Results 61 to 70 of about 56,286 (316)

Strategies to Improve the Lipophilicity of Hydrophilic Macromolecular Drugs

Advanced Healthcare Materials, EarlyView.
Hydrophilic macromolecular drugs can be successfully lipidized by covalent attachment of lipids, by hydrophobic ion pairing with negatively or positively charged surfactants, and by dry or wet reverse micelle formation. Lipophilicity enhancement of hydrophilic macromolecules has several benefits including stability and bioavailability improvement ...
Sera Lindner, Stefan Keim, Soheil Haddadzadegan, Odile Fernandez Romero, Katrin Zöller, Gabriel Stern, Ilaria Cesi, Krum Kafedjiiski, Andreas Bernkop‐Schnürch   +8 more
wiley   +1 more source

Primary fibroblasts from CSPα mutation carriers recapitulate hallmarks of the adult onset neuronal ceroid lipofuscinosis [PDF]

, 2017
Mutations in the co- chaperone protein, CSPα, cause an autosomal dominant, adult-neuronal ceroid lipofuscinosis (AD-ANCL). The current understanding of CSPα function exclusively at the synapse fails to explain the autophagy-lysosome pathway (ALP ...
Benitez, Bruno A, Sands, Mark S
core   +2 more sources

Engineered ETS1‐Nanoconjugate Restores Immune Homeostasis through Dual Immune‐Vascular Modulation in Relapsing and Progressive Multiple Sclerosis

Advanced Healthcare Materials, EarlyView.
The biomimetic nanoplatform IMNP (ETS1 pDNA/PBAE@ITP‐MM) undergoes targeted disassembly at inflammatory vascular sites to release the ETS1 plasmid (pETS1). This release initiates a cascade of effects that inhibit pathogenic pathways and support immune homeostasis. (Abbreviations: EndMT, endothelial‐to‐mesenchymal transition; EC, endothelial cell; TC, T
Feng Zhang, Si‐Bo Yang, Yan Luo, Xiao‐Di Sun, Bo‐Hao Chang, Da‐Qiang Zhou, Jun‐Jie Jiang, Ya‐Nan Li, Jie‐Hong Wu, Hui‐Juan Jin, Hang Yang, Hang Hu, Yi‐Fan Zhou, Bo Hu   +13 more
wiley   +1 more source

The use of Ambroxol for the treatment of Gaucher disease: A systematic review

eJHaem
Gaucher disease (GD) is a heterogeneous condition requiring tailored treatment approaches. The aim of this systematic review was to synthesise and evaluate current evidence pertaining to the use of Ambroxol for the treatment of GD.
Diego Agustín Abelleyra Lastoria, Simranjeet Grewal, Derralynn Hughes   +2 more
doaj   +1 more source

Prevalence of Lysosomal Storage Disorders [PDF]

JAMA, 1999
Lysosomal storage disorders represent a group of at least 41 genetically distinct, biochemically related, inherited diseases. Individually, these disorders are considered rare, although high prevalence values have been reported in some populations. These disorders are devastating for individuals and their families and result in considerable use of ...
Meikle, P., Hopwood, J., Clague, A., Carey, W.   +3 more
openaire   +3 more sources

Insights into molecular mechanisms of disease in Neurodegeneration with Brain Iron Accumulation; unifying theories. [PDF]

, 2016
Neurodegeneration with brain iron accumulation (NBIA) is a group of disorders characterised by dystonia, parkinsonism and spasticity. Iron accumulates in the basal ganglia and may be accompanied by Lewy bodies, axonal swellings and hyperphosphorylated ...
Adibhatla, Aghajanian, Alazami, Alderson, Andrews, Balboa, Balsinde, Barbeito, Beck, Bosveld, Bras, Bras, Breus, Brissot, Broekemeier, Brunetti, Burn, Campanella, Chen, Cozzi, Crichton, Cui, Curtis, Dall'Armi, Daugherty, Davis, Deas, Dehay, Deng, Deschauer, Di Fonzo, Dick, Duran, Dusi, Eckhardt, Edvardson, Elbaz-Alon, Engel, Friedman, Fukunaga, Gadd, Gitler, Gonzalez-Cuyar, Green, Gregory, Gregory, Grünewald, Haack, Hailey, Hallgren, Hama, Hamasaki, Hartig, Hartig, Hayflick, Hayflick, Hill, Hogarth, Hong, Hortnagel, Houlden, Hönscher, Itoh, Iuso, Jana, Jenkins, Jeong, Jiang, Jin, Kaneko, Keller, Keogh, Kondo, Kono, Kotzbauer, Kruer, Kruer, Kruer, Kruer, Kurz, Kühlbrandt, Landouré, Larsson, Lei, Leonardi, Leoni, Levi, Li, Liou, Lu, Maday, Mahadevan, Malik, Mancuso, Manjithaya, Mariño, McNeill, Moreno, Morgan, Morita, Nemazanyy, Nousbeck, Oide, Ong, Paisan-Ruiz, Paisán-Ruiz, Park, Perry, Pivtoraiko, Poli, Polishchuk, Potter, Proikas-Cezanne, Ramirez, Ramonet, Rana, Riku, Ross, Ross, Rouault, Rouault, Roy, Saitsu, Scherz-Shouval, Schmidt, Schneider, Schneider, Seleznev, Shinzawa, Siudeja, Song, Strokin, Strokin, Tan, Tooze, Tsunemi, Usenovic, Vidal, Walia, Williams, Wu, Xu, Yadrick, Yang, Yoshida, Yoshida, Zhang, Zhou, Zorzi   +148 more
core   +1 more source

Ferritin‐Doped Nanoparticles Triggered Tumor‐Specific Darkening for Enhanced Photothermal Tumor Ablation and Immune Activation

Advanced Healthcare Materials, EarlyView.
Ferritin‐doped hybrid nanoparticles triggered accurate tumor‐specific hemorrhage for enhanced in situ photothermal therapy and evoked anti‐tumor immunity. Notably, HFn/GA‐Fe specifically induced tumor hemorrhage 12 h after intravenous injection, resulting in visible color darkening. Through photoacoustic imaging, tumor vessels could be clearly observed
Haidong Zha, Xiao Liang, Long Xi, Jiamao Chen, Wenzhao Zhong, Zhen Yuan, Meng Xu, Ying Zheng   +7 more
wiley   +1 more source

Lyso-IP: Uncovering Pathogenic Mechanisms of Lysosomal Dysfunction

Biomolecules, 2022
Lysosomes are ubiquitous membrane-bound organelles found in all eukaryotic cells. Outside of their well-known degradative function, lysosomes are integral in maintaining cellular homeostasis. Growing evidence has shown that lysosomal dysfunction plays an
Chase Chen, Ellen Sidransky, Yu Chen
doaj   +1 more source

Targeted delivery of lysosomal enzymes to the endocytic compartment in human cells using engineered extracellular vesicles. [PDF]

, 2019
Targeted delivery of lysosomal enzymes to the endocytic compartment of human cells represents a transformative technology for treating a large family of lysosomal storage diseases (LSDs).
Brown, Annie, Do, Mai Anh, Levy, Daniel, Lu, Biao, Marriott, Gerard   +4 more
core   +1 more source

Intrinsically Disordered Protein‐Inspired Nanovector‐Based Coacervates for the Direct Cytosolic Transport of Biomacromolecules

Advanced Materials, EarlyView.
Intrinsically disordered protein‐inspired nanovectors (IDP‐NVs) form stable nanocoacervates (NCs) with diverse biomacromolecules. In situ conformational changes confer stability and adaptability to NCs under dynamically changing physiological conditions.
Soyeong Jin, Hyemin Park, Seuk‐Min Ryu, Dagyeong Guk, Jaeeun Lee, Seongeon Jin, Changjoon Keum, Jinyoung Park, Myoung‐Hwan Park, Chaekyu Kim, Hojun Kim, Jaegeun Noh, Kwan Hyi Lee, Ja‐Hyoung Ryu, Youngdo Jeong   +14 more
wiley   +1 more source