LYSOCIL CEDOC-NMS I FCM special seminar with Rosa Puertollano M.D., Ph.D. Senior Investigator

December 15, 2021


Special LYSOCIL CEDOC-NMS I FCM seminar with Rosa Puertollano M.D., Ph.D. Senior Investigator from Cell and Developmental Biology Center, National Heart, Lung, and Blood Institute, National Instituted of Health, Bethesda, MD, USA occurred  on 15th December 2021, at 3 pm, via Zoom.


Title: How lysosomes sense, integrate, and cope with stress


Abstract: Our understanding of lysosomes has dramatically changed in the last decade. From being considered as mere degradative organelles, lysosomes are now recognized as critical regulators or nutrient sensing, energy homeostasis and cellular response to stress. A critical discovery was the realization that the number and activity of lysosomes fluctuate in response to changes in environmental conditions. This lysosome to nucleus communication is mainly mediated by TFEB and TFE3, two members of the MiT/TFE family of transcription factors. TFEB and TFE3 play a critical role mediating cellular response to a variety of stress conditions, including nutrient deprivation, oxidative stress, and pathogen infection. While the pathways of TFEB/TFE3 nuclear translocation are well characterized, little is known about the mechanisms that may help modulating the strength, duration, and selectivity of their transcriptional responses. By using Rapid Immunoprecipitation Mass spectrometry of Endogenous proteins (RIME) we have recently identified several novel TFEB/TFE3 posttranslational modification and binding partners, providing important insight on the epigenetic regulation of lysosomal biogenesis. Given the key role played by lysosomes as regulators of cellular homeostasis, it is not surprising that defective lysosomal function leads to disease. Lysosomal dysfunction is the underlying cause of a class of metabolic disorders known as Lysosomal Storage Diseases (LSDs). My laboratory is focus on exploring different approaches to improve the treatment efficiency for Pompe Disease, a devastating LSD caused by deficient breakdown of glycogen inside lysosomes.



Rare Disease