Neurodegeneration research at the Waisman Center from gene to organelle to cell to brain

Alzheimer’s disease, Parkinson’s disease, amyotrophic lateral sclerosis (ALS), Charcot-Marie-Tooth disease, and retinitis pigmentosa all have different manifestations and affect different body functions, but they are all connected by one mechanism: neurodegeneration.

Understanding autism from the minute to the masses: Autism research at the Waisman Center

Autism spectrum disorder (ASD) is an intricate and complicated diagnosis. The spectrum of presentations and severity is as expansive as the theorized causes. Autism’s complexity and breadth of impacts on a person’s life means that it has a multitude of facets to investigate.

The identities of enzymes: study further defines the function of a potential target for Alzheimer’s therapy

A new study from the lab of UW-Madison professor of medicine Luigi Puglielli, MD, PhD, opens a door to potential treatments for diseases of age, such as Alzheimer’s disease, by defining the roles of two enzymes that are imperative to protein production.

Luigi Puglielli, MD, PhD – Slide of the Week

Nε-lysine acetylation in the ER is an essential component of the quality control machinery. ER acetylation is ensured by a membrane transporter, AT-1/SLC33A1, which translocates cytosolic acetyl-CoA into the ER lumen, and two acetyltransferases, ATase1 and ATase2, which acetylate nascent polypeptides within the ER lumen. Dysfunctional AT-1, as caused by gene mutation or duplication events, results in severe disease phenotypes. 

Luigi Puglielli, MD, PhD – Slide of the Week

Nε-lysine acetylation of nascent glycoproteins within the endoplasmic reticulum (ER) lumen regulates the efficiency of the secretory pathway. The ER acetylation machinery consists of the membrane transporter, acetyl-CoA transporter 1 (AT-1/SLC33A1), and two acetyltransferases, ATase1/NAT8B and ATase2/NAT8. Dysfunctional ER acetylation is associated with severe neurological diseases with duplication of AT-1/SLC33A1 being associated with autism spectrum disorder, intellectual disability, and dysmorphism.