Results 271 to 280 of about 4,690,905 (353)

Harnessing Frémy's salt for tyrosine-directed bioconjugations. [PDF]

RSC Adv
Samuels ZV, Stoddard A, Mark Kao WS, Cornejo MA, Strugala E, McGlone SA, Furer L, Rodriguez C, Zeglis BM.   +8 more
europepmc   +1 more source

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

Balancing personalized medicine and scalability in base editing for phenylketonuria. [PDF]

Mol Ther Nucleic Acids
Nicosia L.
europepmc   +1 more source

In‐Depth Profiling Highlights the Effect of Efgartigimod on Peripheral Innate and Adaptive Immune Cells in Myasthenia Gravis

Annals of Clinical and Translational Neurology, EarlyView.
ABSTRACT Background Myasthenia gravis (MG) is an autoimmune disorder characterized by antibody‐mediated complement activation. Efgartigimod, a neonatal Fc receptor (FcRn) antagonist, is approved for treating generalized MG (gMG). However, its modulatory effects on upstream innate and adaptive immune cells remain largely unexplored.
Lei Jin, Liu Dong, Ying Wang, Dingxian He, Chong Yan, Jianying Xi, Haoqin Jiang, Yu Shi, Ming Guan, Chongbo Zhao, Jie Song, Sushan Luo   +11 more
wiley   +1 more source

Toward an age of CRISPR delivery with non-viral biologics. [PDF]

Mol Ther Nucleic Acids
Tompkins J, Ray RM, Scott TA.
europepmc   +1 more source

In Vivo Assessment of Benzoporphyrin Uptake and Singlet Oxygen Generation in Mice for Photodynamic Therapy Monitoring. [PDF]

ACS Photonics
Vikas V, Lu B, Yang W, Wilson BC, Zhu TC, Hadfield RH.   +5 more
europepmc   +1 more source

Drivers of clinical resistance to venetoclax and hypomethylating agents in acute myeloid leukemia and strategies for improving efficacy. [PDF]

Hemasphere
Vänttinen I, Saad J, Ruokoranta T, Kytölä S, Qin G, Tercan B, Ettala P, Partanen A, Pyörälä M, Rimpiläinen J, Siitonen T, Manninen M, Valk PJM, Huls G, Thorsson V, Heckman CA, Kontro M, Kuusanmäki H.   +17 more
europepmc   +1 more source

Strategies of AAV capsid engineering for targeted delivery to brain, muscle, and retina. [PDF]

Front Mol Biosci
Xu X.
europepmc   +1 more source