Genetic medicines, including CRISPR/Cas technologies, extend tremendous promise for addressing unmet medical need in inherited retinal disorders and other indications; however, there remain challenges for the development of therapeutics. Herein, we evaluate genome editing by engineered Cas9 ribonucleoproteins (eRNP) in vivo via subretinal administration using mouse and pig animal models. Subretinal administration of adenine base editor and double strand break-inducing Cas9 nuclease eRNPs mediate genome editing in both species.
View Article and Find Full Text PDFCardiac sarcoidosis (CS) is a rare entity characterized by granulomatous infiltration of the myocardium, which can lead to myocardial fibrosis, conduction abnormalities, and the development of heart failure, thereby elevating the risk of sudden cardiac death (SCD). While endomyocardial biopsy (EMBx) is regarded as the gold standard for diagnosis, its low sensitivity and inherent procedural risks may limit its practical application. This study retrospectively explored the role of advanced imaging modalities, specifically cardiovascular magnetic resonance imaging (CMR) and fluorodeoxyglucose positron emission tomography (FDG-PET), in the diagnosis and management of CS within a single center.
View Article and Find Full Text PDF