Niuzheng Chai

Deciphering novel TCF4-driven molecular origins and mechanisms underlying a common triplet repeat expansion-mediated disease

S. Tuft, Nikolas Pontikos, M. Cheetham, 2023, bioRxiv.

Effective AAV-mediated gene replacement therapy in retinal organoids modeling AIPL1-associated LCA4

Almudena Sacristán-Reviriego, J. van der Spuy, P. Perdigão, 2024, Molecular therapy. Nucleic acids.

Deciphering novel TCF4-driven mechanisms underlying a common triplet repeat expansion-mediated disease

S. Tuft, Nikolas Pontikos, M. Cheetham, 2024, bioRxiv.