Results 51 to 60 of about 788,142 (267)

Reduced Muscular Carnosine in Proximal Myotonic Myopathy—A Pilot 1H‐MRS Study

Annals of Clinical and Translational Neurology, EarlyView.
ABSTRACT Objective Myotonic dystrophy type 2 (proximal myotonic myopathy, PROMM) is a progressive multisystem disorder with muscular symptoms (proximal weakness, pain, myotonia) and systemic manifestations such as diabetes mellitus, cataracts, and cardiac arrhythmias.
Alexander Gussew, Maryam Kargaran, Maik Rothe, Andreas Deistung, Dietrich Stoevesandt, Walter A. Wohlgemuth, David Strube, Thomas Kendzierski, Anna Katharina Kölsch, Maurits Gerhard Abraham Heuschen, Markus Otto, Alexander Mensch   +11 more
wiley   +1 more source

LOXHD1 and RHOB Expression by Monocytes Predicts Progressive Systemic Sclerosis associated Interstitial Lung Disease

Arthritis Care &Research, Accepted Article.
Objective A leading cause of death among scleroderma (SSc) patients, interstitial lung disease (ILD) remains challenging to prognosticate. The discovery of biomarkers that accurately determine which patients would benefit from close monitoring and aggressive therapy would be an essential clinical tool.
Cristina M Padilla, Robert Lafyatis, Kevin F Gibson, Christina Morse, Eleanor Valenzi, Xiaoping Chen, Xiaoyun Li, Yanwu Zhao, Yongseok Park, Dana Ascherman, Jonathan Minden, Robyn Domsic, Yingze Zhang, Daniel J Kass   +13 more
wiley   +1 more source

Low-density lipoprotein receptor-related protein 2 (LRP2) is required for lipid export in the midgut of the migratory locust, Locusta migratoria

Journal of Integrative Agriculture
Low-density lipoprotein receptor-related protein 2 (LRP2) is a multifunctional endocytic receptor expressed in epithelial cells. In mammals, it acts as an endocytic receptor that mediates the cellular uptake of cholesterol-containing apolipoproteins to ...
Yiyan Zhao, Weimin Liu, Xiaoming Zhao, Zhitao Yu, Hongfang Guo, Yang Yang, Hans Merzendorfer, Kun Yan Zhu, Jianzhen Zhang   +8 more
doaj   +1 more source

Lipid Nanoparticles for the Delivery of CRISPR/Cas9 Machinery to Enable Site‐Specific Integration of CFTR and Mutation‐Agnostic Disease Rescue

Advanced Functional Materials, EarlyView.
Lipid nanoparticles (LNPs) are optimized to co‐deliver Cas9‐encoding messenger RNA (mRNA), a single guide RNA (sgRNA) targeting the endogenous cystic fibrosis transmembrane conductance regulator (CFTR) gene, and homologous linear double‐stranded donor DNA (ldsDNA) templates encoding CFTR.
Ruth A. Foley, Paul G. Ayoub, Vrishti Sinha, Colin Juett, Alicia Sanoyca, Emily C. Duggan, Lindsay E. Lathrop, Priyanka Bhatt, Kevin Coote, Beate Illek, Brigitte N. Gomperts, Donald B. Kohn, Steven J. Jonas   +12 more
wiley   +1 more source

FeDSNP‐Pa Nanoassemblies: A Triple‐Action Therapeutic Strategy Targeting Oxidative Stress, Inflammation, and Pyroptosis for Retinal Ganglion Cell Protection in Glaucoma

Advanced Functional Materials, EarlyView.
FeDSNP‐Pa, a metallized nanoparticle loaded with sodium pyruvate (Pa), exerts triple therapeutic effects by scavenging reactive oxygen species (ROS), suppressing inflammatory responses, and inhibiting pyroptosis signaling pathways. This multifunctional neuroprotective strategy protecting retinal ganglion cells (RGCs) from elevated intraocular pressure ...
Yukun Wu, Xue Li, Tong Wu, Dan Ji, Yugang Bai, Xuezhi Zhou   +5 more
wiley   +1 more source

Peptide–Oligonucleotide Conjugation: Chemistry and Therapeutic Applications

Current Issues in Molecular Biology
Oligonucleotides have been identified as powerful therapeutics for treating genetic disorders and diseases related to epigenetic factors such as metabolic and immunological dysfunctions.
Anna L. Malinowska, Harley L. Huynh, Sritama Bose   +2 more
doaj   +1 more source

Synergistic Osteogenesis After Co‐Administration of cmRNAs Encoding BMP‐2 and BMP‐7 Utilizing a Transcript‐Activated Matrix

Advanced Functional Materials, EarlyView.
This study demonstrates that the dual delivery of BMP‐2/‐7 coding cmRNAs for bone healing is demonstrated as feasible, safe, and highly osteogenic. Compared to single BMP‐2 or BMP‐7 cmRNAs, the combination enhances the production of both mineral and organic components of the extracellular matrix when delivered using a collagen‐HA scaffold, supporting ...
Claudia Del Toro Runzer, Joanna M. Sadowska, Nadia Roumans, Christian Plank, Christopher H. Evans, Fergal J. O'Brien, Martijn van Griensven, Elizabeth R. Balmayor   +7 more
wiley   +1 more source

Challenges and opportunities in technologies and methods for lncRNA structure determination

Cell & Bioscience
Long non-coding RNAs (lncRNAs) play pivotal roles in diverse cellular processes ranging from gene regulation and chromatin remodeling to RNA stability and epigenetic modifications. Despite the identification of approximately 95,000 lncRNA genes in humans,
Maximilia Frazao De Souza Degenhardt, Yun-Xing Wang   +1 more
doaj   +1 more source

CRISPR/Cas9‐Assisted Microrobots for Fast and Ultrasensitive “On‐The‐Fly” Next‐Generation DNA Detection

Advanced Functional Materials, EarlyView.
This work presents self‐propelled CRISPR/Cas9‐functionalized Au–MRs for rapid, amplification‐free, “on‐the‐fly” DNA detection. By harnessing motion‐assisted signal recovery, the platform achieved the limit of detection in low fM DNA concentrations, enabling detection across a wide dynamic range within only 5 min, which is significantly faster than any ...
Jyoti, Petr Michálek, Zbyněk Heger, Martin Pumera   +3 more
wiley   +1 more source

A novel bispecific siRNA concept: Efficient dual knockdown of YAP1 and WWTR1 with a single guide strand

Molecular Therapy: Nucleic Acids
Yes-associated protein (YAP, encoded by YAP1) and transcriptional coactivator with PDZ-binding motif (TAZ, encoded by WWTR1) are master transcriptional regulators in the Hippo pathway. Because of their complementary roles, simultaneously inhibiting these
Kaito Ueda, Yuki Yamada, Hiroyuki Tanaka, Tatsuki Sato, Jumpei Sasaki, Sachiko Sakamoto, Kenjiro Minomi, Hiroshi Yamada, Masayuki Sugimoto   +8 more
doaj   +1 more source