Misplaced Pages

AB-1001

Article snapshot taken from Wikipedia with creative commons attribution-sharealike license. Give it a read and then ask your questions in the chat. We can research this topic together.
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.

References

  1. Parkin, Georgia M.; Corey-Bloom, Jody (2023). "Considerations and Advances in Huntington's Disease Clinical Trial Design". Biomarkers for Huntington's Disease: Improving Clinical Outcomes. Springer International Publishing. pp. 405–431. doi:10.1007/978-3-031-32815-2_17. ISBN 978-3-031-32815-2.
  2. Schultz, Meredith; Durr, Alexandra; Karachi, Carine; O’Callaghan, Michael; Alves, Sandro; Lamontagne, Alain; Hurmic, Hortense; Cartier, Nathalie (25 April 2023). "Investigating the Effects of Brain Cholesterol Metabolism Using CYP46A1 Gene Therapy in Subjects with Huntington's Disease (P1-11.016)". Neurology. 100 (17 Supplement 2). doi:10.1212/WNL.0000000000203493. ISSN 0028-3878. S2CID 258404880.
Categories: