Advances and Challenges in Gene Therapy for Alzheimer’s Disease

Issue title: Therapeutic Trials in Alzheimer’s Disease: Where Are We Now?

Guest editors: Paula I. Moreira, Jesus Avila, Daniela Galimberti, Miguel A. Pappolla, Germán Plascencia-Villa, Aaron A. Sorensen, Xiongwei Zhu and George Perry

Article type: Review Article

Authors: Morroni, Fabiana^{a; *} | Caccamo, Antonella^{b; *}

Affiliations: [a] Department of Pharmacy and Biotechnology, Alma Mater Studiorum, University of Bologna, Bologna, Italy | [b] Department of Chemical, Biological, Pharmaceutical and Environmental Sciences, University of Messina, Messina, Italy

Correspondence: [*] Correspondence to: Dr. Antonella Caccamo, Department of Chemical, Biological, Pharmaceutical and Environmental Sciences, University of Messina, Viale F. Stagno d’Alcontres 31, 98168 Messina, Italy. E-mail: [email protected] and Prof. Fabiana Morroni, Department of Pharmacy and Biotechnology, Alma Mater Studiorum University of Bologna, Via Irnerio 48, 40126 Bologna, Italy. E-mail: [email protected].

Abstract: Alzheimer’s disease (AD) is a progressive neurodegenerative disorder characterized by cognitive decline, memory loss, and behavioral impairments. Despite extensive research efforts, effective treatment options for AD remain limited. Recently, gene therapy has emerged as a promising avenue for targeted intervention in the pathogenesis of AD. This review will provide an overview of clinical and preclinical studies where gene therapy techniques have been utilized in the context of AD, highlighting their potential as novel therapeutic strategies. While challenges remain, ongoing research and technological advancement continue to enhance the potential of gene therapy as a targeted and personalized therapeutic approach for AD.

Keywords: Adeno associated virus, Alzheimer’s disease, amyloid-β, neurotrophins, tau

DOI: 10.3233/JAD-230783

Journal: Journal of Alzheimer's Disease, vol. 101, no. s1, pp. S417-S431, 2024