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Zhirui Liu et al.
Clustered regularly interspaced short palindromic repeats (CRISPR) screens represent a transformative force in biological discovery, enabling the unbiased interrogation of gene function in a wide range of applications. Traditional screening...
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Allogeneic hematopoietic stem cell transplantation (allo-HSCT) represents a curative therapy for hematological malignancies, with T-cell immune reconstitution playing a pivotal role in determining clinical outcomes. This review comprehensiv...
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Yuanliang Peng et al.
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Jotaro Yamamoto et al.
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Zhe Chen,Chaojie Wang,Xupeng Chen et al.
Zhe Chen et al.
The yolk sac drives vertebrate embryonic hematopoiesis through primitive hematopoiesis and endothelial-to-hematopoietic transition (EHT) waves. However, dynamic cellular and molecular changes during EHT of the yolk sac remain to be elucidat...
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Chen Zhao,Hai-Xia Fu,Xiao-Dong Mo et al.
Chen Zhao et al.
Chronic graft-versus-host disease (cGVHD) is a major complication of haploidentical hematopoietic stem cell transplantation (haplo-HSCT). In our previous studies, we speculated that cGVHD might be closely related to immune-mediated encephal...
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Ziying Yang,Siqi Liu,Changya Chen
Ziying Yang
Single-cell multiomics technologies have significantly advanced our understanding of cellular heterogeneity and biological complexity. The joint profiling of single-cell RNA sequencing (scRNA-seq) and single-cell ATAC sequencing (scATAC-seq...
Chinese guidance for the clinical application of adeno-associated virus vector-based gene therapy for hemophilia B (2025) [0.03%]
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Feng Xue,Yu Hu,Renchi Yang et al.
Feng Xue et al.
Currently, factor IX replacement is the mainstay treatment for hemophilia B in clinical practice. However, since the disease cannot be cured, lifelong treatment and frequent infusion is required. In recent years, hemophilia B gene therapy h...
G2B: an optimized lentiviral vector with enhanced titer and β-globin expression for improved β-thalassemia gene therapy [0.03%]
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Zhu-Ying Gao,Zhi-Xue Yang,Dong-Hao Deng et al.
Zhu-Ying Gao et al.
Gene therapy using lentiviral vectors offers a promising alternative treatment for β-thalassemia, a common monogenic blood disorder. Although the BB305 vector has demonstrated long-term clinical success-enabling most β-thalassemia patient...