Gene therapy, which involves the delivery of genetic material into cells to correct an underlying genetic problem, has emerged as a promising approach for treating various conditions. To promote research in this rapidly evolving field, we developed the Gene Therapy Omnibus (GTO) (http://www.inbirg.com/gto/), a comprehensive resource containing detailed clinical trial data and molecular information related to gene therapy. The GTO includes 6333 clinical trial records and 3466 transcriptome profiles, with information on 614 altered genes and 22 types of gene therapy, including DNA therapies, RNA therapies and genetically-modified cell therapies. For each gene therapy product in a clinical trial, detailed information, such as altered gene name, structural components, indication, vector information, phase of the clinical trial, clinical outcomes and adverse effects, is provided when available. Additionally, 345 comparison datasets, including 29 single-cell RNA-sequencing datasets comprising information on both gene therapy and control samples, were established. Differential gene expression and downstream functional enrichment analyses were performed through standardized pipelines to elucidate the molecular alterations induced by gene therapy. The user-friendly interface of the GTO supports efficient data retrieval, visualization and analysis, making it an invaluable resource for researchers and clinicians performing clinical research on gene therapy and the underlying mechanisms.

中文翻译：

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GTO：全面的基因治疗综合体


基因疗法涉及将遗传物质递送到细胞中以纠正潜在的遗传问题，已成为治疗各种疾病的一种有前途的方法。为了促进这一快速发展领域的研究，我们开发了基因治疗综合 （GTO） （http://www.inbirg.com/gto/），这是一个包含与基因治疗相关的详细临床试验数据和分子信息的综合资源。GTO 包括 6333 份临床试验记录和 3466 份转录组图谱，以及 614 个突变基因和 22 种基因疗法的信息，包括 DNA 疗法、RNA 疗法和转基因细胞疗法。对于临床试验中的每种基因治疗产品，都会提供详细信息，例如改变的基因名称、结构成分、适应症、载体信息、临床试验阶段、临床结果和不良反应。此外，还建立了 345 个比较数据集，包括 29 个单细胞 RNA 测序数据集，包含基因治疗和对照样本的信息。通过标准化管道进行差异基因表达和下游功能富集分析，以阐明基因治疗诱导的分子改变。GTO 的用户友好界面支持高效的数据检索、可视化和分析，使其成为研究人员和临床医生对基因治疗及其潜在机制进行临床研究的宝贵资源。