Results 131 to 140 of about 51,591 (290)
, 2020 Purpose: To evaluate the retinal features of elderly patients affected by pseudoxanthoma elasticum (PXE). Materials and methods: This is a retrospective case series of 62 eyes of 31 elderly PXE patients (age > 50 years). Clinical data, ultra-widefield
Quaglino D. +7 more
core +1 more source
Status and future of recombinant adeno‐associated virus vector manufacturing
Biotechnology Progress, EarlyView.Abstract Sixty years of adeno‐associated virus (AAV) research illustrates a trajectory marked by basic science exploration, iterative innovation, persistent challenges, a number of clinical setbacks, as well as commercial therapeutic triumphs. This continual evolution has led to recombinant AAV (rAAV) becoming a cornerstone of modern gene therapy ...
Frank Agbogbo, David Dismuke
wiley +1 more source
Optimization of CEST MRI Reporter Protein Design Using Cation‐Pi Networks
Chemistry – A European Journal, EarlyView.A novel engineering approach can produce reporter proteins for cell and viral therapy tracking with unique magnetic resonance imaging (MRI) signatures, detectable with chemical exchange saturation transfer (CEST). We discover how cation‐π interactions between amino acid groups can help us fine‐tune magnetic resonance properties for noninvasive ...
David E. Korenchan +8 more
wiley +1 more source
Clinical Pharmacology &Therapeutics, EarlyView.Ruxolitinib pharmacokinetics (PK) has been characterized in clinical trials but remains poorly documented in real‐world practice. This project aimed to investigate ruxolitinib PK in routine clinical practice, identify factors driving its variability, and explore exposure–response relationships to assess the potential role of therapeutic drug monitoring.
Jérémie Tachet +11 more
wiley +1 more source
Prevalence of generalized retinal dystrophy in Denmark
, 2014 PURPOSE: Generalized retinal dystrophy is a frequent cause of visual impairment and blindness in younger individuals and a subject of new clinical intervention trials.
Bertelsen, Mette +3 more
core +1 more source
Fhod3 in zebrafish supports myofibril stability during growth of embryonic skeletal muscle
Developmental Dynamics, EarlyView.Abstract Background Actin filament organization in cardiomyocytes critically depends on the formin Fhod3, but a role for Fhod3 in skeletal muscle development has not yet been described. Results We demonstrate here that in zebrafish mutated for one of two fhod3 paralog genes, fhod3a, skeletal muscle of the trunk appears normal through 2 days post ...
Aubrie Russell +3 more
wiley +1 more source
, 2014 PowerPoint discussing Cone Dystrophy: Early loss of central and color vision; Color impairment often out of proportion to loss of VA; Hemeralopia ("day blindness") prominent; Light sensitivity and photophobia; Macular changes variable, and may occur ...
Gregory P. Van Stavern, MD
core
, 2020 : The purpose is report a case of reticular pattern dystrophy of the retinal pigment epithelium (RPE) in a 56-year-old woman with breast cancer. Images were obtained using fundus photography, fundus autofluorescence (FAF) imaging, and optical coherence ...
Shinji Makino, Yuka Shimizu
core
Fleck Dystrophy of the Cornea; a Report of Cases from Three Generations of a Family
, 1994 Fleck dystrophy of the cornea is characterized by numerous, tiny, small opacities scattered throughout the entire corneal stroma. The mode of inheritance is autosomal dominant, and this dystrophy is considered to be bilaterally symmetric.
Y.A. Akova, S. Duman, N. Ünlü
core +1 more source
Epileptic Disorders, EarlyView.Abstract Background and Objectives Muscle‐Eye‐Brain disease (MEB) is a dystroglycanopathy that belongs to the congenital muscular dystrophies. Central nervous system manifestations include congenital brain abnormalities, neurodevelopmental delay, and epilepsy, making it a rare but important cause of developmental and epileptic encephalopathy.
Stefania Kalampokini +6 more
wiley +1 more source