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CTAD taskforce: genetic therapies in Alzheimer’s disease

Jakabek, D; Isaacs, AM; De Strooper, B; Tuszynski, M; Lane, R; Uspenskaya, O; McDade, E; ... Mummery, CJ; + view all (2025) CTAD taskforce: genetic therapies in Alzheimer’s disease. The Journal of Prevention of Alzheimer's Disease , Article 100269. 10.1016/j.tjpad.2025.100269. (In press). Green open access

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Abstract

There are an increasing number of genetic approaches to treating Alzheimer’s disease and other dementias, with some promising results from early-phase trials. This prompted the convention of the first EU-US CTAD Task Force on genetic therapies in Alzheimer’s disease in October 2024. Preclinical studies and clinical trials of genetic therapies in Alzheimer’s disease and other dementias are presented here with key lessons for the field. Importantly, there are several challenges and opportunities unique to neurogenetic therapies which were reviewed and discussed, including means of genetic manipulation, adverse events, monitoring, timing of therapy, and the importance of patient involvement in trial design. Continued collaboration across disciplines will accelerate development of neurogenetic therapeutics.

Type: Article
Title: CTAD taskforce: genetic therapies in Alzheimer’s disease
Open access status: An open access version is available from UCL Discovery
DOI: 10.1016/j.tjpad.2025.100269
Publisher version: https://doi.org/10.1016/j.tjpad.2025.100269
Language: English
Additional information: Copyright © 2025 The Author(s). Published by Elsevier Masson SAS on behalf of SERDI Publisher. This is an open access article under the CC BY license (http://creativecommons.org/licenses/by/4.0/).
Keywords: RCTs; Genetic therapy; siRNA; ASO; AAV
UCL classification: UCL
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Brain Sciences
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Brain Sciences > UCL Queen Square Institute of Neurology
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Brain Sciences > UCL Queen Square Institute of Neurology > Neurodegenerative Diseases
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Brain Sciences > UCL Queen Square Institute of Neurology > UK Dementia Research Institute
URI: https://discovery.ucl.ac.uk/id/eprint/10212623
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