Huntingtin HTT1a is generated in a CAG repeat-length-dependent manner in human tissues.

Franziska HoschekJulia NatanMaximilian WagnerKirupa SathasivamAlshaimaa AbdelmoezBjörn von EinemGillian P BatesG Bernhard LandwehrmeyerAndreas Neueder

Published in: Molecular medicine (Cambridge, Mass.) (2024)

Our results show that HTT1a expression occurs throughout a wide range of tissues and likely with all CAG lengths. Our data from peripheral sample sources demonstrate that HTT1a is indeed generated throughout the body in a CAG repeat-length-dependent manner. Therefore, the levels of HTT1a might be a sensitive marker of disease state and/or progression and should be monitored over time, especially in clinical trials targeting HTT expression.

Keyphrases

poor prognosis
clinical trial
gene expression
endothelial cells
binding protein
randomized controlled trial
electronic health record
drinking water
induced pluripotent stem cells
machine learning
deep learning
open label
pluripotent stem cells