Results 201 to 210 of about 2,058,692 (279)

In Utero HSC Transplantation for Sickle Cell Disease: A Potential Therapeutic Approach That Overcomes Complications of Current Therapies

Prenatal Diagnosis, EarlyView.
ABSTRACT Sickle cell disease (SCD) affects millions worldwide but has limited treatment options, most of which carry significant side effects. At present, the only curative treatment for SCD is allogeneic or gene‐modified autologous hematopoietic stem cell (HSC) transplantation (Tx).
Oluwaseun O. Babatunde, Madeleine G. Bibby, Anthony Atala, Graça Almeida‐Porada, Christopher D. Porada   +4 more
wiley   +1 more source

PEG-lipid micelles enable cholesterol efflux in Niemann-Pick Type C1 disease-based lysosomal storage disorder. [PDF]

Sci Rep, 2016
Brown A, Patel S, Ward C, Lorenz A, Ortiz M, DuRoss A, Wieghardt F, Esch A, Otten EG, Heiser LM, Korolchuk VI, Sun C, Sarkar S, Sahay G.   +13 more
europepmc   +1 more source

Liquiritigenin Ameliorates High‐Fat Diet‐Induced Hepatic Steatosis by Promoting Lipid Droplet Catabolism via PLIN2‐ATGL Regulation

Phytotherapy Research, EarlyView.
Liquiritigenin promotes expression of CGI‐58 and enhances lipolysis by down‐regulating expression of PLIN2, thereby facilitating the catabolism of larger‐sized lipid droplets and producing smaller‐sized lipid droplets, resulting in enhanced activity of lipophagy and ameliorated hepatic steatosis. ABSTRACT Metabolic‐associated fatty liver disease (MAFLD)
Zhuoya Xu, Jingmiao Chen, Hong Qin, Yansong Fu, Cunqi Lv, Zhipeng Wang, Xi Liu, Wenya Zheng   +7 more
wiley   +1 more source

Naturally occurring lysosomal storage disease consistent with neuronal ceroid lipofuscinosis in a group of 5 related captive rhesus macaques (<i>Macaca mulatta</i>). [PDF]

Vet Pathol
Olstad K, Pastenkos G, Barrera-Zarate J, Kanthaswamy S, Sammak R, Lemoy MJ.   +5 more
europepmc   +1 more source

MXene Bioinks for 3D Bioprinting: Design and Translation

Small, EarlyView.
This study establishes a comprehensive framework for MXene‐based bioinks in 3D bioprinting, highlighting the interplay between rheological engineering and electroactive hydrogels. By optimizing electrical percolation and mediated signaling, these formulations drive tissue‐specific biological outcomes and accelerated regeneration.
Begüm Sarac, Seydanur Yücer, Fatih Ciftci   +2 more
wiley   +1 more source

A Ferrous‐Supply‐Regenerating Lipid Nanoparticle Integrating RNAi Induces Ferroptosis for Cancer Therapy

Small, EarlyView.
ABSTRACT Targeting iron‐dependent ferroptosis with siRNA has emerged as a promising strategy for cancer treatment. The concept of ferrous‐supply regeneration, inspired by electro‐Fenton technology, has also gained interest as a non‐apoptotic approach to induce ferroptosis.
Dun Hu, Yun‐Hua Xu, Dan Peng, Qi Tan, Jun‐Lin Jiang, Sheng‐Dan Nie, Shan Wang   +6 more
wiley   +1 more source

Immunomodulatory Nanozymes as Programmable Redox–Immune Set‐Point Regulators

Small, EarlyView.
Conceptual phase space linking reactive oxygen species level/dynamics to immune state, with pathological set‐points for chronic inflammation, tumor escape, and degeneration contrasted against a central healthy redox–immune set‐point. Buffering and amplifying nanozymes are depicted as devices that dial redox tone up or down to steer diseased ...
Sumi Choi, Minjeong Kim, Sang Jin Lee, Su‐Hwan Kim   +3 more
wiley   +1 more source

Endocytosis‐independent cytosolic entry of messenger RNA via fluorous bilayer zippering attenuating Toll‐like receptor signaling and enables ischemic tissue salvage

Smart Molecules, EarlyView.
The present study establishes a paradigm shift in synthetic mRNA delivery through the convergence of three orthogonal chemistries—perfluoro‐acylation, bio‐orthogonal click cross‐linking, and disulfide reduction—within a single, modular polymer architecture. Abstract A fundamental constraint of conventional messenger RNA (mRNA) delivery systems is their
Yue Wang, Haitao Xie, Guoqing Xiang, Yanhua Li, Changgui Tong, Hongyan Cui, Qixian Chen, Haidong Li, Yan Zhao   +8 more
wiley   +1 more source

Neurodegenerative Diseases in Children: A Comprehensive Review. [PDF]

Int J Mol Sci
Ailioaie C, Ailioaie LM, Stan CI, Sava A, Chiran DA.   +4 more
europepmc   +1 more source

Gene-wise association of variants in four lysosomal storage disorder genes in neuropathologically confirmed Lewy body disease. [PDF]

PLoS One, 2015
Clark LN, Chan R, Cheng R, Liu X, Park N, Parmalee N, Kisselev S, Cortes E, Torres PA, Pastores GM, Vonsattel JP, Alcalay R, Marder K, Honig LL, Fahn S, Mayeux R, Shelanski M, Di Paolo G, Lee JH.   +18 more
europepmc   +1 more source