自引率: 3.8%
被引量: 6795
通过率: 暂无数据
审稿周期: 2.33
版面费用: 暂无数据
国人发稿量: 65
投稿须知/期刊简介:
Gene Therapy is a high quality research journal publishing papers and reporting data on methods and clinical trials for introducing genes into cells for treatment of inherited disease, cancer, the developing of animal models, and all aspects of gene therapy research. At the forefront of medicine, this journal publishes the latest research into identification of gene structure and disease processes and the development of clinical methods to treat disease.
期刊描述简介:
Gene Therapy is a high quality research journal publishing papers and reporting data on methods and clinical trials for introducing genes into cells for treatment of inherited disease, cancer, the developing of animal models, and all aspects of gene therapy research. At the forefront of medicine, this journal publishes the latest research into identification of gene structure and disease processes and the development of clinical methods to treat disease.
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Precision medicine: toward restoring fat with gene therapy in inherited lipodystrophy.
被引量：- 发表：1970
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BRD9 promotes the progression of gallbladder cancer via CST1 upregulation and interaction with FOXP1 through the PI3K/AKT pathway and represents a therapeutic target.
被引量：- 发表：1970
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Activated factor X delivered by adeno-associated virus significantly inhibited bleeding and alleviated hemophilic synovitis in hemophilic mice.
In hemophilia, deficiency of factor VIII or IX prevents the activation of the common coagulation pathway, and inhibits the conversion of FX to activated FXa, which is required for thrombin generation. We hypothesized that the direct expressed FXa has the potential to activate the common pathway and restore coagulation in hemophilia patients. In this study, the cassettes that expressed FXa, FXaop and FXa-FVII were packaged into an engineered AAV capsid, AAV843, and were delivered into hemophilia A and B mice by intravenous injection. AAV-FXaop could be stably expressed in vivo and showed the best immediate and prolonged hemostatic effects, similar to those of commercial drugs (Xyntha and Benefix). AAV-FXaop also significantly inhibited bleeding in hemophilia A mice with inhibitors. In addition, FXa expression in joints significantly alleviated the occurrence of hemophilic synovitis. AAV-delivered FXa may be a novel target for treating hemophilic and hemophilic synovitis.
被引量：- 发表：1970
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Intravesical nerve growth factor antisense therapy for bladder hypersensitivity induced by psychological stress.
被引量：- 发表：1970
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Lentiviral vector gene therapy and CFTR modulators show comparable effectiveness in cystic fibrosis rat airway models.
Mutation-agnostic treatments such as airway gene therapy have the potential to treat any individual with cystic fibrosis (CF), irrespective of their CF transmembrane conductance regulator (CFTR) gene variants. The aim of this study was to employ two CF rat models, Phe508del and CFTR knockout (KO), to assess the comparative effectiveness of CFTR modulators and lentiviral (LV) vector-mediated gene therapy. Cells were isolated from the tracheas of rats and used to establish air-liquid interface (ALI) cultures. Phe508del rat ALIs were treated with the modulator combination, elexacaftor-tezacaftor-ivacaftor (ETI), and separate groups of Phe508del and KO tracheal epithelial cells were treated with LV-CFTR followed by differentiation at ALI. Ussing chamber measurements were performed to assess CFTR function. ETI-treated Phe508del ALI cultures demonstrated CFTR function that was 59% of wild-type level, while gene-addition therapy restored Phe508del to 68% and KO to 47% of wild-type level, respectively. Our findings show that rat Phe508del-CFTR protein can be successfully rescued with ETI treatment, and that CFTR gene-addition therapy provides significant CFTR correction in Phe508del and KO ALI cultures to levels that were comparable to ETI. These findings highlight the potential of an LV vector-based gene therapy for the treatment of CF lung disease.
被引量：- 发表：1970
