A5026392648- Folate and B Vitamins Research
- Mitochondrial Function and Pathology
- Metabolism and Genetic Disorders
- Advanced Chemical Physics Studies
- Virus-based gene therapy research
- CAR-T cell therapy research
- Atmospheric Ozone and Climate
- Atomic and Molecular Physics
- Monoclonal and Polyclonal Antibodies Research
Cell and Gene Therapy Catapult
2025
Hefei National Center for Physical Sciences at Nanoscale
2022
University of Science and Technology of China
2022
<title>Abstract</title> Combined methylmalonic acidemia and homocystinemia (cblC) is an autosomal recessive disorder characterized by aberrant organic acid metabolism. The c.80A > G mutation in the <italic>MMACHC</italic> gene has been documented numerous studies linked to cblC phenotypes. However, this mutation's pathogenic mechanisms remain elusive, as it not yet validated through functional studies. In a previous study, we developed murine model with Mmachc elucidate intricacies of...
<title>Abstract</title> Combined methylmalonic acidemia and homocystinemia (cblC) is an autosomal recessive disorder characterized by aberrant organic acid metabolism. The c.80A > G mutation in the <italic>MMACHC</italic> gene has been documented numerous studies linked to cblC phenotypes. However, this mutation's pathogenic mechanisms remain elusive, as it not yet validated through functional studies. In a previous study, we developed murine model with Mmachc elucidate intricacies of...
Anti-AAV neutralizing Abs (NAbs) titer is usually measured by cell-based microneutralization (MN) assay and crucial for patient screening in AAV-based gene therapy clinical trials. However, achieving uniform operation comparable results among different laboratories remains challenging. Here, we established a standardized MN anti-AAV9 NAbs human sera or plasma transferred the method to other two research teams. Then, validated its parameters tested set of eight samples blind across all...