High-precision Base Editing Technology in Primates and Human Retina Research Background Stargardt disease is a currently untreatable, inherited neurodegenerative disease that leads to macular degeneration and blindness due to loss-of-function mutations in the ABCA4 gene. The protein encoded by the ABCA4 gene is a membrane lipid flippase localized i...
Breakthrough in Gene Therapy: Repair Drive Technology Enables In Vivo Expansion of Hepatocytes Academic Background Gene therapy has become a hot topic in medical research in recent years, especially for liver diseases. Due to the central role of the liver in metabolism, it has become a critical target for research. Although existing gene-editing te...
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...
Report on the Compatibility of mRNA-Lipid Nanoparticle-Based Gene-Engineered Platelets with Blood Banking Practices Academic Background Platelets play an essential role in hemostasis, inflammation, sepsis, and cancer. However, clinical applications of platelet transfusions are primarily limited to managing thrombocytopenia and bleeding. To broaden ...
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...
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...
New Gene Therapy Approach May Control Epileptic Seizures Caused by Focal Cortical Dysplasia Focal Cortical Dysplasia (FCD) is a type of disorder caused by abnormal cortical development, often accompanied by drug-resistant epilepsy and cognitive and behavioral disorders. According to the classification criteria proposed by Najm et al. in 2022 for th...