Results 61 to 70 of about 3,638 (152)

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

The effects of globotriaosylceramide tail saturation level on bilayer phases

Soft Matter, 2015
The globotriaosylceramide acyl chains from one leaflet interdigitate into the opposing leaflet and lead to significant bilayer rigidification and immobilisation of the lipid tails. Globotriaosylceramide with saturated acyl chains can form a highly ordered, relatively immobile phase which is resistant to bending.
Weria Pezeshkian, Vitaly V. Chaban, Ludger Johannes, Julian Shillcock, John H. Ipsen, Himanshu Khandelia   +5 more
openaire   +4 more sources

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

Self‐Immolative Systems in Diagnostic and Therapeutic Applications

ChemMedChem, Volume 21, Issue 5, 13 March 2026.
Self‐Immolative Systems enable the precise, residue‐free release of active molecules, drugs, probes, or sensors, via cyclization or elimination, triggered by specific stimuli. Their versatility drives advances in drug delivery, diagnostics, nanotechnology, materials chemistry, and biosensing, offering chemists control over molecular activation and ...
Windbedema Prisca Ouédraogo, Thomas Cailly, Valérie Collot   +2 more
wiley   +1 more source

Globotriaosylceramide, Gb3, is an alternative functional receptor for Shiga-like toxin 2e [PDF]

Infection and Immunity, 1995
We reexamined the binding specificity of the Shiga-like toxin variant associated with porcine edema disease, SLT2e, which is reported to be more cytotoxic for Vero cells than for HeLa cells, by using receptor-deficient cells and a liposomal insertion system for purified glycolipids.
G T, Keusch, M, Jacewicz, D W, Acheson, A, Donohue-Rolfe, A V, Kane, R H, McCluer   +5 more
openaire   +2 more sources

RNA‐Based Therapies for Inherited Metabolic Disorders

Journal of Inherited Metabolic Disease, Volume 49, Issue 2, March 2026.
ABSTRACT Inherited metabolic disorders (IMDs) are a diverse and complex group of genetic conditions resulting from deficiencies in enzymes, transporters, or cofactors. These deficiencies lead to metabolic dysfunction and severe clinical consequences. Despite significant progress in understanding their molecular basis, treatment options remain limited ...
Reddy Sreekanth Vootukuri, Sonam Gurung, Roopkatha Ghosh, Philippa B. Mills, Julien Baruteau, Haiyan Zhou   +5 more
wiley   +1 more source

Nephropathy in Fabry disease: possibilities for the radical improvement of prognosis for orphan diseases

Терапевтический архив, 2013
The paper discusses the specific features of the diagnosis and treatment of kidney involvement in patients with Fabry disease.
V V Fomin, A A Pulin, N A Mukhin
doaj  

A Meta‐Analysis to Unveil the Diagnostic Gaps in Anderson–Fabry Disease in Women

Journal of Inherited Metabolic Disease, Volume 49, Issue 2, March 2026.
ABSTRACT Anderson–Fabry disease (AFD) is an X‐linked lysosomal storage disorder caused by mutations in the GLA gene, leading to deficient α‐galactosidase A activity. Although historically considered a male disease, it is now recognized that heterozygous women can present with a wide range of symptoms. However, diagnosis in women remains challenging, as
L. Lenzini, G. Pintus, G. Gugelmo, A. P. Burlina, G. P. Fadini, A. B. Burlina, N. Vitturi   +6 more
wiley   +1 more source

Migalastat improves diarrhea in patients with Fabry disease: clinical-biomarker correlations from the phase 3 FACETS trial

Orphanet Journal of Rare Diseases, 2018
Background Fabry disease is frequently characterized by gastrointestinal symptoms, including diarrhea. Migalastat is an orally-administered small molecule approved to treat the symptoms of Fabry disease in patients with amenable mutations.
Raphael Schiffmann, Daniel G. Bichet, Ana Jovanovic, Derralynn A. Hughes, Roberto Giugliani, Ulla Feldt-Rasmussen, Suma P. Shankar, Laura Barisoni, Robert B. Colvin, J. Charles Jennette, Fred Holdbrook, Andrew Mulberg, Jeffrey P. Castelli, Nina Skuban, Jay A. Barth, Kathleen Nicholls   +15 more
doaj   +1 more source