Seeking to understand the biology of sphingolipids with the goal of developing therapies for the diseases of sphingolipid metabolism.
Select Publications
- 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 Jun 17) Abstract/Full Text
- Sialidase NEU3 action on GM1 ganglioside is neuroprotective in GM1 gangliosidosis.
- Allende ML, Lee YT, Byrnes C, Li C, Tuymetova G, Bakir JY, Nicoli ER, James VK, Brodbelt JS, Tifft CJ, Proia RL.
- J Lipid Res (2023 Dec) 64:100463. Abstract/Full Text
Last Reviewed August 2024