A human slice model to understand the role of fibrosis in children with bladder exstrophy

人体切片模型了解纤维化在膀胱外翻儿童中的作用

基本信息

  • 批准号:
    MR/X030946/1
  • 负责人:
  • 金额:
    $ 62.58万
  • 依托单位:
  • 依托单位国家:
    英国
  • 项目类别:
    Research Grant
  • 财政年份:
    2024
  • 资助国家:
    英国
  • 起止时间:
    2024 至 无数据
  • 项目状态:
    未结题

项目摘要

Bladder exstrophy is a condition in babies where the bladder and front of the abdomen fail to develop properly, which leaves the open bladder exposed on the front of the tummy at birth. Surgery for affected children is only available in Manchester or Great Ormond Street (GOS) in the UK but outcomes are poor, even with the best care in these specialist centres: many are permanently incontinent with frequent urine infections and have sexual dysfunction in later life. The applicant, Mr Johal, leads exstrophy surgery at GOS and has a unique research background with a PHD focussing on normal and abnormal function of the muscles in the bladder. He has shown that most of the expected bladder muscles are replaced by scar tissue in exstrophy. This occurs via a process called fibrosis, which is also implicated in many other conditions; hence there are a number of new drugs being developed to treat fibrosis which have not yet been tested in exstrophy. Part of the abnormal bladder is removed during routine exstrophy surgery, and this will be used by Mr Johal to test whether fibrosis can be stopped (or even reversed) by these drugs, in collaboration with Professor David Long (Institute of Child Health) who has successfully undertaken similar experiments in the kidney. Taken together, Mr Johal's unique background, access to exstrophy samples and drive to improve outcomes combined with support from Professor Long's innovative laboratory team, this is a timely opportunity to increase understanding of bladder exstrophy and test new therapies that may be used clinically in future.
膀胱外翻是婴儿的一种情况,其中膀胱和腹部前部未能正常发育,这使得出生时开放的膀胱暴露在腹部前部。受影响儿童的手术仅在英国的曼彻斯特或大奥蒙德街(GOS)提供,但结果很差,即使在这些专科中心得到最好的护理:许多人永久性失禁,经常发生尿液感染,并在晚年出现性功能障碍。申请人Johal先生在GOS领导外翻手术,并拥有独特的研究背景,博士学位专注于膀胱肌肉的正常和异常功能。他已经证明,大多数预期的膀胱肌肉被外翻的瘢痕组织所取代。这是通过一个称为纤维化的过程发生的,这也涉及许多其他情况;因此,正在开发一些新的药物来治疗纤维化,这些药物尚未在外翻中进行测试。在常规的外翻手术中,部分异常膀胱被切除,Johal先生将与大卫龙教授(儿童健康研究所)合作,用这一点来测试这些药物是否可以阻止(甚至逆转)纤维化,他已经成功地在肾脏进行了类似的实验。总而言之,Johal先生的独特背景,获得外翻样本并推动改善结果,再加上Long教授创新实验室团队的支持,这是一个及时的机会,可以增加对膀胱外翻的理解,并测试未来可能在临床上使用的新疗法。

项目成果

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Navroop Singh Johal其他文献

Navroop Singh Johal的其他文献

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