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Papillon Therapeutics Receives Rare Pediatric Disease Designation from the U.S. Food and Drug Administration for PPL-002 for the Treatment of Danon Disease
About Danon Disease Danon disease is a rare, X-linked genetic disorder caused by mutations in the LAMP2 gene, leading to the accumulation of autophagic vacuoles in cells, primarily in the heart ...
The EMBO Journal1mon
Autophagy adaptors mediate Parkin-dependent mitophagy by forming sheet-like liquid condensates
Peer ReviewDownload a summary of the editorial decision process including editorial decision letters, reviewer comments and author responses to feedback. During PINK1- and Parkin-mediated mitophagy, ...
The EMBO Journal1mon
Unraveling autophagic imbalances and therapeutic insights in Mecp2-deficient models
This correlates with defective lipidation of LC3B, probably caused by a deficiency of the autophagic membrane lipid phosphatidylethanolamine. The administration of the autophagy inducer trehalose ...
eLife6y
Endosomal Rab cycles regulate Parkin-mediated mitophagy
Damaged mitochondria are selectively eliminated by mitophagy. Parkin and PINK1, gene products mutated in familial Parkinson’s disease, play essential roles in mitophagy through ubiquitination of ...