Diversifying the structure of zinc finger nucleases for high-precision genome editing
Genome editing often requires cleavage within a narrow sequence window. Here the authors develop an expanded set of zinc finger nuclease architectures that increase the available configurations by a factor of 64 and can target almost every base at loci ...
David E. Paschon+18 more
doaj +1 more source
Heart failure with preserved ejection fraction (HFpEF) accounts for half of the heart failure cases. It is characterised by microvascular dysfunction, associated with reduced pericyte coverage and diminished STAT3 expression in pericytes. Loss of STAT3 impairs pericyte adhesion, promotes senescence, and activates a pro‐fibrotic gene program.
Leah Rebecca Vanicek+15 more
wiley +1 more source
Dysregulation of master regulator c-MYC (MYC) plays a central role in hepatocellular carcinoma (HCC) and other cancers but remains an elusive target for therapeutic intervention.
William Senapedis+11 more
doaj +1 more source
A quantitative study on the role of TKI combined with Wnt/$β$-catenin signaling and IFN-$α$ in the treatment of CML through deterministic and stochastic approaches [PDF]
We propose deterministic and stochastic models for studying the pharmacokinetics of chronic myeloid leukemia (CML), upon administration of IFN-$\alpha$ (the traditional treatment for CML), TKI (the current frontline medication for CML) and Wnt/$\beta$-catenin signaling (the state-of-the art therapeutic breakthrough for CML).
arxiv +1 more source
Bitter Taste Receptors for Asthma Therapeutics. [PDF]
Clinical management of asthma and chronic obstructive pulmonary disease (COPD) has primarily relied on the use of beta 2 adrenergic receptor agonists (bronchodilators) and corticosteroids, and more recently, monoclonal antibody therapies (biologics ...
Deshpande, Deepak A.+3 more
core +1 more source
Image-guided therapy using maghemite-MOF nanovectors [PDF]
Advances in nanotechnology offer the possibility of tailored delivery of therapeutics with real-time imaging of disease. In this issue of Chem, Steunou and co-workers amalgamate the powerful MRI properties of ultra-small paramagnetic iron oxides with the
Abánades Lázaro, Isabel+1 more
core +1 more source
Ion channel function of polycystin‐2/polycystin‐1 heteromer revealed by structure‐guided mutagenesis
Mutations in polycystin‐1 (PC1) or polycystin‐2 (PC2) cause autosomal‐dominant polycystic kidney disease (ADPKD). We generated a novel gain‐of‐function PC2/PC1 heteromeric ion channel by mutating pore‐blocking residues. Moreover, we demonstrated that PC2 will preferentially assemble with PC1 to form heteromeric complexes when PC1 is co‐expressed ...
Tobias Staudner+7 more
wiley +1 more source
Therapeutics Data Commons: Machine Learning Datasets and Tasks for Drug Discovery and Development [PDF]
Therapeutics machine learning is an emerging field with incredible opportunities for innovatiaon and impact. However, advancement in this field requires formulation of meaningful learning tasks and careful curation of datasets. Here, we introduce Therapeutics Data Commons (TDC), the first unifying platform to systematically access and evaluate machine ...
arxiv
Aptamer-based therapeutics and their potential in radiopharmaceutical design [PDF]
Aptamers, short, single stranded oligonucleotide entities, have been developed in the past 15 years against a plethora of targets and for a variety of applications.
Ferreira, Catia S. M.+1 more
core +3 more sources
Decoding the dual role of autophagy in cancer through transcriptional and epigenetic regulation
Transcriptional and epigenetic regulation controls autophagy, which exerts context‐dependent effects on cancer: Autophagy suppresses tumorigenesis by maintaining cellular homeostasis or promotes tumor progression by supporting survival under stress. In this “In a Nutshell” article, we explore the intricate mechanisms of the dual function of autophagy ...
Young Suk Yu, Ik Soo Kim, Sung Hee Baek
wiley +1 more source