- Ho, Wan;
- Chang, Jer-Cherng;
- Lim, Kenneth;
- Cazenave-Gassiot, Amaury;
- Nguyen, Aivi;
- Foo, Juat;
- Muralidharan, Sneha;
- Viera-Ortiz, Ashley;
- Ong, Sarah;
- Hor, Jin;
- Agrawal, Ira;
- Hoon, Shawn;
- Arogundade, Olubankole;
- Rodriguez, Maria;
- Lim, Su;
- Kim, Seung;
- Ravits, John;
- Ng, Shi-Yan;
- Wenk, Markus;
- Lee, Edward;
- Tucker-Kellogg, Greg;
- Ling, Shuo-Chien
Cholesterol metabolism operates autonomously within the central nervous system (CNS), where the majority of cholesterol resides in myelin. We demonstrate that TDP-43, the pathological signature protein for amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD), influences cholesterol metabolism in oligodendrocytes. TDP-43 binds directly to mRNA of SREBF2, the master transcription regulator for cholesterol metabolism, and multiple mRNAs encoding proteins responsible for cholesterol biosynthesis and uptake, including HMGCR, HMGCS1, and LDLR. TDP-43 depletion leads to reduced SREBF2 and LDLR expression, and cholesterol levels in vitro and in vivo. TDP-43-mediated changes in cholesterol levels can be restored by reintroducing SREBF2 or LDLR. Additionally, cholesterol supplementation rescues demyelination caused by TDP-43 deletion. Furthermore, oligodendrocytes harboring TDP-43 pathology from FTD patients show reduced HMGCR and HMGCS1, and coaggregation of LDLR and TDP-43. Collectively, our results indicate that TDP-43 plays a role in cholesterol homeostasis in oligodendrocytes, and cholesterol dysmetabolism may be implicated in TDP-43 proteinopathies-related diseases.