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Unravelling cell type-specific responses to Parkinson's Disease at single cell ...

Martirosyan, A; Ansari, R; Pestana, F; Hebestreit, K; Gasparyan, H; Aleksanyan, R; Hnatova, S; Poovathingal, S; Marneffe, C; Thal, DR; Kottick, A

Parkinson’s Disease (PD) is the second most common neurodegenerative disorder. The pathological hallmark of PD is loss of dopaminergic neurons and the presence of aggregated α-synuclein, primarily in the substantia nigra pars compacta (SNpc) of the midbrain. However, the molecular mechanisms that underlie the pathology in different cell types is not currently understood. Here, we present a single nucleus transc...


AAV-mediated delivery of an anti-BACE1 VHH alleviates pathology in an Alzheimer...

Marino, M; Zhou, L; Rincon, MY; Callaerts-Vegh, Z; Verhaert, J; Wahis, J; Creemers, E; Yshii, L; Wierda, K; Saito, T; Marneffe, C; Voytyuk, I

Single domain antibodies (VHHs) are potentially disruptive therapeutics, with important biological value for treatment of several diseases, including neurological disorders. However, VHHs have not been widely used in the central nervous system (CNS), largely because of their restricted blood-brain barrier (BBB) penetration. Here, we propose a gene transfer strategy based on BBB-crossing adeno-associated virus (...


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