Results 131 to 140 of about 40,385 (227)

Purifying and profiling lysosomes to expand understanding of lysosomal dysfunction–associated diseases

The Journal of Clinical Investigation
Lysosome storage dysfunction plays a central role in numerous human diseases, but a lack of appropriate tools has hindered lysosomal content profiling in clinical settings. In this issue of the JCI, Saarela et al. introduce a method called tagless LysoIP
Ali Shilatifard, Issam Ben-Sahra
doaj   +1 more source

Plasma membrane remodeling in GM2 gangliosidoses drives synaptic dysfunction.

PLoS Biology
Glycosphingolipids (GSL) are important bioactive membrane components. GSLs containing sialic acids, known as gangliosides, are highly abundant in the brain and diseases of ganglioside metabolism cause severe early-onset neurodegeneration. The ganglioside
Alex S Nicholson, David A Priestman, Robin Antrobus, James C Williamson, Reuben Bush, Shannon J McKie, Henry G Barrow, Emily Smith, Kostantin Dobrenis, Nicholas A Bright, Frances M Platt, Janet E Deane   +11 more
doaj   +1 more source

Infusion rate adjustment in enzyme replacement therapy with pabinafusp alfa for mucopolysaccharidosis II

British Journal of Clinical Pharmacology, EarlyView.
Abstract Aims Enzyme replacement therapy (ERT) for mucopolysaccharidosis II (MPS II) requires long‐term, weekly intravenous infusions often lasting over 3 h each time, which can burden paediatric patients and caregivers and negatively affect their quality of life and treatment compliance.
Kimitoshi Nakamura, Norio Sakai, Hideaki Hirai, Naoko Takasao, Ryo Ibaraki, Tatsuyoshi Yamamoto, Yuji Sato   +6 more
wiley   +1 more source

Gene therapy for lysosomal storage diseases: Current clinical trial prospects. [PDF]

Front Genet, 2023
Kido J, Sugawara K, Nakamura K.
europepmc   +1 more source

Overcoming intranasal delivery barriers with ultrastable polyzwitterionic siRNA nanocages for enhanced glioblastoma therapy

BMEMat, EarlyView.
A ROS‐responsive, zwitterionic nanocage enables stable, intranasal siRNA delivery to glioblastoma, promoting deep tumor penetration via non‐degradative pathways and trigeminal nerve transport. This platform achieves durable gene silencing and tumor suppression, offering a non‐invasive, storage‐stable strategy for treating glioma and other neurological ...
Jingwen Xie, Jingxing Si, Xiaobei Deng, Jing Tan, Ruijie Zhu, Jizhou Qi, Gaoxia Ma, Bin Wang, Yi Wang, Yunzhou Guo, Yuping Chen, Shiqun Shao, Youqing Shen   +12 more
wiley   +1 more source

Carrier‐free self‐assembled nanomedicine for combination‐therapy of acute myeloid leukemia

BMEMat, EarlyView.
CPDS combines drugs with three different mechanisms of action to achieve a multi‐mechanism combination therapy for AML by directly killing tumor cells and activating anti‐tumor immunity. Abstract As the main acute myeloid leukemia (AML) clinical treatment, the chemotherapy alone cannot meet the clinical therapeutic needs due to the high heterogeneity ...
Meihong Chai, Rui Zhang, Haiyin Yang, Jiyue Xing, Tianyuan Hu, Mengxi Zhu, Rui Li, Xiaoli Chen, Beibei Zhao, Yang Lan, Stefan Barth, Yuanyu Huang, Li Zhang, Mengjie Zhang, Huining He   +14 more
wiley   +1 more source

Combined Therapies for Lysosomal Storage Diseases

, 2015
Lysosomal storage diseases (LSDs) is a group consisting of over 50 disorders caused mostly by dysfunctions of lysosomal proteins and resultant accumulation of particular compounds inside cells and extracellular volumes in affected organisms.
Kloska, Anna, Moskot, Marta, Piotrowska, Ewa, Węgrzyn, Alicja, Węgrzyn, Grzegorz, Gabig-Cimińska, Magdalena, Jakóbkiewicz-Banecka, Joanna, Malinowska, Marcelina, Chmielarz, Izabela   +8 more
core   +1 more source

Harnessing ferroptosis from multilayer defense networks to nanoplatforms for specific cancer therapy

BMEMat, EarlyView.
Nanomaterials target metabolically‐regulated ferroptosis for cancer therapy. Iron‐based or alternative nanoplatforms integrate ferroptosis with chemotherapy, immunotherapy, or radiotherapy. They enable stimulus‐responsive therapies (photothermal, photodynamic, sonodynamic) activated by near‐infrared, light, or ultrasound, achieving potent synergistic ...
Xinyue Xu, Xuejian Wang, Wenyan Jiang, Wan Hairul Anuar Kamaruddin, Chunyan Fang, Pengfei Zhang   +5 more
wiley   +1 more source

Transfersomes with core and surface‐loaded NF‐κB p65 siRNA for enhanced transdermal transfection and effective treatment of psoriasis

BMEMat, EarlyView.
TCPL, a transfersome with Tween 80 and PEI, enables efficient transdermal siNF‐κB delivery, resolving PEI's toxicity issue. It promotes lysosomal escape and effective transfection, silences NF‐κB, modulates immunity, and inhibits angiogenesis, alleviating psoriasis. With good biocompatibility, TCPL holds strong clinical translation potential. Schematic
Hui Xing, Yuhui Yang, Ziyi Zhao, Kai Lv, Yiran Shao, Zhi Zhang, Dong Ma   +6 more
wiley   +1 more source

Population Pharmacokinetic/Pharmacodynamic Modeling of Therapeutic Enzymes in Lysosomal Storage Diseases. [PDF]

Clin Pharmacokinet
Barzel I, van der Ploeg AT, Pijnappel WWMP, van der Kuy PHM, van den Hout JMP, Preijers T.   +5 more
europepmc   +1 more source