Results 71 to 80 of about 362,296 (275)
The importance of genetic diagnosis for Duchenne muscular dystrophy
Journal of Medical Genetics, 2016 Duchenne muscular dystrophy (DMD) and Becker muscular dystrophy are caused by mutations in the dystrophin-encoding DMD gene. Large deletions and duplications are most common, but small mutations have been found as well.
Annemieke Aartsma-Rus +2 more
semanticscholar +1 more source
Progressive Insights into 3D Bioprinting for Corneal Tissue Restoration
Advanced Healthcare Materials, EarlyView.This review explores the potential of 3D bioprinting to replicate the complex structure and function of the human cornea. It highlights key advances in bioink development, printing modalities, and in vivo performance, while addressing current challenges and emerging strategies. The review emphasizes bioprinting's promise to overcome donor shortages and
Ilayda Namli +6 more
wiley +1 more source
Advanced Healthcare Materials, EarlyView.This study presents guanidinium‐ and indole‐functionalized polyphosphoesters as degradable, non‐viral gene delivery vectors. Through precise tuning of charge density and hydrophobicity, these polymers form stable polyplexes with low toxicity. Remarkably, minor structural changes yield up to 200‐fold differences in transfection efficiency, highlighting ...
Markus Kötzsche +8 more
wiley +1 more source
Advanced Materials, EarlyView.Helper and ionizable lipids play a crucial role in determining ApoE binding and subsequent liver tropism and LDLR‐mediated uptake. Ionizable lipids primarily govern the LDLR‐independent uptake pathway. This complementary interplay between lipid components ultimately governs LNP delivery performance and therapeutic efficacy in the liver.
Ashish Sarode +16 more
wiley +1 more source
Advanced Science, EarlyView.Impaired anti‐tumor immunity is mediated by TREM2 + macrophages in non‐small cell lung cancer. TREM2 + macrophages prevent anti‐tumor immunity depending on the limitation of CD4 + T and NK cells. TREM2 promotes cholesterol efflux via ABCA1 to limit the production of CX3CL1 in macrophages.
Yunhan Wang +9 more
wiley +1 more source
Cooperative Brønsted Acid and Photo‐Promoted Stereoselective Synthesis of Substituted Piperidones
Angewandte Chemie, EarlyView.A Brønsted acid cooperates with a photoexcited carbonyl triplet to promote stereoselective C─C bond formation in disubstituted piperidones through a chromophore‐activator strategy. The Brønsted acid lowers the excitation energy gap, directs selective C─N bond cleavage over C─C fragmentation after H‐atom transfer, and enables enantioselective C─C bond ...
Qiupeng Peng +5 more
wiley +2 more sources
Quantifying fat replacement of muscle by quantitative MRI in muscular dystrophy
Journal of Neurology, 2017 The muscular dystrophies are rare orphan diseases, characterized by progressive muscle weakness: the most common and well known is Duchenne muscular dystrophy which affects young boys and progresses quickly during childhood.
J. Burakiewicz +5 more
semanticscholar +1 more source
LMO7 Suppresses Tumor‐Associated Macrophage Phagocytosis of Tumor Cells Through Degradation of LRP1
Advanced Science, EarlyView.LMO7 in tumor‐associated macrophages suppresses phagocytosis of tumor cells and limits cytotoxic T lymphocytes infiltration, fostering tumor progression. Mechanistically, LMO7 mediates the ubiquitination and degradation of the phagocytic receptor LRP1, impairing its ability to engulf tumor cells and driving macrophages toward an antitumor phenotype ...
Mengkai Li +12 more
wiley +1 more source
Long-term microdystrophin gene therapy is effective in a canine model of Duchenne muscular dystrophy
Nature Communications, 2017 Duchenne muscular dystrophy (DMD) is an incurable X-linked muscle-wasting disease caused by mutations in the dystrophin gene. Gene therapy using highly functional microdystrophin genes and recombinant adeno-associated virus (rAAV) vectors is an ...
Caroline Le Guiner +34 more
semanticscholar +1 more source
Advanced Science, EarlyView.Polymeric carriers provide versatile platforms for nucleic acid delivery. This review introduces a novel perspective inspired by protein structural organization to explore structure–function relationships of polymeric carriers, highlighting requirements of different nucleic acid payloads.
Hanieh Moradian +3 more
wiley +1 more source