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Oral Levodopa treatment in Improving Visual development in Infants and young children with Albinism-the OLIVIA study

口服左旋多巴治疗改善白化病婴幼儿视觉发育——OLIVIA研究

基本信息

批准号：
MR/R007640/1
负责人：
Helena Lee
金额：
$ 151.49万
依托单位：
University of Southampton
依托单位国家：
英国
项目类别：
Fellowship
财政年份：
2018
资助国家：
英国
起止时间：
2018 至无数据
项目状态：
已结题

来源：
https://gtr.ukri.org/projects?ref=MR%2FR007640%2F1
关键词：
Oral Levodopa treatment Improving Visual

项目摘要

Currently, there are no treatments for the eye problems seen in albinism. The average vision in albinism at 20/80, is below UK driving standards, which has implications for school, work and social life. This is why finding a treatment that can improve eyesight in albinism, was named as a priority by the Sight Loss and Vision Priority Setting Partnership in 2013. We know that the brain has the amazing ability to change and adapt in children. We also know that we make use of the brain's ability to rewire itself, when we improve eyesight in lazy eyes using glasses and patching. In albinism, a chemical called L-DOPA is missing from the eye and this causes problems with eye development. This is why eyesight is so poor in albinism. However, the eye is still able to change and develop in young children with albinism. Similar to the treatment of lazy eyes, we can target this flexibility in albinism. Potentially, replacing L-DOPA in albinism at a young age, will improve eye development and eyesight. The main aim of this study is to prove for the first time that we can change how the eye develops and improve eyesight in albinism after birth, by replacing the missing L-DOPA. We will also figure out what the best dose of L-DOPA is, by testing its effects on eye development and eyesight in mice with albinism, when given at different ages, doses and lengths of time. The second aim of this study is to carry out a small trial of L-DOPA treatment in children with albinism. L-DOPA is a safe medicine that is currently being used to treat infants and young children born with problems in controlling movement of their limbs. We will explore, together with the parents of the affected children, if the treatment and examinations carried out as part of this trial are reasonable. If successful, this study will completely change how children with albinism are treated. It will also set an important precedent for the development of new treatments for other eye diseases that affect children.

目前，还没有治疗白化病的眼睛问题。白化病患者的平均视力为20/80，低于英国驾驶标准，这对学校，工作和社会生活都有影响。这就是为什么找到一种可以改善白化病患者视力的治疗方法，被视力丧失和视力优先设置伙伴关系在2013年列为优先事项。我们知道，儿童的大脑具有惊人的改变和适应能力。我们还知道，当我们用眼镜和眼罩来改善弱视眼的视力时，我们利用了大脑的自我修复能力。在白化病中，一种叫做L-DOPA的化学物质从眼睛中消失，这会导致眼睛发育的问题。这就是为什么白化病患者的视力如此之差。然而，白化病幼儿的眼睛仍然能够发生变化和发育。类似于弱视的治疗，我们可以针对白化病的这种灵活性。潜在地，在年轻时取代白化病患者的左旋多巴，将改善眼睛发育和视力。这项研究的主要目的是首次证明，我们可以改变眼睛的发育方式，通过替换缺失的左旋多巴来改善白化病患者出生后的视力。我们还将找出左旋多巴的最佳剂量是什么，通过测试它对白化病小鼠的眼睛发育和视力的影响，在不同的年龄，剂量和时间长度。本研究的第二个目的是在白化病儿童中进行L-DOPA治疗的小型试验。左旋多巴是一种安全的药物，目前被用于治疗婴儿和幼儿出生时控制肢体运动的问题。我们将与受影响儿童的父母一起探讨作为本试验一部分进行的治疗和检查是否合理。如果成功，这项研究将完全改变白化病儿童的治疗方式。这也将为开发其他影响儿童的眼病的新疗法树立一个重要的先例。

项目成果

期刊论文数量（4）

专著数量（0）

科研奖励数量（0）

会议论文数量（0）

专利数量（0）

Human equivalent doses of L-DOPA rescues retinal morphology and visual function in a murine model of albinism.

DOI：
10.1038/s41598-023-44373-3
发表时间：
2023-10-11
期刊：
SCIENTIFIC REPORTS
影响因子：
4.6
作者：
Sanchez-Bretano, Aida;Keeling, Eloise;Scott, Jennifer A.;Lynn, Savannah A.;Soundara-Pandi, Sudha Priya;Macdonald, Sarah L.;Newall, Tutte;Griffiths, Helen;Lotery, Andrew J.;Ratnayaka, J. Arjuna;Self, Jay E.;Lee, Helena
通讯作者：
Lee, Helena

Retinal and optic nerve changes in microcephaly: An optical coherence tomography study.

DOI：
10.1212/wnl.0000000000005950
发表时间：
2018-08-07
期刊：
Neurology
影响因子：
9.9
作者：
Papageorgiou E;Pilat A;Proudlock F;Lee H;Purohit R;Sheth V;Vasudevan P;Gottlob I
通讯作者：
Gottlob I

Human equivalent doses of L-DOPA rescues retinal morphology and visual function in a murine model of albinism

人类等效剂量的左旋多巴可挽救白化病小鼠模型中的视网膜形态和视觉功能