Quantifying mutant huntingtin protein in human cerebrospinal fluid to support the development of huntingtin-lowering therapies
Abstract Huntington’s disease (HD) is caused by a cytosine adenine guanine-repeat expansion in the huntingtin gene. This results in the production of toxic mutant huntingtin protein (mHTT), which has an elongated polyglutamine (polyQ) stretch near the protein’s N-terminal end. The pharmacological lo...
| Main Authors: | , , , , , , , , |
|---|---|
| Format: | Article |
| Language: | English |
| Published: |
Nature Portfolio
2023-04-01
|
| Series: | Scientific Reports |
| Online Access: | https://doi.org/10.1038/s41598-023-32630-4 |