AB-1001 explained

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.^{[1] [2]}

Notes and References

1. Book: Parkin . Georgia M. . Corey-Bloom . Jody . Biomarkers for Huntington's Disease: Improving Clinical Outcomes . 2023 . Springer International Publishing . 978-3-031-32815-2 . 405–431 . https://link.springer.com/chapter/10.1007/978-3-031-32815-2_17 . en . Considerations and Advances in Huntington’s Disease Clinical Trial Design. 10.1007/978-3-031-32815-2_17 .
2. Schultz . Meredith . Durr . Alexandra . Karachi . Carine . O’Callaghan . Michael . Alves . Sandro . Lamontagne . Alain . Hurmic . Hortense . Cartier . Nathalie . Investigating the Effects of Brain Cholesterol Metabolism Using CYP46A1 Gene Therapy in Subjects with Huntington's Disease (P1-11.016) . Neurology . 25 April 2023 . 100 . 17 Supplement 2 . 10.1212/WNL.0000000000203493 . 258404880 . en . 0028-3878.