コーパス検索結果 (１語後でソート)
通し番号をクリックするとPubMedの該当ページを表示します
1 Sickle cell disease (SCD) is a complex hemoglobinopathy.
2 rtunity for therapeutic intervention of beta-hemoglobinopathy.
3 of sickle cell anemia (SCA), a prototypical hemoglobinopathy.
4 , and sickle cell anemia is a common type of hemoglobinopathy.
5 ll chemicals may provide a novel therapy for hemoglobinopathy.
6 py is to increase fetal hemoglobin and treat hemoglobinopathy.
7 rythropoiesis to the pathophysiology of this hemoglobinopathy.
8 O processing may characterize a new class of hemoglobinopathy.
9 e ongoing evaluation and treatment of sickle hemoglobinopathies.
10 tal clinical importance for the treatment of hemoglobinopathies.
11 in patients with end-stage organ disease or hemoglobinopathies.
12 lowing therapeutic application for some beta-hemoglobinopathies.
13 express globin at sufficient levels to treat hemoglobinopathies.
14 survival after HCT, except for patients with hemoglobinopathies.
15 c (epsilon) genes in individuals with severe hemoglobinopathies.
16 our combined strategy for patients with beta-hemoglobinopathies.
17 g as the first CRISPR-based therapy for beta-hemoglobinopathies.
18 mising therapeutic approach to induce HbF in hemoglobinopathies.
19 myelogenous leukemia (AML) and possibly beta-hemoglobinopathies.
20 pproaches to reactivate these genes for beta-hemoglobinopathies.
21 reactivation is a promising therapy for beta-hemoglobinopathies.
22 in variants and all types of thalassemia and hemoglobinopathies.
23 man primate lentiviral gene therapy model of hemoglobinopathies.
24 synthesis opens up therapeutic targets for B-hemoglobinopathies.
25 e editing approach for the treatment of beta-hemoglobinopathies.
26 el pharmaceutical strategy for treating beta-hemoglobinopathies.
27 ay represent a new gene therapy approach for hemoglobinopathies.
28 e control with relevance to the treatment of hemoglobinopathies.
29 offer a new therapeutic avenue to treat beta-hemoglobinopathies.
30 kle globin expression as a treatment of beta-hemoglobinopathies.
31 a validated therapeutic target for the beta-hemoglobinopathies.
32 sease and other compound heterozygous sickle hemoglobinopathies.
33 s in the red cells of patients with unstable hemoglobinopathies.
34 lkylator regimens for MHC-mismatched BMT for hemoglobinopathies.
35 ential as a treatment for patients with beta-hemoglobinopathies.
36 for genome-editing-mediated therapy of beta-hemoglobinopathies.
37 for Wiskott-Aldrich syndrome (WAS) and beta-hemoglobinopathies.
38 for therapeutic benefit in treating the beta-hemoglobinopathies.
39 on complication in patients with sickle cell hemoglobinopathies.
40 or therapeutic targeting in the treatment of hemoglobinopathies.
41 ng epigenetic approach for treatment of beta-hemoglobinopathies.
42 ght offer new opportunities for treatment of hemoglobinopathies.
43 in variants and all types of thalassemia and hemoglobinopathies.
44 therapy for Wiskott-Aldrich syndrome or beta hemoglobinopathies.
45 n understanding of phenotypic variability in hemoglobinopathies.
46 KLF1 mutations can play a modulatory role in hemoglobinopathies.
47 therapeutic genome engineering for the beta-hemoglobinopathies.
48 for therapeutic targeting of BCL11A in beta-hemoglobinopathies.
49 ll trait but may be confounded by concurrent hemoglobinopathies.
50 nology holds vast promises for a cure to the hemoglobinopathies.
51 re higher levels of corrected cells, such as hemoglobinopathies.
52 for several hematopoietic diseases including hemoglobinopathies.
53 s for fetal hemoglobin induction in the beta-hemoglobinopathies.
54 promise for improved treatment of the major hemoglobinopathies.
55 successful clinical outcome in patients with hemoglobinopathies.
56 goal of translational research aimed toward hemoglobinopathies.
57 domide as an innovative new therapy for beta-hemoglobinopathies.
58 ifier of fetal hemoglobin levels in the beta hemoglobinopathies.
59 f stem cell-based gene therapy in the severe hemoglobinopathies.
60 merged as a potential therapeutic target for hemoglobinopathies.
61 otential clinical trials of gene therapy for hemoglobinopathies.
62 lopment of new treatment rationales for beta hemoglobinopathies.
63 c goal for patients with beta-thalassemia or hemoglobinopathies.
64 t the efficacy of gene therapy in the severe hemoglobinopathies.
65 are needed for optimal treatment of the beta-hemoglobinopathies.
66 els have proven benefit for people with beta-hemoglobinopathies, all current HbF-inducing agents have
67 It is estimated that 10% of patients with hemoglobinopathies and 0.5% of patients with HIV infecti
68 can enhance HbF induction for treating beta-hemoglobinopathies and could be used as a model to simul
69 d may have direct implications to alpha/beta hemoglobinopathies and design of oxidatively stable Hb-b
71 success of hematopoietic transplantation for hemoglobinopathies and hematological malignancies has be
73 al target for therapeutic genome editing for hemoglobinopathies and highlight the power of chromosome
74 nancies, marrow failure, immunodeficiencies, hemoglobinopathies and inherited metabolic diseases.
77 vides explanations of the pathophysiology of hemoglobinopathies and other disease states associated w
78 and unpublished genetic variation related to hemoglobinopathies and thalassemia and implemented micro
79 d epsilon globin in individuals with defined hemoglobinopathies and thalassemias, would serve as a ph
81 management of iron toxicity in patients with hemoglobinopathies and transfusion-dependent anemias and
82 tically beneficial for treatment of the beta hemoglobinopathies and useful for the oral treatment of
83 encompass the clear interaction between this hemoglobinopathy and both malarial and nonmalarial infec
84 Plt12 mouse is a model of high O(2)-affinity hemoglobinopathy and provides insights into hematopoiesi
85 potential for the treatment of malignancies, hemoglobinopathies, and autoimmune diseases, as well as
86 licting RBCs, including bone marrow failure, hemoglobinopathies, and malaria, and also preclinical te
87 zed that sickle cell disease (SCD) and other hemoglobinopathies are associated with a state of chroni
88 rld's most common monogenic disorders, the B-hemoglobinopathies are at the forefront of bringing geno
93 Many gene editing efforts to treat the beta-hemoglobinopathies attempt to correct beta-globin mutati
95 ely to benefit from new advances include the hemoglobinopathies (beta-thalassemia and sickle cell dis
96 s effectively used in the management of beta-hemoglobinopathies by augmenting the production of fetal
97 nce of fetal hemoglobin (HPFH) ameliorates B-hemoglobinopathies by inhibiting the developmental switc
99 ing EBR/GZR and placebo; among patients with hemoglobinopathies, change in mean hemoglobin levels was
100 PNG), numerous blood group polymorphisms and hemoglobinopathies characterize the human population.
101 Sickle cell disease (SCD) is a hereditary hemoglobinopathy characterized by painful vaso-occlusive
102 of celiac disease and in at least 1 case of hemoglobinopathy, characterized by shortened erythrocyte
103 T) offers curative therapy for patients with hemoglobinopathies, congenital immunodeficiencies, and o
104 population raises the possibility that these hemoglobinopathies contribute to a decline in kidney fun
105 dult animal model for the most severe of the hemoglobinopathies, Cooley anemia, which should prove us
106 who are hemodynamically stable and without a hemoglobinopathy, cyanotic cardiac condition, or severe
108 y is limited among patients with sickle cell hemoglobinopathy despite guidelines recommending dilated
109 Hospital of Essen summarizes the results of hemoglobinopathies diagnosed between August 2018 and Sep
110 tion, cancer, and genetic diseases including hemoglobinopathies, Duchenne muscular dystrophy (DMD), h
111 ting presents an effective strategy for beta-hemoglobinopathies, enabling durable HbF reactivation wi
112 th electrophoretically confirmed sickle cell hemoglobinopathies followed by the University of Illinoi
113 te understanding of the genetics of the beta-hemoglobinopathies for several decades, definitive treat
114 f oncoretroviral vectors in gene therapy for hemoglobinopathies has been impeded by low titer vectors
115 Progress toward gene therapy of beta-chain hemoglobinopathies has been limited in part by poor expr
116 ce fetal globin (HbF) for patients with beta-hemoglobinopathies has the potential to be a curative st
117 based vector system in gene therapy of human hemoglobinopathies in general and sickle-cell anemia and
118 ove useful in nonmalignant disorders such as hemoglobinopathies in which moderate levels of donor chi
119 hways are used in beta-thalassemia, a common hemoglobinopathy in which beta-globin gene mutations cau
120 d to antagonize EKLF function in adults with hemoglobinopathy, in an attempt to reactivate gamma-glob
121 in targeting BCL11A as a treatment for beta-hemoglobinopathies, including sickle cell disease (SCD)
122 his consensus statement does not cover other hemoglobinopathies, including thalassemia intermedia and
123 r important causes of anemia in children are hemoglobinopathies, infection, and other chronic disease
124 sociated with splenectomy, and patients with hemoglobinopathies is a possible consequence of increase
126 ry challenge in lentiviral gene therapy of B-hemoglobinopathies is to maintain low vector copy number
129 ) globin gene expression for therapy of beta-hemoglobinopathies likely requires local chromatin modif
131 moglobin-based blood substitute therapy, the hemoglobinopathies, malaria, and other acquired and gene
132 y RNFL thinning in patients with sickle cell hemoglobinopathies occurred faster in patients with a hi
133 viduals with beta-thalassemia intermedia and hemoglobinopathies of equivalent severity who are infreq
134 Mutations in the adult form cause inherited hemoglobinopathies or globin disorders, including sickle
135 of age, without evidence of iron deficiency, hemoglobinopathy, or chronic inflammation, found an aver
136 c study conducted at the Pediatric and Adult Hemoglobinopathy Outpatient Units of the University Hosp
140 in (HbF) synthesis for the treatment of beta-hemoglobinopathies probably involve protein modification
141 of Plasmodium falciparum suggests that this hemoglobinopathy provides a selective advantage against
142 included nondiabetic adults with sickle cell hemoglobinopathy receiving care from a Wilmer Eye Instit
143 at 8 weeks after dosing) and editing of two hemoglobinopathy-relevant loci, BCL11A and HBG1/2 (26% a
144 ell polymorphism, ABO blood group, and other hemoglobinopathies remain the few major determinants in
149 globin SS = 184; and 116 with other sickling hemoglobinopathies: SC, SD, and S-beta thalassemia); alb
155 n (HbF) ameliorates the clinical severity of hemoglobinopathies such as beta-thalassemia and sickle c
156 lls (ECs) may be pathologically important in hemoglobinopathies such as sickle cell disease and thala
158 cal trials have been successfully applied to hemoglobinopathies, such as sickle cell disease (SCD) an
159 to ameliorate symptoms of co-inherited beta-hemoglobinopathies, such as sickle cell disease and beta
161 in patients with iron overload due to inborn hemoglobinopathies, suggesting an inverted Vdelta2+/Vdel
163 nes may offer therapeutic approaches for the hemoglobinopathies, the most common single gene disorder
165 to fetal hemoglobin (HbF) induction for beta-hemoglobinopathy therapy, though heterogeneity in edit a
166 pedigree is the absence of any cosegregating hemoglobinopathy, thus allowing observation of the segre
167 United States to determine the prevalence of hemoglobinopathy traits and quantify their influence on
169 the influence of sickle cell trait and other hemoglobinopathy traits on anemia in dialysis patients h
174 photographs from 190 adults with sickle cell hemoglobinopathy were independently graded by 2 masked r
175 ingly carried alleles for G6PD deficiency or hemoglobinopathies, which were associated with character
177 s photographs from patients with sickle cell hemoglobinopathy, with potential applications for improv
178 e (SCD) is one of the most common hereditary hemoglobinopathies worldwide, affecting almost 400,000 n