Publications by authors named "Marlene Scheffold"
Contractile rings are formed from cytoskeletal filaments during cell division. Ring formation is induced by specific crosslinkers, while contraction is typically associated with motor protein activity. Here, we engineer DNA nanotubes and peptide-functionalized starPEG constructs as synthetic crosslinkers to mimic this process.
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- The study presents a high-throughput screening method to find therapies for a rare childhood disease (AP-4 deficiency) that causes mislocalization of a protein called ATG9A.
- Researchers screened over 28,000 small molecules and discovered a promising compound, BCH-HSP-C01, that can correct the protein's abnormal movement in various disease models, including patient cells.
- The findings also include insights into how BCH-HSP-C01 works on a molecular level, laying the groundwork for future research related to treatment options for AP-4 deficiency.
Article Synopsis
- This study focused on developing a high-throughput screening method to identify new treatment targets for AP-4 deficiency, a rare childhood disease that causes problems with protein transport in cells.
- Researchers screened a large library of 28,864 small molecules and found a promising lead compound that corrected the mislocalization of the autophagy protein ATG9A in various disease models, including patient-derived cells.
- The study used advanced techniques to investigate how the lead compound affects molecular targets and cellular mechanisms, ultimately providing crucial insights for future drug development aimed at treating AP-4 deficiency.