Current treatment options and novel nanotechnology-driven enzyme replacement strategies for lysosomal storage disorders
A Del Grosso, G Parlanti, R Mezzena… - Advanced Drug Delivery …, 2022 - Elsevier
Lysosomal storage disorders (LSDs) are a vast group of more than 50 clinically identified
metabolic diseases. They are singly rare, but they affect collectively 1 on 5,000 live births …
metabolic diseases. They are singly rare, but they affect collectively 1 on 5,000 live births …
[HTML][HTML] A new hypothesis for Parkinson's disease pathogenesis: GTPase-p38 MAPK signaling and autophagy as convergence points of etiology and genomics
J Obergasteiger, G Frapporti, PP Pramstaller… - Molecular …, 2018 - Springer
The combination of genetics and genomics in Parkinson´ s disease has recently begun to
unveil molecular mechanisms possibly underlying disease onset and progression. In …
unveil molecular mechanisms possibly underlying disease onset and progression. In …
[HTML][HTML] Impaired Autophagy in Krabbe Disease: The Role of BCL2 and Beclin-1 Phosphorylation
N Papini, R Todisco, P Giussani, M Dei Cas… - International Journal of …, 2023 - mdpi.com
Autophagic impairment was identified in many lysosomal storage diseases and adult
neurodegenerative diseases. It seems that this defect could be directly related to the …
neurodegenerative diseases. It seems that this defect could be directly related to the …
[HTML][HTML] Rapamycin alleviates protein aggregates, reduces neuroinflammation, and rescues demyelination in globoid Cell leukodystrophy
DS Lin, YW Huang, TH Lee, L Chang, ZD Huang… - Cells, 2023 - mdpi.com
We have shown in vivo and in vitro previously that psychosine causes dysfunction of
autophagy and the ubiquitin-proteasome system underlying the pathogenesis of globoid cell …
autophagy and the ubiquitin-proteasome system underlying the pathogenesis of globoid cell …
Brain-targeted enzyme-loaded nanoparticles: A breach through the blood-brain barrier for enzyme replacement therapy in Krabbe disease
A Del Grosso, M Galliani, L Angella, M Santi… - Science …, 2019 - science.org
Lysosomal storage disorders (LSDs) result from an enzyme deficiency within lysosomes.
The systemic administration of the missing enzyme, however, is not effective in the case of …
The systemic administration of the missing enzyme, however, is not effective in the case of …
Mechanisms of demyelination and neurodegeneration in globoid cell leukodystrophy
Globoid cell leukodystrophy (GLD), also known as Krabbe disease, is a lysosomal storage
disorder causing extensive demyelination in the central and peripheral nervous systems …
disorder causing extensive demyelination in the central and peripheral nervous systems …
[HTML][HTML] Lithium chloride promotes osteogenesis and suppresses apoptosis during orthodontic tooth movement in osteoporotic model via regulating autophagy
Osteoporosis is a widely distributed disease that may cause complications such as
accelerated tooth movement, bone resorption, and tooth loss during orthodontic treatment …
accelerated tooth movement, bone resorption, and tooth loss during orthodontic treatment …
Autophagy in hemorrhagic stroke: mechanisms and clinical implications
H Li, J Wu, H Shen, X Yao, C Liu, S Pianta, J Han… - Progress in …, 2018 - Elsevier
Accumulating evidence advances the critical role of autophagy in brain pathology after
stroke. Investigations employing autophagy induction or inhibition using pharmacological …
stroke. Investigations employing autophagy induction or inhibition using pharmacological …
[HTML][HTML] Lithium promotes recovery of neurological function after spinal cord injury by inducing autophagy
D Zhang, F Wang, X Zhai, XH Li… - Neural regeneration …, 2018 - journals.lww.com
Lithium promotes autophagy and has a neuroprotective effect on spinal cord injury (SCI);
however, the underlying mechanisms remain unclear. Therefore, in this study, we …
however, the underlying mechanisms remain unclear. Therefore, in this study, we …
Cross-Linked enzyme aggregates as versatile tool for enzyme delivery: Application to polymeric nanoparticles
M Galliani, M Santi, A Del Grosso… - Bioconjugate …, 2018 - ACS Publications
Polymeric nanoparticles (NPs) represent one of the most promising tools in nanomedicine
and have been extensively studied for the delivery of water-insoluble drugs. However, the …
and have been extensively studied for the delivery of water-insoluble drugs. However, the …