Purpose Defects in primary cilia and ciliary signaling are associated with an array of neurodegenerative diseases. Primary cilia are sensory organelles that transmit and regulate various cellular communication pathways, including the significant Sonic hedgehog and Wnt signal pathways. Extensive studies have shown that...
Ke Ning, Matthew Tran, Xin Xia et al.· Investigative Ophthalmology...· 0 citations
SUMMARY Programmable gene activation has broad therapeutic potential but remains constrained by large effector size, limited multiplexing capacity, and challenges in in vivo delivery. Here, we develop TIGRa, a compact transcriptional activator derived from the TIGR-TasR system that is mechanistically distinct from CRIS...
Zhi-Quan Liu, Si-Yu Chen, Wen-Min Wang et al.· Cell Stem Cell· 0 citations
The results showed that AAV2-OCRL demonstrated the highest transduction efficiency in patient iPSC-derived trabecular meshwork models (iHTM) among the three AAV-OCRL vectors evaluated, establishing it as a promising delivery vector.
Si-Yu Chen, Zhi-Quan Liu, Wen-Min Wang et al.· Advancement of science· 0 citations
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