Kidney Research Institute, New York City, NY, USA.
Institute of Molecular Biology and Genetics (Biogem), Ariano Irpino, Italy.
Nat Rev Nephrol. 2024 Jul;20(7):460-472. doi: 10.1038/s41581-024-00828-y. Epub 2024 Apr 4.
Notable progress in basic, translational and clinical nephrology research has been made over the past five decades. Nonetheless, many challenges remain, including obstacles to the early detection of kidney disease, disparities in access to care and variability in responses to existing and emerging therapies. Innovations in drug development, research technologies, tissue engineering and regenerative medicine have the potential to improve patient outcomes. Exciting prospects include the availability of new drugs to slow or halt the progression of chronic kidney disease, the development of bioartificial kidneys that mimic healthy kidney functions, and tissue engineering techniques that could enable transplantable kidneys to be created from the cells of the recipient, removing the risk of rejection. Cell and gene therapies have the potential to be applied for kidney tissue regeneration and repair. In addition, about 30% of kidney disease cases are monogenic and could potentially be treated using these genetic medicine approaches. Systemic diseases that involve the kidney, such as diabetes mellitus and hypertension, might also be amenable to these treatments. Continued investment, communication, collaboration and translation of innovations are crucial to realize their full potential. In addition, increasing sophistication in exploring large datasets, implementation science, and qualitative methodologies will improve the ability to deliver transformational kidney health strategies.
在过去的五十年中，基础、转化和临床肾脏病学研究取得了显著进展。尽管如此，仍有许多挑战存在，包括早期发现肾脏疾病的障碍、获得护理的机会不平等以及对现有和新兴疗法的反应存在差异。药物开发、研究技术、组织工程和再生医学的创新有可能改善患者的预后。令人兴奋的前景包括可用于减缓或阻止慢性肾脏病进展的新药、开发模拟健康肾脏功能的生物人工肾脏，以及组织工程技术，使可移植的肾脏能够从受者的细胞中创建，消除排斥的风险。细胞和基因疗法有可能用于肾脏组织的再生和修复。此外，约 30%的肾脏疾病是单基因疾病，可能可以通过这些基因治疗方法进行治疗。涉及肾脏的系统性疾病，如糖尿病和高血压，也可能适合这些治疗。持续的投资、沟通、合作和创新的转化对于充分发挥其潜力至关重要。此外，通过探索大型数据集、实施科学和定性方法的不断完善，将提高提供变革性肾脏健康策略的能力。
