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HEIndividual fellowship2022–2024

BEndTarG · Brain Endothelium - TARgeted Gene therapy to improve Aβ clearance in Alzheimer’s Disease

Horizon Europe — Marie Skłodowska-Curie Actions

Duration
2022-09-01 → 2024-08-31
EU contribution
€165,313
Participants
1
Scheme
HORIZON-TMA-MSCA-PF-EF

Lines connect the coordinator with its partners.

Results in brief

Brain Endothelium - TARgeted Gene therapy to improve Aβ clearance in Alzheimer’s Disease

Alzheimer's disease (AD) is a progressive neurodegenerative disorder with no cure and limited treatment options. It is the most common cause of dementia, and its prevalence is expected to double in Europe by 2050 due to the ageing population. Despite significant research efforts, current therapies are limited in their ability to halt disease progression. To tackle this pressing problem, researchers have focused on grasping the pathogenesis of Alzheimer's disease (AD) and creating novel therapies aimed at enhancing the brain's protective systems, such as the blood-brain barrier, while also diminishing the buildup of amyloid-beta (Aβ) proteins. To tackle this pressing problem, the present project has been focused on developing an innovative gene therapy approach aimed at targeting and modulating the blood-brain barrier (BBB) to enhance the overall clearance of Aβ. The proposed Aβ lowering intervention relies on re-establishing proper expression levels of a novel key intervenient in the process of Aβ clearance across the brain endothelium. By delivering such therapeutic genes specifically to the brain endothelium, one aims to: i) restore target gene expression at BBB and enhance brain endothelial cells clearance function; ii) modulate Aβ accumulation within the brain, a key contributor to AD pathology; and iii) promote neuroprotection from Aβ-associated damage and death, thereby slowing cognitive decline.

Data: CORDIS, © European Union

Project objective

Alzheimer disease (AD) is the most common cause of dementia (60-80% of cases) with a prevalence near 50 million people. There is no cure available and there are only two classes of approved drugs that are effective in treat some of the cognitive symptoms and potentially delay the clinical decline. Despite the considerable progress achieved in the research of best performing disease-modifying agents, the main clinical challenge remains the accomplishment of an efficient and safe therapeutic option that can arrest the disease progression and prevent cognitive failure. In this project, I am proposing an innovative approach to improve AD pathophysiology by targeting the brain endothelium to improve the Amyloid-β (Aβ) clearance using gene therapy. I will use super-selective nanocarriers to carry relevant genes for the enhancement of the Aβ clearance mechanism. These nanocarriers will be functionalised to allow the specific targeting of brain endothelial cells and to increase its potency to deliver the therapeutic gene into these cells. This project will combine for the first time, the use of super-selective binding strategies and gene therapy for brain endothelium targeting and modulation in the context of AD. Overall, I will develop an innovative therapy for AD treatment, capable of reaching the BBB in a non-invasive way, being able to target the brain endothelium, overcome the defective Aβ transendothelial clearance, and potentially arrest the disease progression and prevent cognitive failure. Therefore, this project will reach a global impact by substantially contributing with a novel avenue to change the clinical course of AD pathogenesis.

Original text from CORDIS.

Participants

  • FUNDACIO INSTITUT DE BIOENGINYERIA DE CATALUNYA · BarcelonaCoordinatorSpain

Links

Data: CORDIS, © European Union