Targeting Alzheimer's disease with gene and cell therapies

R. Loera-Valencia, A. Piras, M. A.M. Ismail, S. Manchanda, Helga Eyjólfsdóttir, T. C. Saido, J. Johansson, M. Eriksdotter, B. Winblad, P. Nilsson*

*Corresponding author for this work

Research output: Contribution to journalReview articlepeer-review

25 Citations (Scopus)


Alzheimer's disease (AD) causes dementia in both young and old people affecting more than 40 million people worldwide. The two neuropathological hallmarks of the disease, amyloid beta (Aβ) plaques and neurofibrillary tangles consisting of protein tau are considered the major contributors to the disease. However, a more complete picture reveals significant neurodegeneration and decreased cell survival, neuroinflammation, changes in protein and energy homeostasis and alterations in lipid and cholesterol metabolism. In addition, gene and cell therapies for severe neurodegenerative disorders have recently improved technically in terms of safety and efficiency and have translated to the clinic showing encouraging results. Here, we review broadly current data within the field for potential targets that could modify AD through gene and cell therapy strategies. We envision that not only Aβ will be targeted in a disease-modifying treatment strategy but rather that a combination of treatments, possibly at different intervention times may prove beneficial in curing this devastating disease. These include decreased tau pathology, neuronal growth factors to support neurons and modulation of neuroinflammation for an appropriate immune response. Furthermore, cell based therapies may represent potential strategies in the future.

Original languageEnglish
Pages (from-to)2-36
Number of pages35
JournalJournal of Internal Medicine
Issue number1
Publication statusPublished - Jul 2018

Bibliographical note

Funding Information:
We would like to thank Ping Sui at Ping's Scientific Illustration for figure design (email: This work is financially supported by Swedish Research Council (2015-02774 to BW, 2016-02776 to PN), Hållstens Forskningsstiftelse to PN, Alzheimerfonden and Hjärnfonden to PN and WB. RLV was financially supported by Mexico's National Council for Science and Technology (CONACYT) CVU: 209252, Margaretha af Ugglas foundation and by Olle Enqvist Foundation grant no. 2014/778.

Publisher Copyright:
© 2018 The Association for the Publication of the Journal of Internal Medicine

Other keywords

  • Alzheimer's disease
  • cell therapy
  • gene therapy
  • induced neurogenesis
  • neprilysin
  • tau


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