ライフサイエンスコーパス: SCID

1 SCID (severe combined immunodeficiency) mice underwent l

2 SCID disorders are split into groups based on their pres

3 SCID has been associated with impaired purine nucleotide

4 SCID mice were also more insulin sensitive with increase

5 SCID mice with human HCT116 cancer xenografts were image

6 SCID mice with human PC3 prostate cancer xenografts (Gro

7 SCID mice, which lack an adaptive immune system due to t

8 SCID or RAG1-deficient mice, which lack T and B cells, d

9 SCID was detected in a newborn before the onset of infec

10 ional young adult Arabian-pony crosses and 1 SCID foal were then inoculated with plasma containing on

11 etic stem cell transplantation (HSCT) and 14 SCID-X1 patients treated with gene therapy over the same

12 n a mouse model of chronic infection, 5 of 6 SCID/beige mice (83.3%) were cured after treatment with

13 tivity (IC50 = 10 nM) and oral activity in a SCID mouse model of Pf infection with an ED50 of 100 and

14 progenitor T cells, which is in line with a SCID phenotype at the level of early T cell development

15 dynamics and diversity in a cohort of 15 ADA-SCID children treated with gammaretroviral vectors and f

16 dicines Agency approved gene therapy for ADA-SCID patients without a suitable bone marrow donor.

17 icient severe combined immunodeficiency (ADA-SCID) patients have been treated with 4 distinct gammare

18 ausing severe combined immunodeficiency (ADA-SCID), often referred to as the "bubble boy" disease.

19 ta, in combination with results of other ADA-SCID gene therapy trials, suggest that disease backgroun

20 atment of choice for ADA-deficient SCID (ADA-SCID) is hematopoietic stem cell transplant from an HLA-

21 of gene therapy (GT) in 18 patients with ADA-SCID for whom an HLA-identical family donor was not avai

22 Eight additional SCID mice bearing HCT116 xenografts in dorsal skinfold w

23 In an SCID mouse xenograft model, low-dose metronomic paclitax

24 cles (TRECs) began in Wisconsin in 2008, and SCID was added to the national recommended uniform panel

25 further extended to in vivo Swiss albino and SCID mice models also revalidated the anti-carcinogenic

26 is can lead to both megaloblastic anemia and SCID in MTHFD1 deficiency.

27 ablish infection in both immunocompetent and SCID mice and has been proposed to facilitate evasion of

28 odeficiency (scid) mutation (SCID) mice, and SCID bearing a null mutation in the IL-2 common gamma ch

29 rangements in mice transplanted with Artemis-SCID cells.

30 In human artery-SCID chimeras, PD-1 blockade exacerbated vascular inflam

31 g a group of disorders collectively known as SCID.

32 sponses to Candida require an intact TCR, as SCID, IL-7Ralpha(-/-), and Rag1(-/-) mice were susceptib

33 TEMIS) have been described to cause atypical SCID, Omenn syndrome, Hyper IgM syndrome and inflammator

34 ned immunodeficiencies (CIDs) and "atypical" SCID show reduced, not absent T-cell immunity.

35 of 51 had a genetic diagnosis of "atypical" SCID and 14 of 51 of CID.

36 ing immunocompetent hosts into tumor-bearing SCID-NOD immunocompromised mice attenuated tumor growth

37 CB17 SCID mice infected with R. typhi(GFPuv) succumb to the i

38 In the spleen and liver of infected CB17 SCID mice, the bacteria are detectable by immunofluoresc

39 and immune reconstitution in 13 consecutive SCID-X1 patients having undergone haploidentical hematop

40 d is not informative for adenosine deaminase-SCID, whereas hypomorphic mutations lead to less severe

41 s such as severe combined immune deficiency (SCID) and X-linked agammaglobulinemia (XLA); however, ga

42 including severe combined immune deficiency (SCID), autoimmunity, and inflammation.

43 including severe combined immune deficiency (SCID), Wiskott-Aldrich syndrome (WAS), and chronic granu

44 X-linked severe combined immune deficiency (SCID-X1) lacking a matched sibling donor may have better

45 The treatment of choice for ADA-deficient SCID (ADA-SCID) is hematopoietic stem cell transplant fr

46 Ten subjects with confirmed ADA-deficient SCID and no available matched sibling or family donor we

47 efficacy from gene therapy for ADA-deficient SCID, with an excellent clinical safety profile.

48 with mutations in DCLRE1C (Artemis-deficient SCID), there is no optimal approach that uses standard d

49 treatment for adenosine deaminase-deficient SCID (n = 2).

50 in both severe combined immunity-deficient (SCID) and muMT mice indicates that peritoneal B cells al

51 denosine deaminase-deficient (ADA-deficient) SCID when combined with reduced intensity conditioning (

52 In streptozotocin-induced diabetic SCID/beige mice, the injection of 750 rat islet equivale

53 njected intrarectally into nonobese diabetic/SCID mice.

54 lop a uniform set of criteria for diagnosing SCID and related disorders and has evaluated the results

55 n screening has been effective in diagnosing SCID patients early in life, there is an urgent need to

56 of severe combined immunodeficiency disease (SCID) and related disorders varies among institutions an

57 uctured Clinical Interview for DSM disorder (SCID).

58 reduced in trigenic mice (Tie2(cre)/Osx(f/f)/SCID) with endothelial-specific deletion of osteoblast c

59 ant OSX compared with bigenic mice (Osx(f/f)/SCID).

60 nts leading to efficacy in the P. falciparum SCID mouse malaria model.

61 a undetectable in vivo using a P. falciparum SCID mouse model.

62 ID during 2000-2009, diagnostic criteria for SCID, and the pilot project of newborn screening for SCI

63 Additional interventions for SCID and non-SCID T-cell lymphopenia included immunoglob

64 dy of infants identified by means of NBS for SCID who received care at the University of California,

65 Newborn screening (NBS) for SCID permits identification of affected infants before d

66 ing (NGS)-based multigene-targeted panel for SCID and other severe PIDDs requiring rapid therapeutic

67 ore than 800 subjects on PIDTC protocols for SCID, and enrollment in the studies on WAS and CGD is un

68 d the pilot project of newborn screening for SCID in the Navajo Nation.

69 to pose the greatest threat to survival for SCID patients.

70 yme replacement therapy, or gene therapy for SCID and related disorders.

71 nfirmed over the long term, gene therapy for SCID-X1 appears to be an equal, if not superior, alterna

72 e development of autologous cell therapy for SCID-X1 subjects.

73 es of hematopoietic cell transplantation for SCID during 2000-2009, diagnostic criteria for SCID, and

74 BM samples from SCID patients with IL-2RG (n = 3) or JAK3 deficiency (n

75 ementary determining region 3 sequences from SCID and OS iPSC-derived cells, whereas control iPSCs yi

76 ins give rise to a phenotypic spectrum, from SCID to extreme growth failure, with deficiencies in cer

77 he in vivo prostate regeneration assay, host SCID mice carrying Src(Y529F)-transduced regeneration ti

78 geted to generate a zebrafish model of human SCID.

79 tment of a previously unknown cause of human SCID.

80 Moreover, in a humanized SCID mouse model, CD19(+) CD5(-) B cells were more effec

81 In the humanized SCID mouse, local injection of Netrin-1 into skin enhanc

82 ctober 2016, 32 patients with NBS-identified SCID and leaky SCID from California and other states wer

83 group of severe combined immunodeficiencies (SCID) is characterized by lack of T and B cells and is c

84 esent with severe combined immunodeficiency (SCID) and cellular radiosensitivity, but hypomorphic mut

85 Severe combined immunodeficiency (SCID) can be cured by using allogeneic hematopoietic ste

86 results in severe combined immunodeficiency (SCID) caused by a complete lack of T and B lymphocytes.

87 clusion of severe combined immunodeficiency (SCID) in a Europe-wide screening program is currently de

88 re form of severe combined immunodeficiency (SCID) in humans.

89 Severe combined immunodeficiency (SCID) is characterized by arrested T-lymphocyte producti

90 Severe combined immunodeficiency (SCID) is characterized by severely impaired T-cell devel

91 cytes into severe combined immunodeficiency (SCID) mice.

92 fts in the severe combined immunodeficiency (SCID) mouse model of VZV pathogenesis, and observed that

93 Severe combined immunodeficiency (SCID) represents congenital disorders characterized by a

94 ldren with severe combined immunodeficiency (SCID) to a prospective natural history study.

95 eening for severe combined immunodeficiency (SCID) using assays to detect T-cell receptor excision ci

96 Severe combined immunodeficiency (SCID) with a complete absence of T cells was observed in

97 iated with severe combined immunodeficiency (SCID), consistent with the requirement for NHEJ during V

98 mice with severe combined immunodeficiency (SCID), human pluripotent stem cell-derived (PSC-derived)

99 vere form, severe combined immunodeficiency (SCID), presents with profound deficiencies of T cells, B

100 esent with severe combined immunodeficiency (SCID)-like disease.

101 terized by severe combined immunodeficiency (SCID).

102 t model of severe combined immunodeficiency (SCID).

103 nosed with severe combined immunodeficiency (SCID).

104 ross) with severe combined immunodeficiency (SCID).

105 senting as severe combined immunodeficiency (SCID).

106 -sensitive severe combined immunodeficiency (SCID).

107 ients with severe combined immunodeficiency (SCID).

108 ients with severe combined immunodeficiency (SCID).

109 L2RG)/JAK3 severe combined immunodeficiency (SCID).

110 X-linked severe combined immunodeficiency (SCID-X1) caused by mutations in interleukin 2 receptor g

111 X-linked Severe Combined Immunodeficiency (SCID-X1) is a genetic disease that leaves newborns at hi

112 e X-linked severe combined immunodeficiency (SCID-X1).

113 h X-linked severe combined immunodeficiency (SCID-X1).

114 completely abolished in the immunodeficient SCID/beige (bg) variant.

115 ferred into severe combined immunodeficient (SCID) mice to induce ITP.

116 Severe combined immunodeficient (SCID) mice were infected and treated with sulfadiazine t

117 mor-bearing severe combined immunodeficient (SCID) mice.

118 the lung of severe combined immunodeficient (SCID) mice.

119 utants from severe combined immunodeficient (SCID) patient cells showed a failure to sustain progress

120 vivo, in a severe combined immunodeficient (SCID)/beige mouse host.

121 r using tail vein injection of A549 cells in SCID mice.

122 s derived from HCT-116 and NCI-H460 cells in SCID mouse models.

123 n increased frequency of immature T cells in SCID pigs.

124 delta locus rearrangements, were detected in SCID and OS-derived T-lineage cells, consistent with a p

125 DeltagD(-/+gD1) elicited no disease in SCID mice, whereas 1000-fold lower doses of wild-type vi

126 es employing cell-sorted skin equivalents in SCID/NOD mice demonstrated enhanced transepidermal water

127 ne resulting in a 3-fold greater exposure in SCID mice.

128 ive techniques to measure kidney function in SCID mice with adriamycin-induced nephropathy.

129 Furthermore, in SCID mice transplanted with neural progenitors derived f

130 Hypothyroidism was generated in SCID-beige mice using an iodine-deficient diet containin

131 a dramatic reduction of RCC tumor growth in SCID mice in vivo.

132 hagocytes at the skin site of inoculation in SCID and C3H/HeN mice allowed the ospC mutant to establi

133 ue culture infective dose) of MARV/Ang-MA in SCID mice, and i.p. infection at a dose of 1,000x LD50 r

134 injecting 5.5 MBq of 99mTc-anti-CD56 mAb in SCID mice bearing ARO tumor xenografts in the right thig

135 death between 6 and 8 days postinfection in SCID mice.

136 We determined that Bxv1 was also present in SCID mice that were used for in vivo propagation of Endo

137 e were able to enhance anti-TB protection in SCID mice, and the transfer of vaccine-primed B cells al

138 tation elicited a blunted immune response in SCID/bg mice, as demonstrated by macrophage number and m

139 ing mutations in human IL-2Rgammac result in SCID, a primary immunodeficiency characterized by greatl

140 ti-CD56 and to image human NK trafficking in SCID mice bearing human cancer.

141 bcutaneous model of colon (HCT-116) tumor in SCID-bg mice showed that the activity of 1 and 2 signifi

142 MFD-1 was tumorigenic in SCID mouse and proliferative and invasive in 3D cultures

143 ibited peritoneal dissemination of tumors in SCID mice, leading to improved tumor-free survival in a

144 oduce estrogen-dependent xenograft tumors in SCID mice, we also observed lower ERalpha protein levels

145 growth of a PDA cell line as a xenograft in SCID mice, we also show that a slightly pathogenic avian

146 C infection model in human gut xenografts in SCID mice and used it to study the role of T3SS in the p

147 lop lentiviral gene therapy for RAG1-induced SCID and to test its safety.

148 uccessfully treat patients with RAG1-induced SCID while maintaining low vector copy numbers and minim

149 each type per heart) was tested in infarcted SCID (severe combined immunodeficiency)-Beige mice.

150 on during HCV infection because HCV-infected SCID/Alb-uPA mice accumulated higher plasma ketones whil

151 detected on cerebral vessels of the infected SCID and wild-type animals.

152 passive transfer of immune sera to infected SCID mice.

153 3.5 months post-implantation into SCID mice, the micro-computed tomography imaging showed

154 ine-primed T cells from Jh(-/-) KO mice into SCID mice only provided suboptimal protection.

155 ism, and quality of life (QoL) of IL2RG/JAK3 SCID patients >2 years post-HSCT at our center.

156 IL2RG/JAK3 SCID survivors free from immunoglobulin replacement have

157 In both IL-2RG- and JAK3-SCID patients, the early stages of lymphoid commitment f

158 patients with NBS-identified SCID and leaky SCID from California and other states were treated, and

159 al SCID; 13% were classified as having leaky SCID, Omenn syndrome, or reticular dysgenesis; and 3% ha

160 /S723C mice, which serve as a model of leaky SCID, with agonists of the virus-recognizing receptors T

161 mutations can cause milder phenotypes (leaky SCID).

162 ing detected 52 cases of typical SCID, leaky SCID, and Omenn syndrome, affecting 1 in 58,000 infants

163 including the diagnosis of typical vs leaky SCID/Omenn syndrome, diagnosis via family history or new

164 Most of the patients with leaky SCID were compound heterozygous for 1 loss-of-function a

165 patients with typical SCID and 32 with leaky SCID, Omenn syndrome, or reticular dysgenesis.

166 proteins and are often associated with leaky SCID.

167 The infant had "leaky" SCID (i.e., a form of SCID in which a minimal degree of

168 Importantly, reconstitution of NOD/Lt-SCID/gammac(-/-) (NSG) mice with BaEV-LV-transduced hCD3

169 .071 +/- 0.035 mm, SCID: 0.137 +/- 0.032 mm, SCID/bg: 0.804 +/- 0.039 mm; P < 0.001).

170 ge luminal diameter, WT: 0.071 +/- 0.035 mm, SCID: 0.137 +/- 0.032 mm, SCID/bg: 0.804 +/- 0.039 mm; P

171 d immunodeficiency caused by ILR2G mutation (SCID-X1) despite the occurrence of genotoxicity caused b

172 e combined immunodeficiency (scid) mutation (SCID) mice, and SCID bearing a null mutation in the IL-2

173 In newborn SCID-X1 dogs, injection of a foamy virus vector expressi

174 in the Artemis gene that cause T(-)B(-)NK(+) SCID in pigs.

175 y were shown to be affected by T(-)B(-)NK(+) SCID, representing, to our knowledge, the first example

176 NK(-)SCID disorders are highly permissive for donor T-cell en

177 Infants with NK(-)SCID were more likely to survive than NK(+) recipients (

178 l administration of CLH001 to BALB/c and NOD SCID gamma (NSG) mice resulted in complete survival with

179 g cells were adoptively transferred into NOD SCID gammaC-deficient mice, which were given isotype or

180 When injected into NOD SCID mice, control GFP NCM-1 cells fail to grow whereas

181 ly increased the survival of PEL bearing NOD-SCID mice in an orthotopic xenograft model as compared w

182 ized mouse model in which male or female NOD-SCID-beta2m(-/-) were transplanted with human progenitor

183 abetic/severe combined immunodeficiency (NOD-SCID) mice resulted in the formation of microvessels der

184 burden in BLCL-engrafted immunodeficient NOD-SCID/Il2rg(-/-) mice.

185 reventing metastasis and angiogenesis in NOD-SCID mice, while being non-toxic in vivo.

186 human diffuse large B lymphoma model in NOD-SCID mice.

187 ved tumors and xenografts established in NOD-SCID or nude mice, low MCPIP1 levels correlated strongly

188 and in their capacity to metastasize in NOD-SCID-Il2rg(-/-) (NSG) mice.

189 s to high titers in human lung grafts in NOD-SCID/gamma mice, resulting in a robust inflammatory resp

190 estigated by using hu-spl-PBMC-NSG mice, NOD-SCID-IL2rgamma(-/-) (NSG) mice intrasplenically injected

191 firmed in the adoptive transfer model of NOD-SCID mice where tolDCs delayed diabetes onset.

192 h as NOD-Rag1-/-IL2RgammaC-null (NRG) or NOD-SCID-IL2RgammaC-null (NSG) mice are critical for efficie

193 Here we made use of the NOD-SCID-IL-2Rgamma(-/-) xenograft model and lentiviral cell

194 nd Srsf10, but not Ptbp2, in the PLNs of NOD.SCID mice.

195 interleukin-3, and stem cell factor in a NOD/SCID-IL2Rgamma(null) background (NSGS mice), we demonstr

196 the radiation-depleted bone marrow of a NOD/SCID/IL2rg(-/-) (NSG) mouse on which a patient's tumor i

197 Using ICG-001 in a NOD/SCID/IL2Rgamma(-/-) mouse model of engrafted human chron

198 ent protein were transplanted into adult NOD/SCID mice with acute left anterior descending artery lig

199 Human immune system (HIS) BLT-NOD/SCID mice were inoculated intravenously with a low-passa

200 ells maintained their ability to engraft NOD/SCID/IL2rgamma(null) mice and to produce cells from mult

201 ) (NSIN) mice by knocking out Foxn1 from NOD/SCID/IL2rg(-/-) (NSI) mice using the CRISPR/Cas9 system.

202 a P. falciparum infection in a humanized NOD/SCID mouse model system.

203 abetic/severe combined immunodeficiency (NOD/SCID) mice with partially reconstituted immune systems w

204 abetic severe combined immunodeficiency (NOD/SCID) mice.

205 and transplantation into immunodeficient NOD/SCID/interleukin 2 receptor gamma chain null mice.

206 y formation and orthotopic GBM growth in NOD/SCID mice and decelerates the progression of low-grade a

207 Depletion of NK cells in NOD/SCID mice enabled combined systemic and CNS leukemia of

208 pheral blood mononuclear cells (PBMC) in NOD/SCID mice harboring xenografts of MDA-MB-231, a triple-n

209 Death occurred later in NOD/SCID mice receiving REH cells depleted of CD9 for transp

210 Treatment of GBM xenografts in NOD/SCID mice with NK cells from a KIR2DS2(+) donor lacking

211 2 knockdown invasive MDA-MB-231 cells in NOD/SCID mice, and compared parental and bone-derived varian

212 te that the injection of TLR7 agonist in NOD/SCID mice, in C57BL/6 wild-type, and TLR7-deficient mice

213 potent in vivo antileukemic activity in NOD/SCID mouse xenograft models of relapsed and chemotherapy

214 mutant ER-expressing tumor xenografts in NOD/SCID-gamma mice after oral or subcutaneous administratio

215 engrafted at a markedly higher level in NOD/SCID/IL-2 receptor gamma chain-null (NSG) mice compared

216 significant delay in AML progression in NOD/SCID/IL2Rg(null) mice, but the persistence of adoptively

217 y assays as well as adoptive transfer in NOD/SCID/IL2Rgamma mice were used to assess for pathogen-spe

218 Interestingly, NOD/SCID mice, which have a deficiency in T, B, and NK cells

219 disease (GvHD) model of humanized mice (NOD/SCID/IL-2Rgammac(-/-) [NSG] mice).

220 udy, we generated a novel strain of nude NOD/SCID/IL2rg(-/-) (NSIN) mice by knocking out Foxn1 from N

221 d significantly prolongs the survival of NOD/SCID mice engrafted with primary ALL.

222 cardiotoxin-injured skeletal muscles of NOD/SCID mice reveals survival and engraftment of the donor

223 astatic nodule formation in the lungs of NOD/SCID mice.

224 In particular, NOD/SCID/IL2rg(-/-) mice can support the growth of various t

225 tive T cells in HIV-infected brain using NOD/SCID/IL-2rcgamma(-/-) mice reconstituted with human PBMC

226 en human B-cell lymphoma in a xenogeneic NOD/SCID/IL2rg(null) mouse model.

227 Additional interventions for SCID and non-SCID T-cell lymphopenia included immunoglobulin infusion

228 ted, and 42 patients with NBS-identified non-SCID T-cell lymphopenia were followed.

229 ces influenced the rates of detection of non-SCID T-cell lymphopenia.

230 The usefulness of detection of non-SCID T-cell lymphopenias by the same screening remains t

231 ore sensitive to ionizing radiation than non-SCID piglets, eliminating the RAG1 and RAG2 genes.

232 cterizes a group of patients with nontypical SCID T-cell deficiencies from a therapeutic perspective.

233 ge, the first example of naturally occurring SCID in pigs.

234 Early detection of SCID could reduce the cost of treatment by euro50,000-10

235 s study compares human T-cell development of SCID vs OS patients, and elucidates important difference

236 Early diagnosis of SCID through population-based screening of newborns can

237 The infant had "leaky" SCID (i.e., a form of SCID in which a minimal degree of immune function is pre

238 e NK(+) (n = 24) and NK(-) (n = 53) forms of SCID.

239 ng experiments were performed on 2 groups of SCID mice inoculated subcutaneously with increasing numb

240 Genetic heterogeneity of SCID frequently delays the diagnosis; a specific diagnos

241 The incidence of SCID is estimated at 1 in 100,000 births.

242 lin treatment resulted in a dramatic loss of SCID-repopulating cells (SRCs), treatment with OKT3 or U

243 n orthotopically implanted into the lungs of SCID-beige mice.

244 and potentially saved by early management of SCID.

245 ted CD34+ cells produced a greater number of SCID-repopulating cells and established multilineage hem

246 were coinjected into the mammary fat pad of SCID mice.

247 egs was also observed upon reconstitution of SCID mice with CD4(+) T cells from CD25 knockout mice (w

248 Rescue of SCID fibroblast radiosensitivity by human Artemis protei

249 results as part of a retrospective study of SCID in North America.

250 n pancreatic tumors and improved survival of SCID beige mice carrying A549 human lung tumors compared

251 tive family history or clinical suspicion of SCID or other severe PIDD identified deleterious mutatio

252 We performed transplantation of SCID CD34(+) bone marrow stem/progenitor cells into an o

253 inactivating retrovirus for the treatment of SCID-X1.

254 development caused by various major types of SCID.

255 of radiolabeled antibody in the tail vein of SCID mice, which were then sacrificed at 1, 3, 6, and 24

256 zed P. falciparum mouse model of malaria (Pf/SCID model).

257 ned if passive transfer of 1E4 would protect SCID mice against C. burnetii aerosol infection.

258 nsitive severe combined immunodeficiency (RS-SCID).

259 cell transplantation for radiation-sensitive SCID suggest that minimizing exposure to alkylating agen

260 rest in thymic development caused by several SCID mutations.

261 The SCID pig can be an important biomedical model, but these

262 rowth was significantly less frequent in the SCID mice treated with anti-CXCL9 serum than in mice tre

263 F invasion into coimplanted cartilage in the SCID mouse model of RA.

264 ZV infection of human skin xenografts in the SCID mouse model of VZV pathogenesis showed both that pC

265 seen after inoculation of human skin in the SCID mouse model or monolayers with higher-titered infec

266 e erythrocytic stage of P. falciparum in the SCID mouse model with an ED90 of 11.7 mg/kg when dosed o

267 ated cartilage invasion were examined in the SCID mouse model.

268 dium berghei malaria model as well as in the SCID mouse P. falciparum model.

269 administered with therapeutic benefit in the SCID-X1 dog, a clinically relevant preclinical model for

270 eved nearly half the patency observed in the SCID/bg mouse (NK Ab: 0.356 +/- 0.151 mm, Asp/Pla: 0.452

271 ted wild-type mice were transferred into the SCID mice in combination with treatment with anti-CXCL9

272 us mutations as the molecular basis for this SCID phenotype.

273 cells are sufficient to transfer TEC H/P to SCID recipients.

274 23(low) B-1a cells upon adoptive transfer to SCID recipients.

275 f in vivo platelet counts in the transferred SCID mice suggesting that anti-CD20 therapy significantl

276 not establish infection in anti-Ly6G-treated SCID and C3H/HeN mice (depletion of neutrophils).

277 ese results open up new avenues for treating SCID-X1 and other diseases.

278 was detected for mutations causing a typical SCID phenotype.

279 ients, 84% were classified as having typical SCID; 13% were classified as having leaky SCID, Omenn sy

280 Screening detected 52 cases of typical SCID, leaky SCID, and Omenn syndrome, affecting 1 in 58,

281 to 2014, including 68 patients with typical SCID and 32 with leaky SCID, Omenn syndrome, or reticula

282 phoma and Hodgkin lymphoma cells and in vivo SCID mouse models.

283 tion caused human multisystem anomalies with SCID and also revealed a prethymic role for BCL11B in he

284 We enrolled nine boys with SCID-X1 in parallel trials in Europe and the United Stat

285 In experiments with SCID mice infected with S. pneumoniae, we found passive

286 Infants with SCID and other diagnoses of T-cell lymphopenia were clas

287 d data retrospectively from 240 infants with SCID who had received transplants at 25 centers during a

288 on protocols for NBS identified infants with SCID, as well as infants with other T-lymphopenic disord

289 hly efficient production of founder NHP with SCID phenotypes, with promises of multiple pre-clinical

290 For treatment costs, patients with SCID admitted to the national reference center for prima

291 assessing the risk of aGVHD in patients with SCID and designing the approach for GVHD prophylaxis.

292 anting CD34(+) stem cells from patients with SCID into a xenograft mouse model provides previously un

293 nal retrospective review of 74 patients with SCID undergoing transplantation between 1988 and 2014.

294 th posttransplantation GVHD in patients with SCID.

295 The proband presented with SCID, megaloblastic anemia, and neurologic abnormalities

296 lls, confirming correction of the cellular X-SCID phenotype.

297 X-linked severe combined immunodeficiency (X-SCID) is an immune disorder caused by mutations in the I

298 ntiation at the T cell progenitor stage in X-SCID cells.

299 To model X-SCID in vitro, we generated a mouse embryonic stem cell

300 y stronger antitumor activity in a xenograft SCID mouse model and depletes B cells in cynomolgus monk