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Lysosomal adaptation to environmental changes is critical for cellular and metabolic homeostasis and requires coordination by the mTORC1 kinase, which conveys nutritional and stress signals into distinct, substrate-specific outputs. The FLCN:FNIP complex serves as a crucial regulator of lysosomal fu...
ORGANISM(S): Homo sapiens (Human) 
2026-09-24 | PXD069600 | Pride
Liver gene therapy with adeno-associated viral (AAV) vectors is under clinical investigation for hemophilia A (HemA), the most common inherited X-linked bleeding disorder. Major limitations are the large size of the F8 transgene, which makes packaging in a single AAV vector a challenge, as well as t...
ORGANISM(S): Homo sapiens (Human) 
2022-03-31 | PXD031884 | Pride
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