Genetics of Development & Disease Section

of the Genetics & Biochemistry Branch

Seeking to understand the biology of sphingolipids with the goal of developing therapies for the diseases of sphingolipid metabolism.

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CNS-targeted base editing of the major late-onset Tay-Sachs mutation alleviates disease in mice.
Allende ML, Kono M, Lee YT, Olmsted SM, Huso V, Bakir JY, Pratto F, Li C, Byrnes C, Tuymetova G, Zhu H, Tifft CJ, Proia RL.
J Clin Invest (2025 Aug 15) 135. Abstract/Full Text
1-Deoxysphingolipids require very-long-chain ceramide synthesis to induce ER stress and cytotoxicity.
Byrnes C, Clarke BA, Zhu H, Lee YT, Majumder S, Kono M, Proia RL.
J Biol Chem (2026 Aug) 302:113281. Abstract/Full Text
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Lab Members

Proia/Tifft Lab Members 2024
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Last Reviewed August 2024