Background and Research Motivation Amyotrophic Lateral Sclerosis (ALS) is a devastating neurodegenerative disease affecting upper and lower motor neurons, leading to progressive muscle paralysis and eventual death. Currently, no effective treatments can significantly delay or halt ALS progression. While various ALS mouse models (such as those carry...

Research Report on the Improvement of CMT-like Neuropathy via AAV9 Vector-based C1ORF194 Gene Therapy Background and Research Motivation Charcot-Marie-Tooth disease (CMT) is a rare group of genetically heterogeneous neuromuscular disorders characterized by progressive muscle weakness and atrophy, along with sensory loss. Despite extensive preclinic...

GYS1 Antisense Therapy Inhibits Pathogenic Aggregates and Epileptiform Discharges in a Mouse Model of Lafora Disease Background and Objectives Lafora disease (LD) is a devastating autosomal recessive genetic disorder characterized by epilepsy and rapidly progressive dementia in adolescence. The disease primarily involves mutations in the EPM2A or E...

D1R-5-HT2AR Uncoupling Alleviates Depressive Behaviors Through HDAC Signaling Research Background Major depressive disorder (MDD) is a globally prevalent and life-threatening mental illness, closely associated with poor physical health. Despite the availability of various treatments, including medications and psychotherapy, many patients exhibit po...

Gut Microbiota-Derived Indole-3-Acetic Acid (3-IAA) Suppresses High Myopia Progression by Promoting Type I Collagen Synthesis Background High myopia (HM) is a serious vision-threatening ocular condition associated with complications such as glaucoma, cataracts, and macular degeneration. Currently, effective interventions for high myopia are lacking...

Developing a Novel Erythrocyte-MHC-I Conjugate for Cancer Treatment: A Revolutionary Immunotherapy Research Background: Traditional Roles and Newly Discovered Potential Erythrocytes, or red blood cells, are the most abundant cells in the human body, comprising approximately 70% of total cell count in adults. Traditionally recognized for their role ...