Proprotein convertase subtilisin/kexin 9 (PCSK9) in the pathophysiology a treatment of dry age-related macular degeneration
Public support
Provider
Ministry of Health
Programme
Programme to support medical applied research in 2015 to 2022
Call for proposals
Zdravotnický AV 3 (SMZ0201701)
Main participants
Univerzita Karlova / Lékařská fakulta v Hradci Králové
Contest type
VS - Public tender
Contract ID
17-29241A
Alternative language
Project name in Czech
Proprotein konvertáza subtilisin/kexin 9 (PCSK9) v patofyziologii a léčbě věkem podmíněné suché formy makulární degenerace
Annotation in Czech
Proprotein konvertáza subtilisin/kexin 9 (PCSK9) je sekreční regulátor receptorů low density lipoprotein (LDL) cholesterolu na povrchu buněk. Snížení PCSK9 představuje velmi slibný nový přístup k léčbě hypercholesterolémie a s ní asociovaných chorob. Lipidy mají také velmi pravděpodobně významnou roli v patogenezi věkem podmíněné makulární degenerace (AMD), příčiny nevratné ztráty zraku starších lidí u nás. Možnosti zastavit/zpomalit progresi AMD jsou dosud omezené; u suché formy AMD se ukázala být slibnou reoferéza. Zcela nedávno se také ukázalo, že rheoferéza kromě odstranění LDL snižuje hladinu PCSK9 v plasmě. V dlouhodobém sledování bude korelován klinický průběh se změnami hladiny PCSK9 a vybranými dalšími humorálními ukazateli ve snaze najít optimální prognostické bioindikátory. Protože pro funkci sítnice má LDL receptor a metabolismus LDL cholesterolu zásadní význam, a pro terapeutický potenciál rheoferézy signifikantně snížit jak koncentraci LDL i PCSK9 v plasmě, předpokládáme, že navrhovaný projekt přinese zcela nové poznatky a rozšíří budoucí možnosti léčby AMD.
Scientific branches
R&D category
AP - Applied research
CEP classification - main branch
FB - Endocrinology, diabetology, metabolism, nutrition
CEP - secondary branch
EI - Biotechnology and bionics
CEP - another secondary branch
FF - ENT (ie. ear, nose, throat), ophthalmology, dentistry
OECD FORD - equivalent branches <br>(according to the <a href="http://www.vyzkum.cz/storage/att/E6EF7938F0E854BAE520AC119FB22E8D/Prevodnik_oboru_Frascati.pdf">converter</a>)
20801 - Environmental biotechnology<br>20802 - Bioremediation, diagnostic biotechnologies (DNA chips and biosensing devices) in environmental management<br>20803 - Environmental biotechnology related ethics<br>20901 - Industrial biotechnology<br>20902 - Bioprocessing technologies (industrial processes relying on biological agents to drive the process) biocatalysis, fermentation<br>20903 - Bioproducts (products that are manufactured using biological material as feedstock) biomaterials, bioplastics, biofuels, bioderived bulk and fine chemicals, bio-derived novel materials<br>30202 - Endocrinology and metabolism (including diabetes, hormones)<br>30206 - Otorhinolaryngology<br>30207 - Ophthalmology<br>30208 - Dentistry, oral surgery and medicine<br>30401 - Health-related biotechnology<br>30402 - Technologies involving the manipulation of cells, tissues, organs or the whole organism (assisted reproduction)<br>30403 - Technologies involving identifying the functioning of DNA, proteins and enzymes and how they influence the onset of disease and maintenance of well-being (gene-based diagnostics and therapeutic interventions [pharmacogenomics, gene-based therapeutics])<br>30404 - Biomaterials (as related to medical implants, devices, sensors)<br>30405 - Medical biotechnology related ethics<br>40401 - Agricultural biotechnology and food biotechnology<br>40402 - GM technology (crops and livestock), livestock cloning, marker assisted selection, diagnostics (DNA chips and biosensing devices for the early/accurate detection of diseases) biomass feedstock production technologies, biopharming<br>40403 - Agricultural biotechnology related ethics
Completed project evaluation
Provider evaluation
U - Uspěl podle zadání (s publikovanými či patentovanými výsledky atd.)
Project results evaluation
The project has achieved its objectives, the results are clearly and transparently presented. Age-related macular degeneration causes irreversible visual impairment in the elderly, and long-term research into the treatment of the dry form of this disease with rheopheresis has shown that improvement of findings, including visual acuity, occurred in 32.2% of cases and therapeutic failure in 12.9%. Other participants did not attend the control during the follow-up period. One dissertation was defended within the project.The project has resulted in publications at various levels, the most important of which are in IF journals (two currently exist and two others are under review). No deficiencies in compliance with the specifications or in the use of the financial resources were found.
Solution timeline
Realization period - beginning
Apr 1, 2017
Realization period - end
Dec 31, 2020
Project status
U - Finished project
Latest support payment
Jun 28, 2018
Data delivery to CEP
Confidentiality
S - Úplné a pravdivé údaje o projektu nepodléhají ochraně podle zvláštních právních předpisů
Data delivery code
CEP21-MZ0-NV-U/03:1
Data delivery date
Jun 25, 2021
Finance
Total approved costs
9,814 thou. CZK
Public financial support
9,814 thou. CZK
Other public sources
0 thou. CZK
Non public and foreign sources
0 thou. CZK