Results 151 to 160 of about 22,003 (247)

Excessive burden of lysosomal storage disorder gene variants in Parkinson's disease. [PDF]

Brain, 2017
Robak LA, Jansen IE, van Rooij J, Uitterlinden AG, Kraaij R, Jankovic J, International Parkinson’s Disease Genomics Consortium (IPDGC), Heutink P, Shulman JM.   +8 more
europepmc   +1 more source

Attitudes Toward Prenatal Interventions in the Fanconi Anemia Community

Prenatal Diagnosis, EarlyView.
ABSTRACT Objective In‐utero cell and gene therapies may offer prenatal treatment options for inherited diseases. Preclinical data suggests in‐utero (IU) hematopoietic stem cell transplantation (HSCT) could prevent Fanconi anemia (FA) related bone marrow failure without genotoxic conditioning or immune suppression.
Tony Lum, Catherine Lee, Tippi MacKenzie, Billie Lianoglou, Agnieszka Czechowicz   +4 more
wiley   +1 more source

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

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

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

Integrative Bioinformatics, Experimental Validation, and Interpretable Machine Learning Reveal Oxyresveratrol‐Mediated Protection Against Cadmium‐Induced Lung Adenocarcinoma‐Related Transcriptional Dysregulation

Environmental Toxicology, EarlyView.
ABSTRACT Cadmium (Cd) is a toxic heavy metal strongly implicated in lung adenocarcinoma (LUAD) through mechanisms involving oxidative stress, epigenetic dysregulation, and chronic inflammation. This study aimed to identify Cd‐responsive genes associated with LUAD and to evaluate the protective effects of oxyresveratrol (O‐RES) against Cd‐induced ...
Murat Isıyel, Hamid Ceylan, Yeliz Demir
wiley   +1 more source

Iron Physiology and Its Impact on Atopic Diseases: An EAACI Taskforce Report

Allergy, EarlyView.
ABSTRACT Iron is essential for oxygen transport, energy metabolism, and immune regulation. Yet iron deficiency is the most common micronutrient disorder across all age groups, affecting nearly one quarter of the global population. Iron deficiency triggers nutritional immunity, a host defense mechanism that withholds and redistributes iron, contributing
Franziska Roth‐Walter, Ioana Agache, Beatriz Cabanillas, Roberto Berni Canani, Pasquale Comberiati, Holger Garn, Cristina Gomez‐Casado, Karin Hufnagl, Ekaterina Khaleva, Gregorio P. Milani, Daniel Munblit, Nikolaos Douladiris, Liam O'Mahony, Frank Redegeld, Carmen Riggioni, Peter K. Smith, Betty van Esch, Emilia Vassilopoulou, Carina Venter, Diego G. Peroni   +19 more
wiley   +1 more source

Relative acidic compartment volume as a lysosomal storage disorder-associated biomarker. [PDF]

J Clin Invest, 2014
te Vruchte D, Speak AO, Wallom KL, Al Eisa N, Smith DA, Hendriksz CJ, Simmons L, Lachmann RH, Cousins A, Hartung R, Mengel E, Runz H, Beck M, Amraoui Y, Imrie J, Jacklin E, Riddick K, Yanjanin NM, Wassif CA, Rolfs A, Rimmele F, Wright N, Taylor C, Ramaswami U, Cox TM, Hastings C, Jiang X, Sidhu R, Ory DS, Arias B, Jeyakumar M, Sillence DJ, Wraith JE, Porter FD, Cortina-Borja M, Platt FM.   +35 more
europepmc   +1 more source

Red lines and green lights: Gene therapy for inherited erythroid disorders beyond the haemoglobinopathies

British Journal of Haematology, EarlyView.
Gene therapy is revolutionizing treatment paradigms for haemoglobinopathies, establishing a translational framework for disorders that impact red blood cell development. In their paper, Joshi et al. describe the preclinical and early clinical landscape of gene therapies for non‐haemoglobinopathy erythroid disorders and highlight common thematic ...
Gaurav Joshi, Nandakumar Natarajan, Kathryn E. Dickerson, Richard A. Voit   +3 more
wiley   +1 more source