ライフサイエンスコーパス: beta-globin

1 and inducing high-level expression of adult beta-globin.

2 n further to erythroblasts expressing normal beta-globin.

3 aused by a single amino acid substitution in beta-globin.

4 an alleles encoding normal alpha- and sickle beta-globin.

5 nine-rich pentanucleotide in a region of the beta-globin 3'untranslated region that has recently been

6 Analyses of beta-globin-3' untranslated region (UTR) fusion transcri

7 tion in patient-derived iPSCs with 2 mutated beta-globin alleles (beta(s)/beta(s)).

8 ENs that induce modification of 54% of human beta-globin alleles near the site of the sickle mutation

9 der the predicted joint changes when the two beta-globin alleles S and C are both variable in the sam

10 its target genes during erythropoiesis (e.g. beta-globin, alpha-hemoglobin stabilizing protein).

11 e show that Ppm1b superactivates EKLF at the beta-globin and BKLF promoters, dependent on intact Ppm1

12 Using the beta-globin and gamma-globin TALENs, we generated cell l

13 eens to identify regulatory elements for the beta-globin and HER2 loci in human cells.

14 ith two well-studied developmental loci: the beta-globin and HOXC cluster regions.

15 Real-time PCR assays for beta-globin and Universal 16S rRNA gene targets were per

16 er erythroblasts express predominantly adult beta-globins and the definitive erythroid-specific trans

17 mon genetic disorders caused by mutations in beta-globin, and we engineered a pair of highly active T

18 geting 191 HPV types and 12 probes targeting beta-globin as a control.

19 lian genomes, using protocadherin (Pcdh) and beta-globin as model genes.

20 Tyr-24, Tyr-42 (alpha-globin), and Tyr-130 (beta-globin) as well as nitrosation on Tyr-24 (alpha-glo

21 ol of regulatory elements (Atoh1 3' enhancer/beta-globin basal promoter) to direct expression of Clrn

22 In cells primarily transcribing beta-globin, BGL3 is not transcribed and BGL3 sequences

23 eted integration of therapeutic, full-length beta-globin cDNA to the endogenous beta-globin locus in

24 terpart, rescued cytoplasmic accumulation of beta-globin cDNA transcripts.

25 conserved CAR-E and inserted it upstream of beta-globin cDNA, which is normally retained/degraded in

26 sickle-cell disease, a point mutation in the beta-globin chain causes haemoglobin to polymerise withi

27 ase hallmarks include imbalance in the alpha/beta-globin chain ratio, ineffective erythropoiesis, chr

28 globally, characterised by reduced or absent beta-globin chain synthesis.

29 se (SCD) is caused by genetic defects in the beta-globin chain.

30 ney tissue including the abundant alpha- and beta-globin chains from hemoglobin.

31 halassemia, unequal production of alpha- and beta-globin chains in erythroid precursors causes apopto

32 by a quantitative defect in the synthesis of beta-globin chains of haemoglobin, leading to the accumu

33 ogy reflects an imbalance between alpha- and beta-globin chains with an excess of free alpha-globin c

34 stabilizes its erythroid complex partners on beta-globin chromatin, even though it is not one of the

35 3C) assay shows that BCL11A reconfigures the beta-globin cluster by modulating chromosomal loop forma

36 agments of rat ASBT were incorporated into a beta-globin coding mRNA construct for analysis of mRNA s

37 Using PTC-containing human genomic beta-globin constructs, we show that a fraction ( 30%) o

38 repeatedly invalid by the cobas test due to beta-globin control failures, highlighting amplification

39 that homopolymer A/T tracts within the human beta-globin CoTC-mediated terminator element play a crit

40 vide therapeutic HbF levels to patients with beta-globin deficiency.

41 universal strategy to ameliorate the severe beta-globin disorders sickle cell disease (SCD) and beta

42 AR1 as an alternative therapeutic target for beta-globin disorders.

43 dults ameliorates the severity of the common beta-globin disorders.

44 in (HbF) modifies the severity of the common beta-globin disorders.

45 cond-generation form of gene therapy for the beta-globin disorders.

46 sents a promising therapeutic avenue for the beta-globin disorders.

47 o were asymptomatic) by quantitative CMV and beta-globin DNA-specific PCR.

48 onic 1 (HS-E1), located within the embryonic beta-globin domain in mouse, which is homologous to a re

49 that the +60ZF-DBD was targeted to the adult beta-globin downstream promoter and that the binding of

50 nstrate that targeting a ZF-DBD to the adult beta-globin downstream promoter region interferes with t

51 ecule RNA FISH experiments revealed that the beta-globin enhancer (LCR) predominantly augments transc

52 journey began with mechanistic studies on a beta-globin enhancer- and promoter-binding factor, GATA-

53 Intrahepatic in utero injections of a beta globin-expressing lentiviral vector (GLOBE), were p

54 eport a clinically relevant forward-oriented beta-globin-expressing vector, which has sixfold higher

55 KLF1) and KLF2 positively regulate embryonic beta-globin expression and have additional overlapping r

56 HIRA is not only critical for beta-globin expression but is also required for activati

57 The requirement of intron 2 for high-level beta-globin expression dictates a reverse-oriented globi

58 Knockdown of beta-globin expression in cultured primary human erythro

59 encoding ADNP/ADNP2 significantly increased beta-globin expression in MEL cells in the absence of an

60 gamma 1 (HBG1) and HBG2, decreases and adult beta-globin expression increases, thereby shifting red b

61 rived erythroid progenitor-2 cells, in which beta-globin expression is similarly affected by triplex

62 immature EBs characterized by low GATA1 and beta-globin expression, but PV cultures generated great

63 thesis, with a reciprocal reduction in adult beta-globin expression.

64 ineage, associated with direct regulation of beta-globin expression.

65 in gamma-globin expression and reduction in beta-globin expression.

66 D34+ cell yields but also provides increased beta-globin expression/VCN and enhanced early human chim

67 Plerixafor+G-CSF cells produced the highest beta-globin expression/VCN.

68 05+/-0.12 (p=0.05), indicating expression of beta-globin from the integrated SB transgene.

69 ct point mutations at two sites in the human beta globin gene.

70 lobin gene was replaced with a second marked beta-globin gene (beta(m)), coupled to an intact LCR, a

71 suggestive of deletions in the locus of the beta-globin gene (beta-globin locus).

72 int mutations or deletions of nucleotides in beta-globin gene (HBB).

73 cells leads to a higher level of endogenous beta-globin gene activation after differentiation.

74 location, in the present study, we compared beta-globin gene activity and location in erythroid cell

75 n of RNA polymerase II (Pol II) at the adult beta-globin gene and at the same time increased the bind

76 rminal repeat and gag gene as well as in the beta-globin gene and LCR.

77 for human beta thalassemia by knocking down beta-globin gene and protein expression in cultured CD34

78 A fourth channel detects the human beta-globin gene as a control for sample adequacy and as

79 cant, binding affinity, particularly for the beta-globin gene but results in a disease phenotype even

80 correlates with increased H3K9me3 across the beta-globin gene cluster and locus control region.

81 It controls the beta-globin gene cluster in concert with other factors.

82 Human gamma- and beta-globin gene competition is evident around the time

83 The human beta-globin gene contains an 18-nucleotide coding strand

84 The adult beta-globin gene contains an E-box located 60 bp downstr

85 tosis, reversal of splenomegaly and up to 7% beta-globin gene correction in HSCs, with extremely low

86 The LCR and beta-globin gene establish proximity in these cells.

87 UNC0638 activates fetal and represses adult beta-globin gene expression in adult human hematopoietic

88 Here we studied UNC0638 effects on beta-globin gene expression using ex vivo differentiatio

89 complexes has been shown to be required for beta-globin gene expression.

90 occupied by KLF1, a major regulator of adult beta-globin gene expression.

91 regulatory DNA element associated with adult beta-globin gene expression.

92 ing new therapeutic targets for disorders of beta-globin gene expression.

93 segment embedded in the second intron of the beta-globin gene forms a DNA.RNA triplex with the HS2 se

94 quantitative trait loci and the geography of beta-globin gene haplotypes, especially those found in t

95 mplate to correct the sickle mutation in the beta-globin gene in hematopoietic stem and progenitor ce

96 ent expression of an effective anti-sickling beta-globin gene in human SCD BM CD34+ progenitor cells,

97 vector-mediated addition of an antisickling beta-globin gene into autologous hematopoietic stem cell

98 uch hyperacetylated domains within mammalian beta-globin gene loci, and determined that within the mu

99 The beta-globin gene locus is a paradigm of cell- and develo

100 lyzed protein-DNA interactions in the murine beta-globin gene locus using the methyltransferase acces

101 ecruitment of transcription complexes to the beta-globin gene locus.

102 imate-specific ERV-9 LTR in the 100 kb human beta-globin gene locus.

103 lassemia, a common hemoglobinopathy in which beta-globin gene mutations cause the accumulation and pr

104 Genome editing to correct a defective beta-globin gene or induce fetal globin (HbF) for patien

105 tor binding sites in LCR HS2 or in the adult beta-globin gene promoter regions exhibit low fractional

106 ate the association of Pol II with the adult beta-globin gene promoter.

107 s from a homozygous missense mutation in the beta-globin gene that causes polymerization of hemoglobi

108 nsequences of the A to T substitution in the beta-globin gene that produces haemoglobin S, which lead

109 rol region (LCR) is necessary for high-level beta-globin gene transcription and differentiation-depen

110 n intact single cells after the induction of beta-globin gene transcription.

111 validated a safe and effective procedure for beta-globin gene transfer in thalassemia patient CD34(+)

112 vectors carrying a reporter gene or a human beta-globin gene under the control of a reduced-size loc

113 gments transcriptional burst fraction of the beta-globin gene with modest stimulation of burst size.

114 a cells reduced the binding of KLF1 with the beta-globin gene, but not with locus control region elem

115 e point mutation in the seventh codon of the beta-globin gene.

116 profile of transcription and splicing of the beta-globin gene.

117 major, which is characterized by a defective beta-globin gene.

118 poly(A) signal in a chromosomally integrated beta-globin gene.

119 but revealed reduced expression of the adult beta-globin gene.

120 r caused by a missense mutation in the adult beta-globin gene.

121 tions to restore expression of the wild-type beta-globin gene.

122 made to detect the sequences anent with the beta-globin gene.

123 aj)-promoter as well as transcription of the beta-globin gene.

124 e disorders caused by mutations in the adult beta-globin gene.

125 (betath) are the result of mutations in the beta-globin gene.

126 coding potential or expression of the adult beta-globin gene.

127 ression of erythroid genes such as the adult beta-globin gene.

128 to single K562 cells for targeting the human beta-globin gene.

129 m (ES) cells with all of the adult alpha and beta globin genes deleted (Hb Null).

130 isease (SCD) is caused by a mutation in both beta globin genes, resulting in chronic hemolysis and mu

131 ating the fetal (gamma) and adult (delta and beta) globin genes (encompassing the HBBP1 and BGLT3 non

132 decrease in the expression of the embryonic beta-globin genes and loss of the domain-wide pattern of

133 e limiting effect on RNA levels occurs after beta-globin genes associate with TFys, at which time HSs

134 f altitudinal differentiation at each of the beta-globin genes drop away quite rapidly in the externa

135 ts of the enhancer cluster controlling human beta-globin genes establishes evidence for composition-b

136 a marked increase in expression of embryonic beta-globin genes Hbb-epsilony and Hbb-betah1.

137 Transcription of beta-globin genes is regulated by the distant locus cont

138 ample is provided by the tandemly duplicated beta-globin genes of deer mice (Peromyscus maniculatus),

139 opulation genetic analysis of the duplicated beta-globin genes of Indian house mice (Mus castaneus) i

140 and linkage disequilibrium in the duplicated beta-globin genes of M. castaneus, (ii) to test the hypo

141 Remarkably, the two beta-globin genes segregate the same pair of functionall

142 upstream of the human fetal gamma- and adult beta-globin genes serves a long-range, host function.

143 positively regulate the embryonic and fetal beta-globin genes through direct promoter binding.

144 ce with different genetic backgrounds at the beta-globin genes.

145 e regulator of the mouse and human embryonic beta-globin genes.

146 alassemia, G6PD A(-) variant deficiency, and beta-globin haplotype in 2 cohorts of children with well

147 ddle East have revealed that two alternative beta-globin haplotypes, Hbb(d) and Hbb(p), are often pre

148 erentiation into erythrocytes, express adult beta-globin (HbA) messenger RNA, which confirms intact t

149 thalassaemia, are caused by mutations in the beta-globin (HBB) gene and affect millions of people wor

150 om point mutations or small deletions in the beta-globin (HBB) gene that ultimately cause anemia.

151 n origins, including the origin at the human beta-globin (HBB) locus.

152 cancers, we observe consistent induction of beta-globin (HBB), but not its partner alpha-globin (HBA

153 integrase-targeted attB element and two dual beta-globin HS4 insulators flanking a reporter transgene

154 necessary to achieve robust transcription of beta-globin in nuclear transcription factories.

155 thymine mutation at nucleotide 654 of human beta-globin intron 2 (beta(IVS2-654)) is one of the most

156 of immature EBs with low levels of GATA1 and beta-globin irrespective of dexamethasone stimulation.

157 Altogether, these observations suggest that beta-globin is selectively deregulated in cancer cells,

158 in in the presence of defective synthesis of beta-globin is the primary mechanism for anaemia in beta

159 maintain therapeutically relevant levels of beta-globin it would require lifelong administration.

160 for+G-CSF were transduced with the TNS9.3.55 beta-globin lentivector and compared for transducibility

161 neration lentiviral vectors (LVs) carrying a beta-globin-like gene have revolutionized this field by

162 S3 protein compromised a fourth of the total beta-globin-like transcripts and hemoglobin (Hb) tetrame

163 2 Mb on human chromosome 11 encompassing the beta-globin locus and flanking olfactory receptor genes.

164 e chromatin occupancy of BCL11A at the human beta-globin locus and other genomic regions in vivo by h

165 nstrate that GATA-1 induces extrusion of the beta-globin locus away from its chromosome territory at

166 acting factors binding to these sites in the beta-globin locus by using chromatin immunoprecipitation

167 Transgenic mice that contain the human beta-globin locus complete their fetal-to-adult hemoglob

168 mediates long-range interaction between the beta-globin locus control region (LCR) and active globin

169 mediates long-range interaction between the beta-globin locus control region (LCR) and gene in adult

170 The beta-globin locus control region (LCR) is necessary for

171 aseI hypersensitive sites (HSs) of the human beta-globin locus control region (LCR) may function as p

172 via LMO2 and is required for looping of the beta-globin locus control region (LCR) to the active bet

173 A and protein and KLF2 binding to HS2 of the beta-globin locus control region and enhanced -globin mR

174 acetylation and nucleosome occupancy in the beta-globin locus control region and gamma-globin gene.

175 d to establish spatial proximity between the beta-globin locus control region and gene and for transc

176 cure these disorders is the discovery of the beta-globin locus control region and several associated

177 tment of ADNP, similar to Brg1, to the mouse beta-globin locus control region in MEL cells.

178 ed that within the murine locus, neither the beta-globin locus control region nor the gene promoters

179 RNA triplex with the HS2 sequence within the beta-globin locus control region, a major regulator of g

180 surveyed the protein complexes that bind at beta-globin locus control region, and purified and chara

181 Ey- and betah1-globin genes, and also to the beta-globin locus control region, as demonstrated by ChI

182 ion, with ADNP directly associating with the beta-globin locus control region.

183 differentiation-dependent relocation of the beta-globin locus from the nuclear periphery to the cent

184 ll-length beta-globin cDNA to the endogenous beta-globin locus in 19% of cells prior to selection as

185 occupancy and chromatin conformation of the beta-globin locus in human erythroid cells.

186 ired for normal histone modifications in the beta-globin locus in mouse embryos.

187 The human beta-globin locus is comprised of embryonic, fetal, and

188 er in GATA1 null erythroblasts, in which the beta-globin locus is relaxed and inactive.

189 Deletion of HS-E1 from the endogenous murine beta-globin locus results in significant decrease in the

190 in vivo model, we used mice carrying a human beta-globin locus transgene with combinations of Klf1 kn

191 In this work, a dual human beta-globin locus transgenic and KLF knockout mouse mode

192 The beta-globin locus undergoes dynamic chromatin interactio

193 binding 1 (LDB1) protein is recruited to the beta-globin locus via LMO2 and is required for looping o

194 Demethylation at the erythroid-specific beta-globin locus was coincident with global DNA demethy

195 monstrate efficient targeted cleavage at the beta-globin locus with minimal off-target modification.

196 a 400-bp insulator fragment from the chicken beta-globin locus within the self-inactivating long-term

197 stinguish between these possibilities, human beta-globin locus yeast artificial chromosome (beta-YAC)

198 reduces OGT and OGA promoter interactions in beta-globin locus yeast artificial chromosome (beta-YAC)

199 etions in the locus of the beta-globin gene (beta-globin locus).

200 The major transcriptional enhancer of the beta-globin locus, called the locus control region (LCR)

201 We describe an interaction at the human beta-globin locus, in which an RNA segment embedded in t

202 hroblasts from mice transgenic for the human beta-globin locus, like human fetal erythroblasts, expre

203 Further studies revealed mosaic UPD of the beta-globin locus, more SS erythroid progenitors than AS

204 three families with unusual deletions in the beta-globin locus, we identified an intergenic region ne

205 particularly well analyzed at the mammalian beta-globin locus, where transcription factors such as e

206 pin RNA, compromising its association in the beta-globin locus.

207 fications were largely maintained across the beta-globin locus.

208 e DNA replication-Initiation Region from the beta-globin locus.

209 ly occur at the transcriptional level at the beta-globin locus.

210 r activity in the erythroid-specific chicken beta-globin locus.

211 sage of the origin of DNA replication at the beta-globin locus.

212 g patterns at the developmentally controlled beta-globin locus.

213 ee genomic FRT-like sequences located in the beta-globin locus.

214 globin subunit beta gene (HBB; which encodes beta-globin), mainly sickle cell disease (SCD) and beta-

215 Amplifiable beta-globin (melting temperature [Tm], 87.2 degrees C +/

216 lyses that identified a cytoplasm-restricted beta-globin messenger ribonucleoprotein (mRNP) complex i

217 rinted H19-Igf2 locus as well as the complex beta-globin, MHC class II and IFN-gamma loci.

218 itive GFP (roGFP2) sensor under control of a beta-globin mini-promoter, directing expression specific

219 ntron or heterologous introns from the human beta-globin, mouse Down syndrome critical region 1, or h

220 ound that both simvastatin and tBHQ suppress beta-globin mRNA and KLF1 and BCL11A mRNA and protein, s

221 hich showed restoration of correctly spliced beta-globin mRNA and led to haemoglobin A synthesis, and

222 sm for the posttranscriptional regulation of beta-globin mRNA during normal erythropoiesis, providing

223 of the mRNP to normal steady-state levels of beta-globin mRNA in erythroid precursors.

224 FISH) and measured the NMD of PTC-containing beta-globin mRNA in intact single cells after the induct

225 unt for the constitutively high half-life of beta-globin mRNA in the cytoplasm of their anucleate ery

226 that decay of the majority of PTC-containing beta-globin mRNA occurs soon after its export into the c

227 recently been implicated as a determinant of beta-globin mRNA stability.

228 non-erythroid cell types, similar to native beta-globin mRNA that was also expressed at low levels.

229 IHK-beta-globin mRNA was found in non-erythroid cell types,

230 Beta-globin mRNA was reduced by 90% compared to controls

231 Factors that regulate beta-globin mRNA within the nucleus of early erythroid p

232 similar to the half-life of normal PTC-free beta-globin mRNA, indicating that it had evaded NMD.

233 We have previously shown that human beta-globin mRNAs carrying PTCs in close proximity to th

234 n the 1960s and on translation of alpha- and beta-globin mRNAs in the early 1970s.

235 ersal strategy to correct different types of beta-globin mutations in beta-Thal iPSCs for disease mod

236 tic stem cells expressing curative levels of beta-globin on differentiation.

237 lated with increase in CDC6 occupancy on the beta-globin origin of replication, suggesting increment

238 expression approximated the one-copy normal beta-globin output.

239 l pattern whereby perfect LD between the two beta-globin paralogs (which are separated by 16.2 kb) is

240 nd reconstructed haplotype networks for both beta-globin paralogs revealed extensive allele sharing w

241 haplotypes harbor two functionally distinct beta-globin paralogs, HBB-T1 (which encodes the beta-cha

242 with both valid oral and genital specimens (beta-globin-positive).

243 This mutation results in aberrant beta-globin pre-mRNA splicing and prevents synthesis of

244 nt (RRE) allows intron 2 to be retained, and beta-globin production is observed in transplanted macaq

245 matopoiesis that generates erythrocytes with beta-globin production.

246 ress GFP under the control of the endogenous beta-globin promoter and tdTomato under the control of t

247 n transcriptional bursting, we forced an LCR-beta-globin promoter chromatin loop.

248 specifically dissociate only from the adult beta-globin promoter concomitant with its activation but

249 as9-directed tethering of mutant LDB1 to the beta-globin promoter forced LCR loop formation in the ab

250 id cells, the locus control region (LCR) and beta-globin promoter form a chromatin loop that requires

251 cial zinc fingers (ZF) to tether Ldb1 to the beta-globin promoter in GATA1 null erythroblasts, in whi

252 show that PIAS3 preferentially occupies the beta-globin promoter in undifferentiated murine erythrol

253 egalovirus early enhancer/chicken beta-actin/beta-globin promoter into the Rosa26 locus, respectively

254 1 or only its self-association domain to the beta-globin promoter substantially activated beta-globin

255 CRISPR/Cas9 editing of the beta-globin promoter to eliminate the RNA Pol II PIC by

256 self-association domain (SA) of Ldb1 to the beta-globin promoter via artificial zinc fingers.

257 Similar to its role at the beta-globin promoter, KLF1 induces factor recruitment an

258 3 variant is differentially recruited to the beta-globin promoter.

259 bin locus control region (LCR) to the active beta-globin promoter.

260 f interactions between the LCR and the adult beta-globin promoter.

261 a-type globin promoters but not to the adult beta-globin promoter.

262 The normal accumulation of beta-globin protein in terminally differentiating erythr

263 has been shown to restore expression of the beta-globin protein, but to maintain therapeutically rel

264 pre-mRNA splicing and prevents synthesis of beta-globin protein.

265 t form of LDB1 in LDB1 knock down cells: LCR/beta-globin proximity was restored without mediator core

266 diator occupancy and resulted in loss of LCR/beta-globin proximity.

267 establishment of locus control region (LCR)/beta-globin proximity.

268 Impaired expression of beta-globin reduces adult hemoglobin (alpha2beta2) produ

269 tment, the level of therapeutic antisickling beta-globin remained high (approximately 50% of beta-lik

270 essential asymmetric region within the human beta-globin Rep-P replicator and includes hnRNP C1/C2, S

271 egion (S/MAR) for episomal retention and the beta-globin Replicator, the DNA replication-Initiation R

272 oter SFFV instead of CMV and the addition of beta-globin Replicator, transferred into CD34(+) cells,

273 or in both introns 1 and 2 of an integrated beta-globin reporter gene caused a shift in relative dis

274 on, significantly decreased the stability of beta-globin reporter mRNA.

275 ract to increase the levels of an intronless beta-globin reporter RNA.

276 ertion of this sequence into the 3' UTR of a beta-globin reporter transcript conferred instability to

277 Lentiviral-mediated transduction of beta-globin shRNA (beta-KD) caused imbalanced globin cha

278 Following completion of the gamma- to-beta-globin switch, adult erythroid cells synthesize low

279 ndition, mutations attenuate gamma-globin-to-beta-globin switching, causing high-level HbF expression

280 In beta-thalassemia, beta-globin synthesis is reduced causing alpha-globin ac

281 anemia caused by partial or complete loss of beta-globin synthesis, leading to ineffective erythropoi

282 transcription to approximately 85% of total beta-globin synthesis, with a reciprocal reduction in ad

283 tal genes to improve the deficiency in adult beta-globin synthesis.

284 and deletions (indels) and found that, with beta-globin-targeting TALENs, similar levels of on- and

285 We found that the human beta-globin terminator was an efficient inhibitor of dow

286 An N-terminal NEAT domain binds alpha/beta globin through a site distant from the globin heme

287 The mature erythroid cells had an increased beta-globin to gamma-globin ratio from 0.66+/-0.08 to 1.

288 r when a full-length intron-containing human beta-globin transcript is expressed.

289 beta-globin promoter substantially activated beta-globin transcription in the absence of GATA1.

290 f fetal gamma-globin and repression of adult beta-globin transcription.

291 creased gamma-globin combined with decreased beta-globin transcripts resulting in gamma-globin rising

292 th and co-transcriptionally degrades nascent beta-globin transcripts, mutated to inhibit splicing or

293 e adult marrow proerythroblasts express only beta-globin transcripts.

294 Beauty vectors in carrying an insulated IHK-beta-globin transgene for gene therapy of sickle cell di

295 od CD34(+) cells with DsRed and a hybrid IHK-beta-globin transgene.

296 insulator elements to protect DsRed and IHK-beta-globin transgenes from silencing in long-term cultu

297 In human beta-globin transgenic Eto2 null mice and in human CD34+

298 e a conserved molecular mechanism leading to beta-globin variants underlying phenotypic diversity and

299 c, but not in the fetal liver; and wild-type beta-globin was co-expressed in adult mice.

300 lobin molecules that failed to assemble with beta-globin were selectively ubiquitinated by UBE2O.