Kwi Hye Kim on Muck Rack

Kwi Hye Kim

As seen in: Cell Reports Medicine
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Articles

Recreating pathophysiology of CLN2 disease and demonstrating reversion by TPP1 gene therapy in hiPSC-derived retinal organoids and retina-on-chip

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By Ting Chen, Adelina Botezatu, Ke Ma, Arianna Tolone, Ryan M. Reddinger, Christopher Tully, Mikayla Higgins, Alexander Kleger, Markus Breunig, Madalena Cipriano, Sylvia Bolz, Marius Ueffing, Nicholas Buss, Peter Loskill, Stefan Liebau, Serena Corti, Kwi Hye Kim, Virginia Cora, Natalia Pashkovskaia, Anamaria Bernal Vergara, Denise Sperlich, Kaushambee Dave, Paul Lopatta, Svenja Wingerter, Kevin Achberger
| Cell Reports Medicine Verified
Keywords neuronal ceroid lipofuscinosis type 2 CLN2 tripeptidyl peptidase 1 TPP1 retinal organoids retina-on-chip gene therapy subunit C of mitochondrial ATP synthase SCMAS Introduction Neuronal ceroid lipofuscinosis type 2 (CLN2), or late infantile neuronal ceroid lipofuscinosis, is one of the most common forms of neuronal ceroid lipofuscinosis (NCL) affecting children and young adults.
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