Evidence map›Paper›PMID 41113105›Full record

ArticleBlood science (Baltimore, Md.)2025

G2B: an optimized lentiviral vector with enhanced titer and β-globin expression for improved β-thalassemia gene therapy.

Zhu-Ying Gao, Zhi-Xue Yang, Dong-Hao Deng, Feng Zhang, Bing Yu, Yang-Yang Zhang, Xiang Lv, Yukio Nakamura, An Gong, Tao Cheng and 2 more

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Article in Blood science (Baltimore, Md.), 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.

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1 · What the graph read from it

What it found

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2 · The registry

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3 · Its place in the literature

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4 · The record

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5 · Who and what money

Authors and funding

12 authors.

Zhu-Ying GaoState Key Laboratory of Experimental Hematology, National Clinical Research Center for Blood Diseases, Haihe Laboratory of Cell Ecosystem, Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College, Tianjin 300020, China.
Zhi-Xue YangState Key Laboratory of Experimental Hematology, National Clinical Research Center for Blood Diseases, Haihe Laboratory of Cell Ecosystem, Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College, Tianjin 300020, China.
Dong-Hao DengState Key Laboratory of Experimental Hematology, National Clinical Research Center for Blood Diseases, Haihe Laboratory of Cell Ecosystem, Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College, Tianjin 300020, China.
Feng ZhangState Key Laboratory of Experimental Hematology, National Clinical Research Center for Blood Diseases, Haihe Laboratory of Cell Ecosystem, Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College, Tianjin 300020, China.
Bing YuCollege of Computer Science and Technology, China University of Petroleum (East China), Qingdao 266000, China.
Yang-Yang ZhangState Key Laboratory of Experimental Hematology, National Clinical Research Center for Blood Diseases, Haihe Laboratory of Cell Ecosystem, Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College, Tianjin 300020, China.
Xiang LvState Key Laboratory of Medical Molecular Biology, Institute of Basic Medical Sciences, Chinese Academy of Medical Sciences & Peking Union Medical College, Beijing, 100005, P.R. China.
Yukio NakamuraCell Engineering Division, RIKEN BioResource Center, Tsukuba, Ibaraki, Japan.
An GongCollege of Computer Science and Technology, China University of Petroleum (East China), Qingdao 266000, China.
Tao ChengState Key Laboratory of Experimental Hematology, National Clinical Research Center for Blood Diseases, Haihe Laboratory of Cell Ecosystem, Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College, Tianjin 300020, China.
Jian-Ping ZhangState Key Laboratory of Experimental Hematology, National Clinical Research Center for Blood Diseases, Haihe Laboratory of Cell Ecosystem, Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College, Tianjin 300020, China.
Xiao-Bing ZhangState Key Laboratory of Experimental Hematology, National Clinical Research Center for Blood Diseases, Haihe Laboratory of Cell Ecosystem, Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College, Tianjin 300020, China.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

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 patients to become transfusion-independent and substantially reducing vaso-occlusive events in sickle cell disease-there remains a significant opportunity to enhance its efficacy and lower production costs. Here, we present G2B, a novel hemoglobin beta gene (HBB) lentiviral vector derived from BB305-like constructs (BB305L), engineered by shortening the locus control region (LCR) and incorporating a mutated HBG2 promoter. Compared to BB305L, G2B achieves a ~4-fold increase in lentiviral titer and a 60% boost in β-globin expression. In a transplantation model using Hbb

Indexed as

Gene therapyHemoglobinopathiesHemoglobin subunit beta (HBB)Integration safetyLentiviral vectorLocus control regionβ-Thalassemia

Identifiers

PMID41113105
PMCPMC12530315

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Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.