Results 61 to 70 of about 1,547,751 (329)
This study reveals how prime editing guide RNA (pegRNA) secondary structure and reverse transcriptase template length affect prime editing efficiency in correcting the phospholamban R14del cardiomyopathy‐associated mutation. Insights support the design of structurally optimized enhanced pegRNAs for precise gene therapy.
Bing Yao+7 more
wiley +1 more source
Medicinal chemistry plays a fundamental and underlying role in chemical biology, pharmacology, and medicine to discover safe and efficacious drugs.
A. Gioiello+3 more
semanticscholar +1 more source
Cyclic nucleotide signaling as a drug target in retinitis pigmentosa
Disruptions in cGMP and cAMP signaling can contribute to retinal dysfunction and photoreceptor loss in retinitis pigmentosa. This perspective examines the mechanisms and evaluates emerging evidence on targeting these pathways as a potential therapeutic strategy to slow or prevent retinal degeneration.
Katri Vainionpää+2 more
wiley +1 more source
Boronic Acids and Their Derivatives in Medicinal Chemistry: Synthesis and Biological Applications
Boron containing compounds have not been widely studied in Medicinal Chemistry, mainly due to the idea that this group could confer some toxicity. Nowadays, this concept has been demystified and, especially after the discovery of the drug bortezomib, the
M. Silva+3 more
semanticscholar +1 more source
Exposure to common noxious agents (1), including allergens, pollutants, and micro‐nanoplastics, can cause epithelial barrier damage (2) in our body's protective linings. This may trigger an immune response to our microbiome (3). The epithelial barrier theory explains how this process can lead to chronic noncommunicable diseases (4) affecting organs ...
Can Zeyneloglu+17 more
wiley +1 more source
Nitrogen containing heterocycles are of immense research interest because they are often found as naturally occurring bioactive compounds. The prominence of N-heterocycles makes it vital to develop methods to increase their synthetic efficiencies and ...
M. Henary+5 more
semanticscholar +1 more source
Three‐dimensional (3D) biological systems have become key tools in lymphoma research, offering reliable in vitro and ex vivo platforms to explore pathogenesis and support precision medicine. This review highlights current 3D non‐Hodgkin lymphoma models, detailing their features, advantages, and limitations, and provides a broad perspective on future ...
Carla Faria+3 more
wiley +1 more source
Aryl Phosphoramidates of 5-Phospho Erythronohydroxamic Acid, A New Class of Potent Trypanocidal Compounds [PDF]
RNAi and enzymatic studies have shown the importance of 6-phosphogluconate dehydrogenase (6-PGDH) in Trypanosoma brucei for the parasite survival and make it an attractive drug target for the development of new treatments against human African ...
Alibu, Vincent P.+6 more
core +3 more sources
From omics to AI—mapping the pathogenic pathways in type 2 diabetes
Integrating multi‐omics data with AI‐based modelling (unsupervised and supervised machine learning) identify optimal patient clusters, informing AI‐driven accurate risk stratification. Digital twins simulate individual trajectories in real time, guiding precision medicine by matching patients to targeted therapies.
Siobhán O'Sullivan+2 more
wiley +1 more source
The anabolic steroid stanozolol is a potent inhibitor of human MutT homolog 1
MutT homolog 1 (MTH1) is a member of the NUDIX superfamily of enzymes and is an anticancer drug target. We show that stanozolol (Stz), an anabolic steroid, is an unexpected nanomolar inhibitor of MTH1. The X‐ray crystal structure of the human MTH1–Stz complex reveals a unique binding scaffold that could be utilized for future inhibitor development ...
Emma Scaletti Hutchinson+7 more
wiley +1 more source