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КартаВръзкиТемиБългарияНощта (K-TRIO)Мария Склодовска-КюриЗа ученициЗа учителиМетодологияИзненадай ме
H2020Обмен на изследователи2017–2022

LysoMod · Genetic and Small Molecule Modifiers of Lysosomal Function

„Хоризонт 2020“ — Действия „Мария Склодовска-Кюри“

Период
2017-03-01 → 2022-08-31
Финансиране от ЕС
1 102 500 €
Участници
14
Схема
MSCA-RISE

Линиите свързват координатора с партньорите.

Накратко на български

Лизо좀ната функция и генетичните вариации се анализират, като например се изследва как ацетил-леуцин защитава нервните клетки. Това помага за разбирането на причините за различните симптоми при пациентите и търсенето на нови терапевтични подходи.

Този кратък обзор е генериран от изкуствен интелект

Кратко обяснение, генерирано от езиков модел по текста на CORDIS. Оригиналът е по-долу.

Резултати накратко

Genetic and Small Molecule Modifiers of Lysosomal Function

The LysoMod project contributed innovations in the area of personalized medicine for disorders linked to lysosomal dysfunction (both rare and common) by implementing an international staff-exchange program between highly complementary and multidisciplinary academic experts and non-academic partners. Research over the past decades has clearly shown that as new aspects of lysosomal biology are revealed, new therapeutic strategies can be devised, in most cases based on the manipulation of specific molecular pathways. In summary, the LysoMod project has 1) identified genetic variation that may explain why different patients with the same causative mutation (including siblings) present a wide range of clinical symptoms; 2) demonstrated that acetyl-leucine (a derivative of amino acid leucine) exerts a neuroprotective effect in mouse models and in patients with lysosomal diseases; and 3) uncovered cellular components and signaling pathways involved in lysosomal dysfunction that may be targets for the development of new therapeutic approaches.

Текст от CORDIS, на английски · Данни: CORDIS, © Европейски съюз

Цел на проекта

LysoMod will innovate in the area of personalized medicine for disorders linked to lysosomal dysfunction. This will be achieved by implementing a collaborative staff-exchange program between highly complementary and multidisciplinary academic and non-academic partners with expertise in pharmacology, medicinal chemistry, cell biology, biochemistry, mouse and human genetics, transcriptomics, proteomics and lipidomics. Based on the critical role that lysosomes play in cells, a better understanding of lysosomal function will have a major impact on human health, fostering the development of new strategies to improve quality of life for people affected by a variety of diseases, ranging from lysosomal storage diseases (LSDs) to age-related neurodegenerative disorders. LysoMod’s specific objectives are: 1) to develop and further optimize existing therapies for LSDs; 2) to identify new targets for personalized therapies for LSDs; and 3) to investigate the cross-talk between lysosomal function, signalling pathways and gene expression regulation. The pioneer work of a participant in the consortium led to the development of a drug that is approved for clinical use. LysoMod will i) investigate the mechanisms of action of this and other drugs in lysosome-related disorder; ii) identify modifier genes involved in LSD pathology and test their potential as new targets for personalized therapeutic approaches; iii) identify candidate RNAs that can be targeted to enhance lysosomal function. The companies in the consortium will ensure a rapid transfer of new knowledge into applications for diagnostics and clinical trials. Prioritising lysosomal dysfunction as a highly relevant biomedical problem, the LysoMod consortium will implement a mentored staff-exchange program to provide young researchers with high-level training in innovative approaches for exploring biological systems, preparing the next generation of researchers for careers either in the private or public health sectors.

Оригинален текст от CORDIS (на английски).

Участници

Връзки

Данни: CORDIS, © Европейски съюз