Background: Alzheimer's disease (AD) is the main cause of dementia and it is a progressive
neurogenerative disease characterized by the accumulation of neurofibrillary tangles and senile
plaques. There is currently no cure; however, some treatments are available to slow down the progression
of the disease, including gene therapy, which has been investigated to have great potential for the
treatment of AD.
Objective: The aim of this review was to identify the efficacy of gene therapy to restore cognition in
Methods: A systematic review was carried out using papers published up to May 2020 and available
in the Web of Science, Scopus, and Medline/PUBMED databases. Articles were considered for inclusion
if they were original researches that investigated the effects of gene therapy on cognition in AD.
The methodological quality of the selected studies was evaluated using the Risk of Bias Tool for Animal
Intervention Studies (SYRCLE’s Rob tool) and the Jadad Scale.
Results: Most preclinical studies obtained positive results in improving memory and learning in mice
that underwent treatment with gene therapy. On the other hand, clinical studies have obtained inconclusive
results related to the delivery methods of the viral vector used in gene therapy.
Conclusion: Gene therapy has shown a great potential for the treatment of AD in preclinical trials, but
results should be interpreted with caution since preclinical studies presented limitations to predict the
efficacy of the treatment outcome in humans.