about
Human induced pluripotent stem cells for monogenic disease modelling and therapyThe therapeutic potential of genome editing for β-thalassemiaTowards in vivo amplification: Overcoming hurdles in the use of hematopoietic stem cells in transplantation and gene therapy5p deletions: Current knowledge and future directions.The potential of gene therapy approaches for the treatment of hemoglobinopathies: achievements and challengesComparative genomics of canine hemoglobin genes reveals primacy of beta subunit delta in adult carnivores.Clinical Effect and Mechanism of Yisui Shengxue Granules in Thalassemia Patients with Mild, Moderate, or Severe Anemia.Quality of life, clinical effectiveness, and satisfaction in patients with beta thalassemia major and sickle cell anemia receiving deferasirox chelation therapy.A validated cellular biobank for β-thalassemia.One-Step Biallelic and Scarless Correction of a β-Thalassemia Mutation in Patient-Specific iPSCs without Drug Selection.Combining Single Strand Oligodeoxynucleotides and CRISPR/Cas9 to Correct Gene Mutations in β-Thalassemia-induced Pluripotent Stem Cells.CRISPR-Cas9 technology and its application in haematological disorders.Eliminating HIV-1 Packaging Sequences from Lentiviral Vector Proviruses Enhances Safety and Expedites Gene Transfer for Gene Therapy.Lentiviral Transfer of γ-Globin with Fusion Gene NUP98-HOXA10HD Expands Hematopoietic Stem Cells and Ameliorates Murine β-Thalassemia.Ground state naïve pluripotent stem cells and CRISPR/Cas9 gene correction for β-thalassemia.Development and characterization of K562 cell clones expressing BCL11A-XL: Decreased hemoglobin production with fetal hemoglobin inducers and its rescue with mithramycin.BCL11A mRNA Targeting by miR-210: A Possible Network Regulating γ-Globin Gene Expression.Rapid and Sensitive Assessment of Globin Chains for Gene and Cell Therapy of Hemoglobinopathies.Orphan Drugs and Potential Novel Approaches for Therapies of β-Thalassemia: Current Status and Future Expectations
P2860
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P2860
description
2015 nî lūn-bûn
@nan
2015年の論文
@ja
2015年学术文章
@wuu
2015年学术文章
@zh-cn
2015年学术文章
@zh-hans
2015年学术文章
@zh-my
2015年学术文章
@zh-sg
2015年學術文章
@yue
2015年學術文章
@zh
2015年學術文章
@zh-hant
name
Recent trends in the gene therapy of β-thalassemia.
@en
type
label
Recent trends in the gene therapy of β-thalassemia.
@en
prefLabel
Recent trends in the gene therapy of β-thalassemia.
@en
P2860
P50
P356
P1476
Recent trends in the gene therapy of β-thalassemia.
@en
P2093
Marina Kleanthous
Roberto Gambari
P2860
P356
10.2147/JBM.S46256
P577
2015-02-19T00:00:00Z