Results 151 to 160 of about 22,029 (242)

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

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

Lysosomal Storage Disorders

, 2019
Daniel Bell, Sepehr Haghighi, Tristan Skalina   +2 more
openaire   +2 more sources

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

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

Mapping Sanfilippo Syndrome: A Multisystem Clinicopathological Autopsy. [PDF]

Diagnostics (Basel)
Trandafirescu MF, Avădănei ER, Filip N, Saveanu CI, Foia I, Toma V, Baroi LG, Anton-Paduraru DT, Moisa SM, Lozneanu L.   +9 more
europepmc   +1 more source