thalassemia.

19 ron overload in mice with nontransfused beta-thalassemia.

20 sts provides a robust ex vivo model for beta-thalassemia.

21 n of renal dysfunction in patients with beta-thalassemia.

22 ties in GFR are common in patients with beta-thalassemia.

23 tivity contributes to the pathophysiology of thalassemia.

24 mpounds as pharmaceutical therapies for beta-thalassemia.

25 erythropoiesis and its dysregulation in beta-thalassemia.

26 ication of iron-loading anemias such as beta-thalassemia.

27 of hemoglobin H disease, a subtype of alpha-thalassemia.

28 ategy for sickle cell disease (SCD) and beta-thalassemia.

29 tations of both sickle cell disease and beta-thalassemia.

30 n patients with sickle cell disease and beta-thalassemia.

31 on, and utilization in diseases such as beta-thalassemia.

32 kle S trait associated to heterozygous alpha thalassemia.

33 ing and in distress, and he had a history of thalassemia.

34 n imbalance in cells from patients with beta-thalassemia.

35 CS-R2 alpha-globin enhancer and causes alpha-thalassemia.

36 es are a frequent finding in patients with b-thalassemia.

37 including sickle cell disease (SCD) and beta-thalassemia.

38 strategy for personalized treatment of beta-thalassemia.

39 in people with sickle cell disease and beta-thalassemia.

40 semia-free survival of class 3 patients with thalassemia.

41 disorders, including sickle cell disease and thalassemia.

42 lpha2beta2) production, the hallmark of beta-thalassemia.

43 all patients born each year with severe beta-thalassemia.

44 the severity of sickle cell disease and beta-thalassemia.

45 eases, including anemia, erythrocytosis, and thalassemias.

46 c potential for sickle cell disease and beta-thalassemias.

47 the anemia and ineffective erythropoiesis in thalassemias.

48 genes contributing to hemoglobinopathies and thalassemias.

49 ty of sickle cell disease (SCD) and the beta-thalassemias.

50 e is still no universally available cure for thalassemias.

51 malaria risk (HbAS polymorphism, 6.3%; alpha-thalassemia, 0.3%; ABO group, 0.3%; and glucose-6-phosph

52 hemoglobin SS (58.7%), 14 SC (30.4%), 4 beta-thalassemia (8.7%), and 1 sickle trait (2.2%).

53 l trait (10/218 [4.6%]) and homozygous alpha+thalassemia (8/216 [3.7%]) were significantly lower amon

54 stigated how these pathways are used in beta-thalassemia, a common hemoglobinopathy in which beta-glo

55 enotypic diversity of hemoglobin (Hb) E beta thalassemia, a patient was encountered with persistently

56 together with varying frequencies of alpha(+)thalassemia across Africa may explain the inconsistent r

57 ed with increased stroke risk, whereas alpha-thalassemia and a SNP in the ADCY9 gene were linked with

58 activated early in the pathogenesis of beta-thalassemia and are essential for excess iron accumulati

59 ncreased erythropoiesis but is pathologic in thalassemia and hemochromatosis.

60 ding to hemoglobin variants and all types of thalassemia and hemoglobinopathies.

61 beta-Thalassemia and HFE-related hemochromatosis are 2 of the

62 variation related to hemoglobinopathies and thalassemia and implemented microattribution to encourag

63 sease variation and pathogenesis in HbE beta-thalassemia and indicates that the epidemiology of beta-

64 itical for progressive iron overload in beta-thalassemia and may be a novel therapeutic target in sev

65 In beta-thalassemia and polycythemia vera (PV), disordered eryth

66 as future therapeutics for untransfused beta-thalassemia and PV.

67 In patients with thalassemia and related anemias, GDF15 expression may co

68 tion and when there is a variation for alpha-thalassemia and S, two unlinked, but epistatic variants.

69 severity of hemoglobinopathies such as beta-thalassemia and sickle cell anemia.

70 Then, we used beta-thalassemia and sickle cell disease mice as models of he

71 dvances include the hemoglobinopathies (beta-thalassemia and sickle cell disease); rare genetic disor

72 s give rise to the beta-globinopathies, beta-thalassemia and sickle cell disease, which begin to mani

73 jor disorders of adult beta-hemoglobin: beta-thalassemia and sickle cell disease.

74 bF inducers to be used in patients with beta-thalassemia and sickle cell disease.

75 The mechanisms by which alpha-thalassemia and sickle cell traits confer protection fro

76 with co-inheritance of heterozygous alpha + -thalassemia and sickle trait.

77 case for its further study in other forms of thalassemia and sickle-cell anemia, particularly when sp

78 that preventing iron overload improves beta-thalassemia and strengthens the essential role of Tmprss

79 ns and 27 ethnic groups for alpha-, and beta-thalassemias and additional querying options in the HbVa

80 mutations responsible for 6 anemias and beta-thalassemias and additional substitutions without clinic

81 e-cell hemoglobin C disease, and sickle-cell thalassemia) and age-similar, race-matched controls unde

82 c conditions, including sickle cell disease, thalassemia, and G6PD deficiency, erythrocyte lifespan i

83 pression by erythropoietin, iron deficiency, thalassemia, and hemochromatosis.

84 lobin S (hemoglobin SC, hemoglobin S/beta(0)-thalassemia, and hemoglobin S/beta(+)-thalassemia).

85 ) of patients with sickle cell disease, beta-thalassemia, and hemophilia A/B or von Willebrand diseas

86 lume regulation include sickle cell disease, thalassemia, and hereditary spherocytosis, in which dehy

87 ameliorating clinically severe forms of beta-thalassemia, and in particular, the very common subgroup

88 r diseases such as hereditary spherocytosis, thalassemia, and malaria.

89 al in individuals with hemochromatosis, beta-thalassemia, and related disorders.

90 Morbidity and mortality in thalassemia are associated with iron burden.

91 Sickle cell disease and beta-thalassemia are common genetic disorders caused by mutat

92 mortality associated with this biomarker in thalassemia are poorly defined.

93 Thalassemias are a heterogeneous group of red blood cell

94 tic diseases, such as sickle cell anemia and thalassemia, are characterized by enhanced release of he

95 ), mainly sickle cell disease (SCD) and beta-thalassemia, become symptomatic postnatally as fetal gam

96 beta-Thalassemia (beta-Thal) is a group of life-threatening b

97 In beta-thalassemia, beta-globin synthesis is reduced causing al

98 oach for translation into a therapy for beta-thalassemia.beta-thalassemia is characterised by the pre

99 beta-thalassemia (betaT) is a genetic blood disorder causing

100 rticipants with SCA (HbSS or HbSbeta degrees thalassemia) between the ages of 5 and 15 years with no

101 on survey of Sri Lankan schoolchildren, beta-thalassemia (but not HbE) trait was associated with incr

102 pathies such as sickle cell disease and beta-thalassemia, but current gamma-globin-inducing drugs off

103 e response in asplenic individuals with beta-thalassemia, but previous PPSV23s affect the memory B-ce

104 artificially engineered model for human beta thalassemia by knocking down beta-globin gene and protei

105 traps may have therapeutic relevance in beta-thalassemia by suppressing the deleterious effects of GD

106 ealthy volunteers, and in patients with beta-thalassemia, by expanding late-stage erythroblasts throu

107 al progression of polycythemia vera and beta-thalassemia, by modulating erythroid proliferation and d

108 The effect of thalassemia carriage on hepcidin is also unknown, but it

109 screened by Doppler echocardiography in the Thalassemia Clinical Research Network.

110 We found a new (deltabeta)(0)-thalassemia deletion and a rare hereditary persistence o

111 We mapped a Kurdish beta(0)-thalassemia deletion, which retains the required interge

112 Presence of alpha-thalassemia deletions was associated with fewer DRBCs.

113 on of HbF to ameliorate sickle cell and beta-thalassemia disease severity.

114 with transfusion dependent hemoglobin E/beta-thalassemia disease was treated with hydroxyurea to indu

115 SCD, particularly hemoglobin SS and Sbeta(0)-thalassemia disease, is ethically sound.

116 ronic graft-vs-host disease; and sickle cell-thalassemia disease-free survival, immunologic recovery,

117 uction in patients with sickle cell and beta-thalassemia diseases because of its good efficacy and sa

118 alpha-thalassemia is co-inherited with beta-thalassemia, excess free alpha-globin chains are reduced

119 ics and current therapeutic standard in beta-thalassemia, explore the definition of ineffective eryth

120 Therefore, beta-thalassemia fits into the broader framework of protein-a

121 -72), the 5-year probability of survival and thalassemia-free survival are 93% and 84%, respectively.

122 ailure/rejection and significantly increased thalassemia-free survival of class 3 patients with thala

123 1%) in MSD groups (P = .15), with respective thalassemia-free survival probabilities of 94% (95% CI,

124 The respective 5-year thalassemia-free survival rates were 73% (95% CI, 51-86%

125 otide polymorphisms (SNPs), as well as alpha-thalassemia, G6PD A(-) variant deficiency, and beta-glob

126 ions, thus representing an optimal graft for thalassemia gene therapy.

127 of normal, heterozygous, and homozygous beta thalassemia genetic disorders.

128 More than 100 varieties of alpha-thalassemia have been identified.

129 on in which patients with and models of beta-thalassemia have provided significant insight.

130 with both nontransfused and transfused beta-thalassemia have very high serum ERFE levels, which decr

131 more, ASO treatment of mice affected by beta-thalassemia (HBB(th3/+) mice, referred to hereafter as t

132 We hypothesize that in beta-thalassemia heme oxygenase (HO) 1 could play a pathogeni

133 ocyte hydration include sickle cell disease, thalassemia, hemoglobin CC, and hereditary spherocytosis

134 ing altered hematocrit, sickle cell disease, thalassemia, hemolytic anemias, and malaria, with both a

135 e highly associated with HbF in Chinese beta-thalassemia heterozygotes.

136 history of hemophilia, sickle cell anemia or thalassemia, history of blood transfusion, cocaine and o

137 Hb S heterozygotes and alpha(+)thalassemia homozygotes were protected from severe malar

138 5% CI, 0.32-0.73, respectively), but alpha(+)thalassemia in combination with Hp2-2 was not protective

139 interaction between Hp genotype and alpha(+)thalassemia in predicting risk of severe malaria: Hp2-1

140 f induced anemia, polycythemia vera and beta-thalassemia in which macrophages were chemically deplete

141 on of Hri during heme deficiency and in beta-thalassemia increases eIF2alpha phosphorylation and inhi

142 exhibit a mild chronic anemia, and HbE/beta-thalassemia individuals show a range of clinical manifes

143 Here we used a murine model of beta-thalassemia intermedia (Hbb(th1/th1) mice) to investigat

144 atment of mouse models of HH (Hfe(-/-)) and -thalassemia intermedia (Hbb(th3/+)) with Tmprss6 siRNA f

145 ts with beta-thalassemia major (TM) and beta-thalassemia intermedia (TI) were consecutively recruited

146 emoglobin (HbF) levels and morbidity in beta-thalassemia intermedia (TI), we analyzed data from 63 un

147 ad in untransfused patients affected by beta-thalassemia intermedia and Hamp modulation provides impr

148 Individuals with beta-thalassemia intermedia and hemoglobinopathies of equival

149 ot cover other hemoglobinopathies, including thalassemia intermedia and sickle cell anemia, in which

150 exacerbates the phenotypic severity of beta-thalassemia intermedia in mice.

151 rload in hereditary hemochromatosis and beta-thalassemia intermedia is caused by hepcidin deficiency.

152 In contrast, in anemic beta-thalassemia intermedia mice, there is altered progressio

153 pothesis, we exploited a mouse model of beta-thalassemia intermedia, Th3/(+) We observed that HO inhi

154 is greatly increased in Hbb(th3/+) mice with thalassemia intermedia, where it contributes to the supp

155 In beta-thalassemia intermedia, which does not require blood tra

156 mited iron overload in a mouse model of beta-thalassemia intermedia.

157 modifying disease-associated morbidities of -thalassemia intermedia.

158 of Hbb(Th3/+) mice (Th3/+), a mouse model of thalassemia intermedia.

159 may transform severe, transfusion-dependent thalassemia into relatively mild forms of anemia.

160 -thalassemia is a congenital anemia caused by partial or

161 beta-thalassemia is a disease characterized by anemia and is

162 Beta-thalassemia is a severe genetic blood disorder caused by

163 In developed countries, treatment of thalassemia is also still far from ideal, requiring life

164 beta-Thalassemia is associated with several abnormalities of

165 nsplantation (BMT) for class 3 patients with thalassemia is challenging due to high rates of graft re

166 ion into a therapy for beta-thalassemia.beta-thalassemia is characterised by the presence of an exces

167 beta-Thalassemia is characterized by ineffective erythropoies

168 When alpha-thalassemia is co-inherited with beta-thalassemia, exces

169 alis syndrome (BHFS) resulting from alpha(0)-thalassemia is considered a universally fatal disorder.

170 Clinical heterogeneity in HbE beta-thalassemia is incompletely explained by genotype, and t

171 donor hematopoietic cell transplantation in thalassemia is not well established.

172 beta-Thalassemia is one of the most common inherited anemias,

173 iology of ineffective erythropoiesis in beta-thalassemia is poorly understood.

174 Hemoglobin E (HbE) beta-thalassemia is the most common severe thalassemia syndro

175 chanism of increased iron absorption in beta-thalassemia is unclear.

176 transfusional iron-overload diseases, e.g., thalassemia, is overviewed.

177 roval and informed consent, 30 patients with thalassemia major (mean age +/- standard deviation, 34.6

178 Patients with thalassemia major (Thal) frequently have low plasma zinc

179 A total of 255 patients with beta-thalassemia major (TM) and beta-thalassemia intermedia (

180 PAH) remains a concern in patients with beta-thalassemia major (TM) and intermedia (TI); however, stu

181 Patients with beta-thalassemia major (TM) and other refractory anemias requ

182 e analyzed the outcomes of 485 patients with thalassemia major (TM) or sickle cell disease (SCD) rece

183 -induced compromised fertility in women with thalassemia major (TM) was evaluated in 26 adult TM fema

184 L)-6, and IL-8 in biofluids of patients with thalassemia major (TM) with or without gingivitis.

185 and treatment of cardiac dysfunction in beta-thalassemia major (TM).

186 till a major cause of death in patients with thalassemia major (TM).

187 31 chronically transfused patients with beta-thalassemia major and collected samples immediately befo

188 Conclusion ECV is significantly increased in thalassemia major and is associated with myocardial iron

189 g cardiac magnetic resonance (MR) imaging in thalassemia major and to investigate the relationship be

190 ion factor-15 (GDF-15) in patients with beta-thalassemia major before and after transfusion, in the c

191 beta-Thalassemia major causes ineffective erythropoiesis and

192 A 34-year-old man with thalassemia major complained of nyctalopia and decreased

193 Hepcidin levels in patients with beta-thalassemia major dynamically reflect competing influenc

194 al incidence of melioidosis in children with thalassemia major from 2001 to 2010 was 140 per 100 000/

195 transplanted more than 20 years ago for beta-thalassemia major had a different health-related quality

196 We report here the correction of alpha-thalassemia major hydrops fetalis in transgene-free iPS

197 total of 197 consecutive patients with beta-thalassemia major or intermedia with at least 10 years o

198 We studied 107 pediatric thalassemia major patients (61 boys, median age 14.4 yea

199 eek) for myocardial iron removal in 197 beta-thalassemia major patients with myocardial siderosis (T2

200 (CMR) plays a key role in the management of thalassemia major patients, but few data are available i

201 tiparametric CMR approach can occur early in thalassemia major patients.

202 ction, and fibrosis in a cohort of pediatric thalassemia major patients.

203 Individuals with beta-thalassemia major require regular lifelong Red Blood Cel

204 Thalassemia major was a major risk factor for melioidosi

205 ron chelation therapy in 2010, no child with thalassemia major was diagnosed with melioidosis in 2011

206 We sought to determine whether, in beta-thalassemia major, transfusion-mediated inhibition of er

207 n several of these disorders, including beta-thalassemia major, which is characterized by a defective

208 re reviewed including 11 (41%) children with thalassemia major.

209 progenitor cells (HPCs) in adults with beta-thalassemia major.

210 ents in the management of patients with beta-thalassemia major.

211 s between basic and clinical studies of beta-thalassemia major.

212 etic resonance imaging and oral chelation in thalassemia management has likely contributed to improve

213 ith ineffective erythropoiesis, such as beta-thalassemia, manifest inappropriately low hepcidin produ

214 Ocular findings in beta-thalassemia may correlate to the disease itself, iron ov

215 Beta-thalassemia may present with various signs, both structu

216 s in 327 subjects with transfusion-dependent thalassemia (mean entry age, 22.1 +/- 2.5 years) from 20

217 ever, Sp100A cannot overcome Daxx- and alpha-thalassemia mental retardation, X-linked (ATRX)-mediated

218 characterize the Arabidopsis thaliana Alpha Thalassemia-mental Retardation X-linked (ATRX) ortholog

219 ting, death-domain-associated protein, alpha thalassemia/mental retardation syndrome X linked, switch

220 Alpha thalassemia/mental retardation syndrome X-linked (ATRX)

221 tions of the telomere binding proteins alpha thalassemia/mental retardation syndrome X-linked (ATRX)

222 ere, we found that the ND10 component, alpha-thalassemia/mental retardation syndrome X-linked (ATRX)

223 h-domain-associated protein) and ATRX (alpha thalassemia/mental retardation syndrome X-linked).

224 which serve as a model of untransfused beta-thalassemia, minihepcidin ameliorates ineffective erythr

225 In older mice with untransfused beta-thalassemia, minihepcidin improves erythropoiesis and do

226 We used the Hbb(th3/+) beta-thalassemia mouse and hemoglobin E (HbE)/beta-thalassemi

227 we identify the SWI/SNF helicase ATRX (alpha-thalassemia/MR, X-linked) as a novel macroH2A-interactin

228 Here, we reported that correction of beta-thalassemia mutations in patient-specific iPSCs using th

229 that CRISPR/Cas9 successfully corrects beta-thalassemia mutations in patient-specific iPSCs.

230 vital process in pathologies, including beta-thalassemia, myelodysplastic syndrome, and viral infecti

231 Nontransfusion-dependent thalassemia (NTDT) patients may develop iron overload an

232 Beta-thalassemia ocular manifestations include ocular surface

233 beta-thalassemia, one of the most common genetic diseases wor

234 s with HCV infection and sickle cell anemia, thalassemia, or hemophilia A/B or von Willebrand disease

235 Co-inheritance of HPFH with beta-thalassemia- or SCD-associated gene mutations alleviates

236 The underlying basis of thalassemia pathology is the premature apoptotic destruc

237 We further assessed whether thalassemia patient CD34(+) HPCs could be transduced wit

238 e procedure for beta-globin gene transfer in thalassemia patient CD34(+) HPCs, which we will implemen

239 of HbF have been studied in the past in beta-thalassemia patient populations, with limited success in

240 gn of future studies of HbF inducers in beta-thalassemia patient populations.

241 r cross-sectional study of 1309 Italian beta-thalassemia patients (mean age 36.4+/-9.3 years; 46% men

242 Sixty thalassemia patients (median age, 7 years; range, 1-37)

243 The prevalence of PAH in beta-thalassemia patients as confirmed on right heart cathete

244 ced pluripotent stem cells (iPSCs) from beta-thalassemia patients could offer an approach to cure thi

245 paration proved to be safe and effective for thalassemia patients given allogeneic HSCT.

246 halassemia mouse and hemoglobin E (HbE)/beta-thalassemia patients to investigate dysregulated neutrop

247 sed a new treatment protocol (Pc 26.1) in 16 thalassemia patients to perform BMT using phenotypically

248 that the Pc 26.1 preparative regimen allows thalassemia patients to safely undergo BMT from RDs who

249 r HSCT showed that the long-term HRQoL of ex-thalassemia patients was very similar to that of the gen

250 nsplant (FACT-BMT) were received from 109 ex-thalassemia patients who underwent hematopoietic stem ce

251 noted in one-third of transfusion-dependent thalassemia patients with a documented value and develop

252 into defective neutrophil maturation in beta-thalassemia patients, which contributes to deficiencies

253 plenectomized and nonsplenectomized HbE/beta-thalassemia patients.

254 defective immune functions observed in beta-thalassemia patients.

255 bution as a contributing factor to the alpha-thalassemia phenotype of ATRX syndrome.

256 between 5 and 16.7 years of age with class 3 thalassemia received HLA-matched sibling BMT following e

257 21 years old with hemoglobin SS or Sbeta(0) thalassemia requiring hospitalization for pain were elig

258 beta-thalassemia results from point mutations or small deleti

259 d ineffective erythropoiesis, for example in thalassemia, results in sustained elevations in iron abs

260 nts receiving conventional treatment of beta-thalassemia revealed poorer outcomes compared with the c

261 in C (SC) profile, 1 was sickle cell-beta(+) thalassemia (S beta(+)-thal), and 4 were sickle cell tra

262 a [Sbeta(0)], 495 SC, and 161 sickle beta(+)-thalassemia [Sbeta(+)]), aged 3 years old and over, were

263 07 SCD patients (1751 SS or sickle beta-zero-thalassemia [Sbeta(0)], 495 SC, and 161 sickle beta(+)-t

264 od transplants (BMT and CBT) for severe beta thalassemia (SBT) and sickle cell disease (SCD) as exper

265 diagnosis, and management suggest that alpha-thalassemias should have a higher priority on global pub

266 follow-up study of 45 patients with HbE beta thalassemia showed that methemoglobin levels were signif

267 is father is a carrier of heterozygous alpha-thalassemia status that it was unknown before.

268 onmental (eg, fetal hemoglobin levels, alpha-thalassemia status) factors are known to modify SCD seve

269 Evidence from beta-thalassemia suggests that regulation of hepcidin by eryt

270 ) beta-thalassemia is the most common severe thalassemia syndrome across Asia, and millions of people

271 ligand trap as a novel therapeutic agent for thalassemia syndrome and other red cell disorders charac

272 HCS should be recognized as a distinct thalassemia syndrome with a high risk of life-threatenin

273 Sickle cell disease (SCD) and thalassemias (Thal) are common congenital disorders, whi

274 icantly more prevalent in patients with beta-thalassemia than previously recognized and correlate wit

275 subgroup of patients with hemoglobin E beta-thalassemia that makes up approximately half of all pati

276 th opioid use, HIV and transfusion-dependent thalassemia, the risk of fracture in these populations i

277 isorders such as sickle cell anemia and beta-thalassemia through activation of the fetal gamma-globin

278 sive review of observational studies on beta-thalassemia, to determine the prevalence and spectrum of

279 ss, including a hypoxic environment and beta-thalassemia, to identify two markedly different response

280 The thalassemias, together with sickle cell anemia and its v

281 In Southeast Asia, Thalassemia trait (TT) and iron deficiency anemia (IDA)

282 is valuable in the differential diagnosis of thalassemia trait and iron deficiency, and in monitoring

283 We also found that: (i) beta-thalassemia trait carriers displayed lower TC and were p

284 progression indicates that the lower IMF in thalassemia trait erythrocytes limits parasite density a

285 Reduction of IMFs in thalassemia trait erythrocytes was confirmed using clini

286 in control, HbAS, HbSS, and alpha- and beta-thalassemia trait erythrocytes, respectively.

287 s with erythrocyte indices and is reduced in thalassemia trait erythrocytes.

288 and indicates that the epidemiology of beta-thalassemia trait requires consideration when planning p

289 2, a marker used for the diagnosis of a beta-thalassemia trait.

290 h advances in the care of patients with beta-thalassemia translate into better patient survival, this

291 ssment of Exjade in Nontransfusion-Dependent Thalassemia) trial assessed the efficacy and safety of d

292 In beta-thalassemia, unequal production of alpha- and beta-globi

293 in a recent LV-based clinical trial for beta-thalassemia, vector integration within the HMGA2 gene in

294 ith a double-heterozygous sickle-cell beta-0 thalassemia was admitted to the internal ward for acute

295 tion with heterozygous or homozygous alpha(+)thalassemia was associated with protection from severe m

296 of ineffective erythropoiesis in humans with thalassemia, was significantly increased in the culture

297 Using a mouse model for dominant beta-thalassemia, we developed disease allele-free PG ES cell

298 glucose-6-phosphate dehydrogenase, and alpha-thalassemia) were the only ones to be associated with al

299 s with sickle cell phenotype with or without thalassemia who underwent nonmyeloablative allogeneic HS

300 n 62 of 69 Sri Lankan patients with HbE beta-thalassemia with moderate or severe phenotype, hepcidin