Results 71 to 80 of about 5,162 (179)

Tri‐Parametric Assessment of α‐Galactosidase A Activity, lysoGb3 and X‐Inactivation Aids Genotype‐Phenotype Categorization of Fabry Disease Female Patients

Journal of Inherited Metabolic Disease, Volume 49, Issue 4, July 2026.
ABSTRACT Fabry disease (FD, OMIM 301500) is an X‐linked lysosomal storage disorder caused by deficient activity of lysosomal alpha‐galactosidase A (AGAL, E.C. 3.2.1.22) due to pathogenic variants in the GLA gene (HGNC:4296, Xq22.1). Plasmatic deacylated globotriaosylceramide (lysoGb3) is elevated in FD patients as a reflection of lysosomal accumulation
Ladislav Kuchar, Lenka Dvorakova, Linda Berna, Martin Reboun, Petra Slavikova, Radovan Bakalar, Jana Ledvinova, Helena Poupetova, Petr Ruzicka, Asfaw Befekadu, Gabriela Dostalova, Stella Reichmannova, Ales Linhart, Jakub Sikora   +13 more
wiley   +1 more source

Design of Nanocarriers for Kidney Targeted Delivery of Nucleic Acid Therapeutics

Macromolecular Bioscience, Volume 26, Issue 7, July 2026.
Nucleic acid therapeutics have been investigated to expand their applications to renal genetic disorders. This review summarizes key considerations in the design and fabrication of nanocarriers for the systemic delivery of nucleic acid therapeutics to the kidneys.
Jun Hyuk Lee, Junku Han, Sehwan Park, Hyejung Mok   +3 more
wiley   +1 more source

Examination of the Usability of Anti-Gb3 Antibody（1A4）for Detection of Gb3 [PDF]

, 2018
globotriaosylceramide（gb3）is the most important cell surface ligand of the rhamnose-binding lectins such as Sirulus asotus lectin（SaL）．SaL recognizes and binds to gb3 expressing tumor cells such as Burkitts lymphoma raji cells and induces them to the g0 ...
細野, 雅祐, ホンダ, ショウタ, 立田, 岳生, Honda, Shota, タツタ, タケオ, Ito, Jun, Sugawara, Shigeki, タカノハシ, ショウコ, Takanohashi, Shoko, スガラワ, シゲキ, 大山, 力, 本田, 捷太, Sato, Makoto, 伊藤, 淳, イトウ, ジュン, 菅原, 栄紀, 鷹觜, 祥子, オオヤマ, チカラ, Tatsuta, Takeo, Hosono, Masahiro, ホソノ, マサヒロ, Ohyama, Chikara, 佐藤, 信, サトウ, マコト   +23 more
core   +1 more source

Septins in the Middle—Makers and Breakers of Membrane Contact Sites

Journal of Neurochemistry, Volume 170, Issue 6, June 2026.
Septins are a family of GTP‐binding proteins that assemble into heteromeric oligomers and polymers, associating with specific membrane domains and organelles according to their subunit composition. Growing evidence places septins at membrane contact sites (MCS) — key hubs for intracellular communication that mediate exchange of ions, lipids, and ...
TrishaJean J. Holt, Elias T. Spiliotis
wiley   +1 more source

Differential recognition of lipid domains by two Gb3-binding lectins

, 2020
The two lectins LecA from Pseudomonas aeruginosa and the B-subunit of Shiga toxin from Shigella dysenteriae (StxB) share the glycosphingolipid globotriaosylceramide (Gb3) as receptor.
Römer, Winfried, Ries, Annika, Patalg, Lukas J., Schubert, Thomas, Werz, Daniel B., Brandel, Annette, Mely, Yves, Sych, Taras, Madl, Josef, Patalag, Lukas J., Thuenauer, Roland, Steinem, Claudia, Roemer, Winfried, Xu, Maokai, Kettelhoit, Katharina, Omidvar, Ramin, Thünauer, Roland   +16 more
core   +1 more source

Shiga toxin type-2 (Stx2) induces glutamate release via phosphoinositide 3-kinase (PI3K) pathway in murine neurons.

Frontiers in Molecular Neuroscience, 2015
Shiga toxin-producing Escherichia coli (STEC) can cause central nervous system (CNS) damage resulting in paralysis, seizures, and coma. The key STEC virulence factors associated with systemic illness resulting in CNS impairment are Shiga toxins (Stx ...
Fumiko eObata, Fumiko eObata, Lauren M Hippler, Progyaparamita eSaha, Dakshina M Jandhyala, Olga eLatinovic, Olga eLatinovic   +6 more
doaj   +1 more source

Development of CAR NK Cell Lines Selectively Targeting Cancer Cells Expressing Membrane Hsp70

MedComm, Volume 7, Issue 5, May 2026.
This work presents a novel CAR NK cell platform targeting membrane‐bound Hsp70, a tumor‐specific antigen broadly expressed on solid tumors but not on normal cells. Computational modeling confirmed strong binding between the CAR construct and the Hsp70‐derived TKD peptide.
Khouloud Hachani, Mina Yazdi, Charlotte Carcopino, Fatemeh Khademi Moghadam, Micholas Dean Smith, Anskar Trill, Marcel P. Trefny, Morteza Hasanzadeh Kafshgari, Cosima C. Hoch, Abdallah Gaballa, Bayan Alkotub, Jennifer Altomonte, A. Graham Pockley, Ernst Wagner, Barbara Wollenberg, Sebastian Kobold, Gabriele Multhoff, Ali Bashiri Dezfouli   +17 more
wiley   +1 more source

Supply and Demand in the Mathematics of Rare Disease Drug Development: Why Choosing the Right Model Is Crucial

Clinical and Translational Science, Volume 19, Issue 5, May 2026.
ABSTRACT Clinical trials for rare diseases face a fundamental mathematical challenge that conventional randomized controlled trial (RCT) designs cannot overcome. With approximately 95% of the estimated 10,000–16,000 rare diseases lacking approved therapies, and drug development programs failing at rates exceeding 75% in non‐oncology indications, the ...
Marshall L. Summar, Janet Woodcock
wiley   +1 more source

Globotriaosylceramide leads to KCa3.1 channel dysfunction: A new insight into endothelial dysfunction in Fabry disease

, 2021
AimsExcessive endothelial globotriaosylceramide (Gb3) accumulation is associated with endothelial dysfunction and impaired endothelium-dependent relaxation in Fabry disease.
한기환, 정성철, 최신규, 서석효, 박성희   +4 more
core   +1 more source

Deciphering the Chemotherapeutic Mechanism of Ferulic Acid: Insight Into the Role Against Multiple Human Cancers

Cancer Innovation, Volume 5, Issue 2, April 2026.
Ferulic acid (FA), a naturally occurring compound, exhibits anticancer activity against 16 cancer types through multiple molecular pathways. Its enhanced delivery via nanoformulations and synergistic effects with other therapies suggest FA as a promising, food‐based adjunct for cancer symptom control until fully approved pharmaceutical options are ...
Sanzida Khatun, Md Sohel, Zitu Barman, Umme Salma, Lubatul Arbia, Md. Rifat Sarker, Jasmin Akter Jame, Badhan Rani Dey, Sultana Parvin, Md. Shah Poran Shuvo, Md. Shahidul Islam, Tania Mannan, Snygdha Rani Das, Md. Mahmudul Hasan   +13 more
wiley   +1 more source