多囊病的实验模型:应用和治疗测试
Cynthia J Sieben1, Peter C Harris
1Division of Nephrology and Hypertension, Mayo Clinic, Rochester, Minnesota.
Kidney360
|July 7, 2023
概括
多囊病 (PKD) 研究需要准确的动物模型. 本综述评估了当前PKD治疗测试模型,强调了它们的成功和局限性.
科学领域:
- 腎臟病學 (nephrology) 是一種醫學專業.
- 遗传学 遗传学 是一个
- 翻译研究是翻译研究.
背景情况:
- 多囊性病 (PKD) 是一种遗传性疾病,导致囊和衰竭,与原发性乳毛功能障碍有关.
- 尽管有遗传学方面的进步,但只有一种疗法得到了FDA的批准,这需要更好的实验模型来开发药物.
- 现有的细胞模型是有限的;有机体具有潜力,但全生物体模型对于评估功能至关重要.
结论:
- 开发和完善正统实验模型对于推进PKD治疗方法至关重要.
- 目前的模型提供了有价值的见解,但需要进一步改进,以充分回顾人类PKD表型,并促进药物发现.
- 动物建模的持续创新是克服这些复杂遗传病治疗挑战的关键.
相关概念视频
Chronic Kidney Disease III: Interprofessional Care
45
Chronic kidney disease (CKD) requires collaborative and comprehensive management. CKD progresses through stages and can lead to end-stage kidney disease (ESKD) if untreated. Interprofessional collaboration and patient education are crucial, enabling patients to manage their health and improve their quality of life.Diagnostic approach for chronic kidney diseaseThe diagnosis of CKD primarily focuses on the glomerular filtration rate (GFR), which assesses kidney function by measuring how well...
45
EPS and iPS Cells in Disease Research
2.8K
Embryonic and induced pluripotent stem cells are excellent models for disease research because of their ability to self-renew and differentiate into most cell types. Somatic cells from a patient are isolated and reprogrammed into induced pluripotent stem cells or iPSCs. These iPSCs are later differentiated into the desired cell type, which mirrors the diseased cell of the patient. In this way, disease models have been created for investigating diseases such as Down syndrome, type I diabetes,...
2.8K
Pharmacokinetic Models: Comparison and Selection Criterion
109
Physiological and compartmental models are valuable tools used in studying biological systems. These models rely on differential equations to maintain mass balance within the system, ensuring an accurate representation of the dynamic processes at play.
Physiological models take a detailed approach by considering specific molecular processes. They can predict drug distribution, metabolism, and elimination changes, providing a comprehensive understanding of how drugs interact with the body.
Physiological models take a detailed approach by considering specific molecular processes. They can predict drug distribution, metabolism, and elimination changes, providing a comprehensive understanding of how drugs interact with the body.
109


