Oral Levodopa treatment in Improving Visual development in Infants and young children with Albinism-the OLIVIA study
Oral Levodopa treatment in Improving Visual development in Infants and young children with Albinism-the OLIVIA study
批准号:
MR/R007640/1
负责人:
Helena Lee
金额:
$151.49万
依托单位国家:
英国
项目类别:
Fellowship
财政年份:
2018
资助国家:
英国
项目状态:
已结题
起止时间:
2018 至 --
中文摘要
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英文摘要
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.
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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
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
DOI:
10.21203/rs.3.rs-2974945/v1
发表时间:
2023
期刊:
影响因子:
--
作者:
[Sanchez-Bretano A]
通讯作者:
Sanchez-Bretano A
DOI:
10.1111/pcmr.12782
发表时间:
2019-09-01
期刊:
PIGMENT CELL & MELANOMA RESEARCH
影响因子:
4.3
作者:
[Lee, Helena, Scott, Jennifer, Lotery, Andrew]
通讯作者:
Lotery, Andrew
海外基金