The specific tau isoforms, such as 3-repeat (3R) and 4-repeat (4R) isoforms, and the distinct conformational strains that misfolded tau can adopt are determinants of the molecular and clinical heterogeneity observed across tauopathies.
Neddylation is a post-translational modification that conjugates the NEDD8 protein to protein substrates, such as the cullins, which once neddylated join complex to form cullin-RING ubiquitin E3 ligases (CRLs), which in turn play a crucial role in regulating proteasomal degradation. The University of Kentucky has presented preclinical data on their neddylation inhibitor TK-59 as a cancer therapeutic.
Nitrated α-synuclein is upregulated in the CSF of patients with Parkinson’s Disease (PD), Lewy body dementia (DLB), multiple system atrophy (MSA), and other synucleinopathies.
Researchers from Voyager Therapeutics Inc. presented preclinical activity data of VY-1706, a blood-brain barrier (BBB)-penetrant gene therapy comprising an adeno-associated virus serotype 9 capsid (AAV9-C9P39) vector encoding primary artificial microRNA (pri-amiRNA) consisting of short-interfering RNA (siRNA) targeting human microtubule-associated protein tau (MAPT) protein.
Heart failure with preserved ejection fraction (HFpEF) is a disease in which the left ventricle (LV) of the heart shows impaired relaxation during cardiac diastole, often accompanied by structural and functional abnormalities.
Researchers from Sanofi SA reported the preclinical characterization of SAR-446159 (ABL-301), a bispecific antibody construct comprising an antibody targeting α-synuclein fused to an engineered antibody fragment that targets IGF-1R and functions as a blood-brain barrier (BBB) shuttle, known as the Grabody-B platform.
Sepsis-induced coagulopathy (SIC) is a complication of sepsis tied to high mortality in patients. Anticoagulation using a coagulation factor IIa and Xa dual inhibitor might have the potential to improve the treatment of this severe condition.
BDNF is the brain’s most abundant neurotrophic factor, playing a key role in neuronal survival and synaptic plasticity through the activation of the transcription factor CREB, which is essential for driving beneficial effects in neurons. CREB is downregulated in Parkinson’s disease, Huntington’s disease, frontotemporal dementia, Alzheimer’s disease and other neurodegenerative conditions.
Transferrin receptor (TfR)-mediated transcytosis is a receptor-mediated mechanism for drug delivery to the brain or other tissues, where, after the antibody binds to transferrin, the therapeutic agent is internalized and released into the brain.
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