ライフサイエンスコーパス: HSPC

1 HSPC expansion using zwitterionic hydrogels has the pote

2 HSPC generation via EHT is thought to be restricted to t

3 HSPC suppression was largely dependent on secreted facto

4 wed that the transplantation of CD33-ablated HSPCs with CD33-targeted immunotherapy leads to leukemia

5 of multilineage descendants of CD33-ablated HSPCs.

6 P, and their enforced co-expression in adult HSPCs reactivated fetal-like B-cell development in vivo

7 g protein (RBP) Lin28b in respecifying adult HSPCs to resemble their fetal counterparts.

8 hich instructs young HSPCs to behave as aged HSPCs.

9 aled that their transcriptome resembled aged HSPCs.

10 tion of either STC1 or HIF-1alpha alleviated HSPC suppression by AML.

11 that represents an alternative to allogeneic HSPC transplantation.

12 d CD34(+) HSPCs with stimulatory EVs-altered HSPC transcriptome, including genes with known roles in

13 hese data show that physical activity alters HSPCs via modulation of their niche, reducing hematopoie

14 Among HSPCs, we found that the receptor for IL-33, ST2, is exp

15 humanized model to study the crosstalk among HSPCs, leukemia, and their MSC niche, and a molecular me

16 that the human fetal thymus generates, in an HSPC/Lin28b-dependent manner, invariant gammadelta T cel

17 o and is also required to drive arterial and HSPC formation.

18 G) can rescue the expression of arterial and HSPC markers in the HE and CHT in plcg1(-/-) mutant embr

19 tor, p53, rescues the loss of both Notch and HSPC phenotypes in supt16h mutants.

20 g-I or Mda5 rescued inflammatory signals and HSPC numbers.

21 ic defects in blood vessel specification and HSPC formation in plcg1(-/-) mutants.

22 detected in hemogenic endothelial cells and HSPCs, suggesting a role as RLR ligands.

23 altered crosstalk between the BMEC niche and HSPCs, which instructs young HSPCs to behave as aged HSP

24 ation in mice compared to currently approved HSPC mobilization methods, it represents an exciting pot

25 lls has the potential to generate autologous HSPCs for clinical applications.

26 Medicinal products based on autologous HSPCs corrected using lentiviral and gammaretroviral vec

27 p66Shc pathway as a mechanistic link between HSPC mobilopathy and excessive myelopoiesis.

28 SC is cell-contact dependent and mediated by HSPC connexin-43 (Cx43).

29 tend these studies by culturing human CD133+ HSPCs on nanofibre scaffolds to mimic the niche for 5-da

30 ment of umbilical cord blood-derived CD34(+) HSPCs with stimulatory EVs-altered HSPC transcriptome, i

31 though a massive expansion of total CD34(+) HSPCs was observed, none of the tested culture condition

32 stromal cells (eMSCs) together with CD34(+) HSPCs creates an in vivo synthetic niche in the dermis o

33 expression in vivo and the number of CD41(+) HSPCs downstream of HE specification.

34 components, including il1b, reduced CD41(+) HSPCs and prevented their expansion in response to metab

35 n IAC cells result in 2 populations of CD45+ HSPCs; an initial wave of lymphomyeloid-biased progenito

36 es absolute Lin-CD34+CD38-CD45RA-CD90+CD49f+ HSPC numbers, while concomitantly decreasing the Lin-CD3

37 the hematopoietic stem and progenitor cell (HSPC) compartment aneuploid cells have reduced fitness a

38 uman hematopoietic stem and progenitor cell (HSPC) depletion in immune-mediated bone marrow failure s

39 for hematopoietic stem and progenitor cell (HSPC) development.

40 otes hematopoietic stem and progenitor cell (HSPC) expansion are largely unknown.

41 Hematopoietic stem and progenitor cell (HSPC) formation and lineage differentiation involve gene

42 e in hematopoietic stem and progenitor cell (HSPC) function.

43 Haematopoietic stem and progenitor cell (HSPC) gene therapy has emerged as an effective treatment

44 -mediated haemopoietic stem/progenitor cell (HSPC) gene therapy is a potentially curative treatment t

45 ates hematopoietic stem and progenitor cell (HSPC) proliferation and leukocyte production, as well as

46 biological role of EVs in osteolineage cell-HSPC crosstalk and promotes the utility of EVs and their

47 9 technology in human stem/progenitor cells (HSPC) and provide evidence that the deletion of CD33 in

48 , human hematopoietic stem/progenitor cells (HSPC) are transduced with lentiviruses expressing a muta

49 tion of hematopoietic stem/progenitor cells (HSPC) from the bone marrow (BM) is impaired in diabetes.

50 recruit hematopoietic stem/progenitor cells (HSPC) from the bone marrow and differentiate them into t

51 Hematopoietic stem/progenitor cells (HSPC) in zebrafish emerge from the aortic hemogenic endo

52 of hematopoietic stem and progenitor cells (HSPC) in zebrafish.

53 of hematopoietic stem and progenitor cells (HSPC) is tightly regulated by their bone marrow (BM) mic

54 row hematopoietic stem and progenitor cells (HSPC), but these cells cannot leave the bone marrow, eve

55 etal hematopoietic stem and precursor cells (HSPCs) caused by high expression of the RNA-binding prot

56 oreover, hematopoietic stem/precursor cells (HSPCs) doubly deficient for Tet2 and Dnmt3a displayed gr

57 hed hematopoietic stem and progenitor cells (HSPCs) and correlate the proteomes to the corresponding

58 Hematopoietic stem and progenitor cells (HSPCs) and leukocytes circulate between the bone marrow

59 Hematopoietic Stem/Progenitor cells (HSPCs) are endowed with the role of maintaining a divers

60 ain hematopoietic stem and progenitor cells (HSPCs) are generally characterized in steady-state condi

61 at haematopoietic stem and progenitor cells (HSPCs) are generated from a transient subset of speciali

62 mal hematopoietic stem and progenitor cells (HSPCs) are impeded in AML-infiltrated bone marrow (BM).

63 ive hematopoietic stem and progenitor cells (HSPCs) arise from the transdifferentiation of hemogenic

64 Hematopoietic stem and progenitor cells (HSPCs) develop in distinct waves at various anatomical s

65 tes hematopoietic stem and progenitor cells (HSPCs) essential for establishment and maintenance of th

66 and hematopoietic stem and progenitor cells (HSPCs) ex vivo is critical to fully realize the potentia

67 ged hematopoietic stem and progenitor cells (HSPCs) exhibit increased ground-stage NF-kappaB activity

68 of hematopoietic stem and progenitor cells (HSPCs) for stem cell transplantation, with a five-day co

69 tion of hematopoietic stem/progenitor cells (HSPCs) from the bone marrow (BM), which can worsen the o

70 of hematopoietic stem and progenitor cells (HSPCs) from the graft.

71 Haematopoietic stem and progenitor cells (HSPCs) have been the focus of developmental and regenera

72 tal hematopoietic stem and progenitor cells (HSPCs) hold promise to cure a wide array of hematologica

73 of hematopoietic stem and progenitor cells (HSPCs) in the bone marrow (BM).

74 Hematopoietic stem and progenitor cells (HSPCs) in the bone marrow are derived from a small popul

75 rts hematopoietic stem and progenitor cells (HSPCs) in the bone marrow is a highly dynamic structure.

76 of hematopoietic stem and progenitor cells (HSPCs) in thrombocytopenia.

77 riming haematopoietic stem/progenitor cells (HSPCs) in vitro with specific chromatin modifying agents

78 the hematopoietic stem and progenitor cells (HSPCs) involves homing to the vasculatures and lodgment

79 of hematopoietic stem and progenitor cells (HSPCs) is essential for maintaining hematopoietic and ti

80 of hematopoietic stem and progenitor cells (HSPCs) posttransplant.

81 in hematopoietic stem and progenitor cells (HSPCs) results in altered hematopoietic function, increa

82 nic hematopoietic stem and progenitor cells (HSPCs) robustly proliferate while maintaining multilinea

83 in hematopoietic stem and progenitor cells (HSPCs) significantly decreased DNA-PKcs activity followi

84 Hematopoietic stem progenitor cells (HSPCs) stimulate revascularization of ischemic areas.

85 of hematopoietic stem and progenitor cells (HSPCs) to sustain long-term engraftment and can specific

86 hat hematopoietic stem and progenitor cells (HSPCs) undergo translational reprogramming mediated by p

87 man hematopoietic stem and progenitor cells (HSPCs) using clonal tracking in patients treated with ge

88 man hematopoietic stem and progenitor cells (HSPCs), each gene-corrected cell and its progeny are mar

89 Hematopoietic stem and progenitor cells (HSPCs), first specified from hemogenic endothelium (HE)

90 tion of hematopoietic stem/progenitor cells (HSPCs), in vitro culture, lentivirus vector transduction

91 (-) hematopoietic stem and progenitor cells (HSPCs), single-cell proteomics, genomics, and functional

92 MDS hematopoietic stem and progenitor cells (HSPCs), the mechanisms responsible for the competitive a

93 man hematopoietic stem and progenitor cells (HSPCs), without impairing cell viability and differentia

94 in hematopoietic stem and progenitor cells (HSPCs).

95 efficacy of hUCB HSCs and progenitor cells (HSPCs).

96 man hematopoietic stem and progenitor cells (HSPCs).

97 nal hematopoietic stem and progenitor cells (HSPCs).

98 (+) hematopoietic stem and progenitor cells (HSPCs).

99 ived(6) hematopoietic stem progenitor cells (HSPCs).

100 man hematopoietic stem and progenitor cells (HSPCs).

101 editing hematopoietic stem/progenitor cells (HSPCs).

102 of hematopoietic stem and progenitor cells (HSPCs).

103 (+) hematopoietic stem and progenitor cells (HSPCs).

104 man hematopoietic stem and progenitor cells (HSPCs).

105 to haematopoietic stem and progenitor cells (HSPCs).

106 and hematopoietic stem and progenitor cells (HSPCs).

107 gulate day/night oscillations of circulating HSPCs and leukocytes.

108 Mutant-clone HSPCs have increased expression of megakaryocyte-associa

109 topoietic stem cells and loss of competitive HSPC repopulation.

110 Engraftment of genetically corrected HSPCs was successful and sustained in all patients.

111 topoietic system partially rescued defective HSPC mobilization in diabetes.

112 th isolated mitochondria from Cx43 deficient HSPCs.

113 CXCR4 accumulates in Gprasp-deficient HSPCs, boosting their function posttransplant.

114 unoccupied by Stag1, even in Stag2-deficient HSPCs.

115 ed the suppression of phenotypically defined HSPC differentiation without affecting their viability.

116 ics and gradual replacement of donor-derived HSPCs from a circulating pool.

117 , we identified a wide range of hPSC-derived HSPCs phenotypes, including a small group classified as

118 e CD34(+) cord blood and bone-marrow-derived HSPCs.

119 disease and beta-thalassemia patient-derived HSPCs, respectively.

120 Mechanistically, loss of mDia2 disrupts HSPC polarization and induced cytoplasmic accumulation o

121 espondingly observed coexistence of distinct HSPC subpopulations expressing high levels of TP53 or MY

122 Host stromal ME recovery and donor HSPC engraftment were augmented after mitochondria trans

123 Our findings demonstrate that healthy donor HSPC not only reconstitute the hematopoietic system afte

124 nterleukin-1-beta (IL1beta) signaling drives HSPC production in response to metabolic activity.

125 immediate progenies, i.e., recently emerged HSPCs.

126 Preclinical studies have shown engineered HSPCs could also be used to cross-correct non-haematopoi

127 loidy peak: rapid expansion of the engrafted HSPC population and bone marrow microenvironment degrada

128 c expression of repetitive elements enhanced HSPC formation in wild-type, but not in Rig-I or Mda5 de

129 cy impaired, while Lgp2 deficiency enhanced, HSPC emergence in zebrafish embryos.

130 y limiting dilution assays, and the expanded HSPCs were capable of hematopoietic reconstitution for a

131 as a conserved metabolic sensor that expands HSPC production in vivo and in vitro.

132 Cdx2-expressing HSPCs demonstrate enrichment of hematopoietic-specific e

133 genes, consistent with enhanced egress of FA HSPCs from bone marrow to peripheral blood.

134 Irradiation-based conditioning for HSPC transplantation led to the loss of most of these po

135 SRF-beta2 integrin signaling is critical for HSPC lodgment to the niches.

136 h-signalling components, genes essential for HSPC development, due to abrogated transcription.

137 regulator HIF-1alpha as limiting factors for HSPC proliferation.

138 ydrogel and the 3D format were important for HSPC self-renewal.

139 ranscription (FACT) complex, is required for HSPC formation.

140 (G-CSF) as the most common regimen used for HSPC mobilization.

141 neous nature of the cell lineage output from HSPCs and provided methods for analyzing these complex d

142 ve demonstrated the existence of functioning HSPCs in human intestines with implications for promotin

143 lial-to-hematopoietic transition to generate HSPCs therefrom.

144 tive role of EC-derived signals in governing HSPC aging.

145 Human gut HSPCs are phenotypically similar to bone marrow HSPCs an

146 Donor healthy HSPC transfer functional mitochondria to the stromal ME,

147 tionally validated LSCs, blasts, and healthy HSPCs, representing a valuable resource helping to desig

148 omparing LSCs to leukemic blasts and healthy HSPCs, we validate candidate LSC markers and highlight n

149 MYC-high HSPCs showed significant downregulation of cell adhesion

150 How HSPCs transmigrate from the vasculature to the niches is

151 Understanding how HSPCs migrate between bone marrow (BM) and peripheral ti

152 uction on HE fate, relevant to de novo human HSPC production.

153 n hematopoietic cell lines and primary human HSPC.

154 essed in primary human LSCs and normal human HSPCs.

155 e the potential of RNP base editing of human HSPCs as a feasible alternative to nuclease editing for

156 Our longitudinal study of human HSPCs carried in intestinal allografts demonstrates thei

157 Here, using 3D culture of human HSPCs in a degradable zwitterionic hydrogel, we achieved

158 ing pathways and decreased survival of human HSPCs.

159 hich IFN-gamma impairs the function of human HSPCs.

160 nitial inflammasome stimulation of Il1rl1(+) HSPCs.

161 imate link between myelopoiesis and impaired HSPC mobilization after G-CSF stimulation was confirmed

162 We speculate that MYC overexpression impairs HSPC function in FA patients and contributes to exhausti

163 Here, we review the most recent advances in HSPC gene therapy and discuss emerging strategies for us

164 phosphate-activated protein kinase (AMPK) in HSPC, dramatically increasing mitochondria transfer to B

165 rovide evidence that the deletion of CD33 in HSPC doesn't impair their ability to engraft and to repo

166 ch was rescued upon re-expression of Cx43 in HSPC or culture with isolated mitochondria from Cx43 def

167 on of Phc2 in mice causes a severe defect in HSPC mobilization through the derepression of Vcam1 in b

168 This was reflected in HSPC-derived cells, which show aberrantly high expressio

169 nvolvement of RIG-I-like receptors (RLRs) in HSPC formation.

170 enin-angiotensin system and are expressed in HSPCs.

171 s that are absent or only lowly expressed in HSPCs.

172 Conditional Cdx2 expression in HSPCs is an inducible model of de novo leukemic transfor

173 Knockout of beta2 integrins in HSPCs phenocopies mDia2 deficient mice.

174 ed no increase in coding region mutations in HSPCs from EGF-treated mice, but increased intergenic co

175 essed MLLT3 localized to active promoters in HSPCs, sustained levels of H3K79me2 and protected the HS

176 escued physiological and genotoxic stress in HSPCs from FA mice, showing that MYC promotes proliferat

177 ulate a prominent LSC-specific AHR target in HSPCs, suggesting that differential mechanisms govern FI

178 macologic inflammasome stimulation increased HSPC number as assessed by in situ hybridization for run

179 ibition impairs hypercholesterolemia-induced HSPC expansion.

180 tion mimicked Yap overexpression and induced HSPCs in embryos lacking blood flow.

181 and safety of lentiviral gene transfer into HSPCs.

182 The technology involves HSPC mobilization and intravenous injection of an integr

183 ivo HSPC gene therapy approach that involves HSPC mobilization and an intravenous injection of integr

184 Significantly, in latent HSPCs, viral transcripts could be detected only in monoc

185 Even though Plerixafor liberates HSPCs and mature immune cells from bone marrow, competit

186 ntation, has been impeded because of limited HSPC availability.

187 /cohesin-mediated NF-kappaB signaling limits HSPC function during aging and selects for cohesin-defic

188 efficiently generate multipotent long-lived HSPCs.

189 ation of RLR expression in mouse fetal liver HSPCs indicated functional conservation among species.

190 Cs are phenotypically similar to bone marrow HSPCs and have multilineage differentiation potential in

191 In response to inflammation, MDS HSPCs switched from canonical to noncanonical NF-kappaB

192 rminant for the competitive advantage of MDS HSPCs and for disease progression.

193 nistic basis for the clonal dominance of MDS HSPCs and indicate that interfering with noncanonical NF

194 onsible for the competitive advantage of MDS HSPCs in an inflammatory milieu over normal HSPCs remain

195 The cell-intrinsic response of MDS HSPCs, which involves signaling through the noncanonical

196 zwitterionic hydrogel culture on mitigating HSPC differentiation and promoting self-renewal might re

197 ally simple technology to genetically modify HSPCs in vivo.

198 n rapidly upregulated GATA1 protein in mouse HSPC promoting their erythroid differentiation.

199 ial cells and the generation of multilineage HSPCs from hemogenic endothelium.

200 the expansion or maintenance of multipotent HSPCs.

201 ciently support the expansion of multipotent HSPCs.

202 ses the repopulating potential of p53 mutant HSPCs.

203 nriched in hemECs and in oligopotent nascent HSPCs.

204 lopathy from myelopoiesis and restore normal HSPC mobilization.

205 vo niche models comprising AML cells, normal HSPCs, and mesenchymal stromal cells (MSCs).

206 HSPCs in an inflammatory milieu over normal HSPCs remain poorly defined.

207 m chronic inflammation as compared to normal HSPCs.

208 oduced less CXCL12, being arguably devoid of HSPC-retaining activity, whereas pioglitazone failed to

209 Understanding the molecular mechanisms of HSPC development in vivo is critical for understanding H

210 regulator, Phc2, as a critical modulator of HSPC trafficking.

211 arrow (BM), which can worsen the outcomes of HSPC transplantation and of diabetic complications.

212 Given the rapid kinetics and potency of HSPC mobilization provided by the VLA4 inhibitor and CXC

213 s critical to fully realize the potential of HSPC-based therapies.

214 y BM adipocytes could limit full recovery of HSPC mobilization.

215 EHT, identifying an additional regulator of HSPC development.

216 AP) as a cyclic stretch-induced regulator of HSPC formation.

217 ietin (TPO), a primary positive regulator of HSPC survival, to its receptor (c-MPL) via steric occlus

218 reliable marker for the earliest branches of HSPCs specification and we showed how its use can foster

219 e intrinsic long-term functional capacity of HSPCs is still impaired in SCI mice.

220 are capable of de novo producing a cohort of HSPCs in situ that harbour a very specific molecular sig

221 conditions, neither classical co-cultures of HSPCs with primary ECs or MSCs, even in combination, nor

222 gic activity causes predominant BM egress of HSPCs and leukocytes via beta(3)-adrenergic receptor.

223 X1 and suppresses RUNX1-induced expansion of HSPCs during development through modulation of RUNX1 act

224 axis provides proper timing and function of HSPCs as they emerge during hematopoietic development or

225 To define the dynamics and heterogeneity of HSPCs that can be generated in vitro from hPSCs, we expl

226 HE, and IAC cells, and the heterogeneity of HSPCs within IACs, we profiled ~40 000 cells from the ca

227 As occurs in AML patients, the majority of HSPCs were quiescent and showed enrichment of functional

228 athetic) dually regulates daily migration of HSPCs and leukocytes.

229 e compromised trans-endothelial migration of HSPCs since their homing to the bone marrow vasculatures

230 Rapid and synergistic mobilization of HSPCs along with an enhanced recruitment of true HSCs wa

231 for Phc2 in controlling the mobilization of HSPCs by finely tuning their bone marrow niche.

232 and the site-specific genome modification of HSPCs using gene editing techniques such as CRISPR-Cas9

233 to 50 000) was correlated with the number of HSPCs per kilogram infused.

234 Transcriptional profiling of HSPCs from mTOR(ECKO) mice revealed that their transcrip

235 It maintains core properties of HSPCs in the steady state, and modulates their prolifera

236 Sequestration of HSPCs in bone marrow after SCI is linked to aberrant che

237 em in the regulation of day/night traffic of HSPCs and leukocytes in mice.

238 em cooperate to orchestrate daily traffic of HSPCs and leukocytes.

239 0 polarization in vivo by transplantation of HSPCs isolated from the Rac2(-/-) mouse model.

240 In this paper, we focus on HSPC development in plcg1(-/-) mutants and show that gin

241 ved prohematopoietic cue, AIBP, orchestrates HSPC emergence from the hemogenic endothelium, a type of

242 duced the clonogenic potential of FA patient HSPCs but rescued physiological and genotoxic stress in

243 (CRPC) compared with hormone-sensitive PCa (HSPC) specimens.

244 binding protein 7 (CHD7) expanded phenotypic HSPCs, erythroid, and myeloid lineages in zebrafish and

245 We use nanostraws to target human primary HSPCs and show efficient delivery of mRNA, short interfe

246 ngle-cell transcriptome profiling of primary HSPCs from FA patients.

247 he competitive advantage of TLR-TRAF6-primed HSPCs could be restored by deletion of A20 or inhibition

248 had a unique capsid that targeted primitive HSPCs through human CD46, a relatively safe SB100X trans

249 ir normal hematopoietic stem and progenitor (HSPC) function, and failed to upregulate a prominent LSC

250 HSPCs following transplantation and promotes HSPC expansion after radiation-induced stress.

251 eading to increased self-renewal and reduced HSPC commitment to the B cell lineage.

252 Rig-I or Mda5 deficiency reduced HSPC numbers by inhibiting inflammatory signals that wer

253 -mediated inflammatory signals that regulate HSPC formation.

254 renergic signals have been shown to regulate HSPC and leukocyte trafficking, but the role of the chol

255 g the levels of H3K27me3 in genes regulating HSPC self-renewal and differentiation.

256 mated minimum number of active, repopulating HSPCs (which ranged from 2000 to 50 000) was correlated

257 macologic macrophage reprogramming to rescue HSPC mobilization.

258 nockout of Osm and p66Shc completely rescued HSPC mobilization.

259 lation factor (G-CSF), and partially rescued HSPC mobilization, but it increased BM adipocytes.

260 expression of SRF or beta2 integrins rescues HSPC engraftment defects associated with mDia2 deficienc

261 ator Traf6 in RLR deficient embryos restored HSPC numbers.

262 to induce Cxcl12 in stromal cells and retain HSPC.

263 an osteolineage sources do not have the same HSPC expansion promoting potential.

264 Gene-corrected SCD HSPCs retained the ability to engraft when transplanted

265 peripheral blood and bone marrow-derived SCD HSPCs, a significant reduction in sickling of red blood

266 blood cells, engraftment of gene-edited SCD HSPCs in vivo and the importance of reducing off-target

267 hip between supt16h, p53 and phc1 to specify HSPCs via modulation of Notch signalling.

268 cularly for those who do not have a suitable HSPC donor available.

269 layed greater losses of DNA methylation than HSPCs singly deficient for Tet2 or Dnmt3a alone, potenti

270 alance of drift and selection imposed by the HSPC population size, and the mutation-selection balance

271 bioinformatics approach, which connects the HSPC gene expression data with the candidate cargo in st

272 A cluster analysis of the HSPC lineage output highlighted the existence of several

273 dingly to provide a rigorous analysis of the HSPC lineage output.

274 exercise on leukocyte production and on the HSPC epigenome and transcriptome persists for several we

275 ECKO)) of young mice and observed that their HSPCs displayed attributes of an aged hematopoietic syst

276 ients with diabetes on pioglitazone therapy, HSPC mobilization after G-CSF was partially rescued.

277 This HSPC gene therapy approach has potential for clinical tr

278 utant p53 confers a competitive advantage to HSPCs following transplantation and promotes HSPC expans

279 se genes was upregulated in LSCs relative to HSPCs; this subset of genes constitutes "LSC-specific" g

280 ntity of endothelial cells that give rise to HSPCs is unknown.

281 ematopoietic cell transition, giving rise to HSPCs that accumulate in intra-arterial clusters (IAC) b

282 Transduced HSPCs homed back to the bone marrow, where they persiste

283 ftment into immunodeficient mice, transduced HSPCs give rise to human myeloid leukemia, whereas untra

284 rial transplantations, exposure of wild-type HSPCs to an mTOR(ECKO) microenvironment was sufficient t

285 ocyte-associated genes compared to wild-type HSPCs, and we provide early validation of G6B as a poten

286 l killer cells) in patients having undergone HSPC gene therapy for Wiskott-Aldrich syndrome or beta h

287 opment in vivo is critical for understanding HSPC expansion, which will have a positive impact in reg

288 human myeloid leukemia, whereas untransduced HSPCs give rise to human immune cells in the same mice.

289 py and discuss emerging strategies for using HSPC gene therapy for a range of diseases.

290 g EV-derived candidate regulators of ex vivo HSPC expansion.

291 h high-risk germ-line mutations, the in vivo HSPC gene therapy approach is a promising strategy that

292 We have developed a simple in vivo HSPC gene therapy approach that involves HSPC mobilizati

293 In vivo HSPC transduction with a GFP-expressing vector and subse

294 In in vivo HSPC-transduced mice with implanted mouse mammary carcin

295 d secondary transplantation of corrected WAS HSPCs into immunodeficient mice showed persistence of ed

296 Delivery of the editing reagents to WAS HSPCs led to full rescue of WASp expression and correcti

297 egulation system that is activated only when HSPCs are recruited to and differentiated by the tumor.

298 sitive arterial endothelial cells from which HSPCs emerge.

299 f TNT formation and function in vitro, while HSPC transplantation into cystinotic mice provides a com

300 BMEC niche and HSPCs, which instructs young HSPCs to behave as aged HSPCs.