氯沙坦在囊性纤维化中的效果(CF)-NIH 资助编号 133240

该数据集来自 clinicaltrials.gov,主题为氯沙坦在囊性纤维化(CF)中的效果,对应 NIH 资助编号 133240

University of Kansas Medical CenterUniversity of Kansas Medical Center
clinicaltrials
浏览 9
基因组Cystic Fibrosis
Effect of Losartan in Cystic Fibrosis (CF)-NIH Grant #133240

基本信息

模态
基因组
创建/更新时间
2026-10-12

资源简介

氯沙坦在囊性纤维化中的效果(CF)-NIH 资助编号 133240

该数据集来自 clinicaltrials.gov,主题为氯沙坦在囊性纤维化(CF)中的效果,对应 NIH 资助编号 133240。囊性纤维化是最常见的遗传性疾病之一,在美国影响约 30,000 人,年度医疗费用至少 18 亿美元,患者中位预测生存年龄在美国已提高至接近 40 岁。近二十年来研究者已发现约 2,000 个 CFTR 基因突变,这些突变被不完善地分为 5 组,针对组特异性 CFTR 突变体的小分子药物正在开发中,其中 CFTR 增强剂 ivacaftor(Kalydeco™)经 FDA 批准主要用于 III 类突变尤其是 G551D,可改善离子转运、临床结局等(来源 clinicaltrials.gov)。

【数据内容】

源站描述(机器翻译,仅供参考):疾病:囊性纤维化。CF 是最常见的遗传性疾病,可导致寿命缩短,在美国影响约 30,000 人,年度医疗费用至少 18 亿美元。这些患者的中位预测生存年龄已有所改善,目前在美国接近 40 岁。在过去二十年中,研究者已在 CF 跨膜传导调节因子(CFTR)基因中发现了约 2,000 个突变。这些突变被不完善地分为 5 组,目前正在开发可挽救组特异性 CFTR 突变体的小分子药物。这些药物已在部分患者中产生显著改善。CFTR 增强剂 ivacaftor(Kalydeco™)经 FDA 批准主要用于 III 类突变尤其是 G551D,可改善离子转运(汗液氯离子大幅下降)、临床结局(FEV1 和体重增加、急性加重减少)以及生活质

【主题与分类】

主题标签:Cystic Fibrosis。本站分类:呼吸道疾病/囊性纤维化。

【获取与许可】

源站页面:https://clinicaltrials.gov/study/NCT03435939

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下载信息

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需要注册 Kaggle 账号并登录后下载,适合需要跟踪下载记录和使用 API 的用户。

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暂未开放

当前数据集主要来源为 Kaggle 公开托管,完整影像包建议通过原始链接或 Kaggle API 获取。

使用方式

数据集获取

源站:https://clinicaltrials.gov/study/NCT03435939

标准获取流程:

  1. 访问源站链接,注册/登录账号;
  2. 进入数据集详情页,按页面指引申请权限或直接下载;
  3. 部分平台提供 SDK,例如:
    • OpenDataLab: pip install openxlab 后 openxlab.dataset.get(...)
    • ModelScope: pip install modelscope 后 modelscope download
    • 天池: 登录后页面提供直链下载
      以源站「数据下载/使用文档」页面为准。

数据说明

Conditions: Cystic Fibrosis. CF is the most common inherited disease causing a shortened life span, affecting ~30,000 people in the United States with annual health care costs of at least $1.8 billion. The median age of predicted survival of these patients has improved and is now almost 40 years in the US. Over the last two decades, investigators have identified ~2,000 mutations in the CF transmembrane conductance regulator (CFTR) gene. These mutations are imperfectly classified into 5 groups, and small molecules are being developed that rescue group-specific CFTR mutants. These agents have produced remarkable improvements in some patients. The CFTR potentiator ivacaftor (Kalydeco™), approved by the FDA mainly for class III mutations especially G551D, improves ion transport (large decrease in sweat chloride), clinical outcome (increased FEV1 and weight, decreased exacerbations), and quality of life. Furthermore, the FDA recently approved Orkambi™ (a corrector and potentiator: lumacaftor plus ivacaftor) because it reduced exacerbation rates by up to 39% in patients homozygous for F508del. It has been demonstrated in vitro that improvements in airway surface liquid (ASL) volume are highly predictive of changes seen in clinical studies and track with tracheal mucus velocities measured in sheep in vivo using the CFTR potentiator ivacaftor, inhaled hypertonic saline and other interventions (preliminary data). ASL volume is regulated by ion fluxes through ENaC, CFTR, CaCC, and BK channels, and TGF-β1-mediated inflammation in CF cells decreases activities of CaCC (8) and BK. These findings suggest that effective and safe anti-inflammatory therapy has the potential to improve mucociliary dysfunction in CF patients, even in the absence of small molecule therapy. Currently used anti-inflammatory therapies such as high-dose ibuprofen and steroids produce unwanted side effects that negate their effectiveness. Other medications showed severe side effects in clinical trials. However, experiments proposed in

数据加载示例(图像类)

from PIL import Image
import glob, os

files = (glob.glob(os.path.join(path, "**", "*.png"), recursive=True)
       + glob.glob(os.path.join(path, "**", "*.jpg"), recursive=True)
       + glob.glob(os.path.join(path, "**", "*.tif"), recursive=True))
print("图像文件数:", len(files))
img = Image.open(files[0]); print("尺寸/模式:", img.size, img.mode)

# torchvision Dataset 方式:
# from torchvision import datasets
# ds = datasets.ImageFolder(path)  # 要求 子目录=类别

目录组织与标注格式以源站说明和下载后实际文件为准。

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