The blood–brain barrier: structure, regulation, and drug delivery

D Wu, Q Chen, X Chen, F Han, Z Chen… - Signal Transduction and …, 2023 - nature.com
Blood–brain barrier (BBB) is a natural protective membrane that prevents central nervous
system (CNS) from toxins and pathogens in blood. However, the presence of BBB …

Fabry disease: Mechanism and therapeutics strategies

X Li, X Ren, Y Zhang, L Ding, M Huo, Q Li - Frontiers in pharmacology, 2022 - frontiersin.org
Fabry disease is a monogenic disease characterized by a deficiency or loss of the α-
galactosidase A (GLA). The resulting impairment in lysosomal GLA enzymatic activity leads …

Lysosomal nanotoxicity: Impact of nanomedicines on lysosomal function

M Uzhytchak, B Smolková, M Lunova, A Frtús… - Advanced Drug Delivery …, 2023 - Elsevier
Although several nanomedicines got clinical approval over the past two decades, the clinical
translation rate is relatively small so far. There are many post-surveillance withdrawals of …

Comparison of three synthetic transferrin mimetic small peptides to promote the blood–brain barrier penetration of vincristine liposomes for improved glioma targeted …

S Mojarad-Jabali, M Farshbaf, S Hemmati… - International Journal of …, 2022 - Elsevier
The existence of the blood–brain barrier (BBB) makes the clinical chemotherapy of glioma a
formidable challenge, because it hinders the passage of different chemotherapeutics into …

Polymer-based drug delivery systems under investigation for enzyme replacement and other therapies of lysosomal storage disorders

M Placci, MI Giannotti, S Muro - Advanced drug delivery reviews, 2023 - Elsevier
Lysosomes play a central role in cellular homeostasis and alterations in this compartment
associate with many diseases. The most studied example is that of lysosomal storage …

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 …

CNS-targeting therapies for lysosomal storage diseases: current advances and challenges

MJ Edelmann, GHB Maegawa - Frontiers in molecular biosciences, 2020 - frontiersin.org
During the past decades, several therapeutic approaches have been developed and made
rapidly available for many patients afflicted with lysosomal storage disorders (LSDs), inborn …

Getting drugs to the brain: advances and prospects of organic nanoparticle delivery systems for assisting drugs to cross the blood–brain barrier

Q Tan, S Zhao, T Xu, Q Wang, M Lan, L Yan… - Journal of materials …, 2022 - pubs.rsc.org
The blood–brain barrier (BBB) plays an irreplaceable role in protecting the central nervous
system (CNS) from bloodborne pathogens. However, the BBB complicates the treatment of …

Harnessing protein corona for biomimetic nanomedicine design

Z Chen, X Chen, J Huang, J Wang, Z Wang - Biomimetics, 2022 - mdpi.com
Nanoparticles (NPs) are usually treated as multifunctional agents combining several
therapeutical applications, like imaging and targeting delivery. However, clinical translation …

Bringing enzymes to the proximity party

GS Tender, CR Bertozzi - RSC Chemical Biology, 2023 - pubs.rsc.org
Enzymes are used to treat a wide variety of human diseases, including lysosomal storage
disorders, clotting disorders, and cancers. While enzyme therapeutics catalyze highly …