Establishment of a Personalized Antisense Oligonucleotide (ASO) Screening Platform Based on Patient-Derived Organoids Academic Background In recent years, with the rapid development of genome sequencing technologies, an increasing number of rare genetic diseases have been found to be associated with specific gene mutations. Antisense oligonucleotid...

Antisense Oligonucleotide Enhances SLC20A2 Expression and Inhibits Brain Calcification in Humanized Mouse Models Background and Research Questions Primary Familial Brain Calcification (PFBC) is an age-related neurogenetic disorder, characterized by bilateral calcifications in brain regions such as the basal ganglia, thalamus, and cerebellum. PFBC p...

Introduction In recent years, oligonucleotide-based therapeutic technologies, such as antisense oligonucleotides (ASOs) and small interfering RNAs (siRNAs), have been widely applied in the treatment of various neurological disorders. The basis for their application is that these technologies can selectively regulate target RNA molecules, which are ...