AB-1001

{{Short description|Experimental gene therapy}}

AB-1001 is an experimental gene therapy developed for Huntington's disease. It is intended to increase the production of the CYP46A1 enzyme and cholesterol levels in the brain.{{cite book |last1=Parkin |first1=Georgia M. |last2=Corey-Bloom |first2=Jody |title=Biomarkers for Huntington's Disease: Improving Clinical Outcomes |date=2023 |publisher=Springer International Publishing |isbn=978-3-031-32815-2 |pages=405–431 |chapter-url=https://link.springer.com/chapter/10.1007/978-3-031-32815-2_17 |language=en |chapter=Considerations and Advances in Huntington’s Disease Clinical Trial Design|doi=10.1007/978-3-031-32815-2_17 }}{{cite journal |last1=Schultz |first1=Meredith |last2=Durr |first2=Alexandra |last3=Karachi |first3=Carine |last4=O’Callaghan |first4=Michael |last5=Alves |first5=Sandro |last6=Lamontagne |first6=Alain |last7=Hurmic |first7=Hortense |last8=Cartier |first8=Nathalie |title=Investigating the Effects of Brain Cholesterol Metabolism Using CYP46A1 Gene Therapy in Subjects with Huntington's Disease (P1-11.016) |journal=Neurology |date=25 April 2023 |volume=100 |issue=17 Supplement 2 |doi=10.1212/WNL.0000000000203493 |s2cid=258404880 |url=https://n.neurology.org/content/100/17_Supplement_2/3739.abstract |language=en |issn=0028-3878|url-access=subscription }}