DDHD2, whose mutations cause spastic paraplegia type 54, enhances lipophagy via engaging ATG8 family proteins.
Fei JiaXiaoman WangYuhua FuShi-Min ZhaoBoxun LuChenji WangPublished in: Cell death and differentiation (2024)
Hereditary spastic paraplegia (HSP) is a group of inherited neurodegenerative disorders characterized by progressive lower limb spasticity and weakness. One subtype of HSP, known as SPG54, is caused by biallelic mutations in the DDHD2 gene. The primary pathological feature observed in patients with SPG54 is the massive accumulation of lipid droplets (LDs) in the brain. However, the precise mechanisms and roles of DDHD2 in regulating lipid homeostasis are not yet fully understood. Through Affinity Purification-Mass Spectroscopy (AP-MS) analysis, we identify that DDHD2 interacts with multiple members of the ATG8 family proteins (LC3, GABARAPs), which play crucial roles in lipophagy. Mutational analysis reveals the presence of two authentic LIR motifs in DDHD2 protein that are essential for its binding to LC3/GABARAPs. We show that DDHD2 deficiency leads to LD accumulation, while enhanced DDHD2 expression reduces LD formation. The LC3/GABARAP-binding capacity of DDHD2 and the canonical autophagy pathway both contribute to its LD-eliminating activity. Moreover, DDHD2 enhances the colocalization between LC3B and LDs to promote lipophagy. LD·ATTEC, a small molecule that tethers LC3 to LDs to enhance their autophagic clearance, effectively counteracts DDHD2 deficiency-induced LD accumulation. These findings provide valuable insights into the regulatory roles of DDHD2 in LD catabolism and offer a potential therapeutic approach for treating SPG54 patients.
Keyphrases
- small molecule
- simultaneous determination
- mass spectrometry
- lower limb
- cell death
- multiple sclerosis
- transcription factor
- binding protein
- poor prognosis
- newly diagnosed
- machine learning
- heat shock protein
- end stage renal disease
- gene expression
- long non coding rna
- ejection fraction
- protein protein
- deep learning
- prognostic factors
- replacement therapy
- high resolution
- single molecule
- patient reported outcomes
- endoplasmic reticulum stress
- heat stress
- blood brain barrier
- endothelial cells
- solid state
- diabetic rats
- patient reported