Stem cell therapeutics and gene therapy for neurologic disorders.

Autor:	Chen KS; Department of Neurology, University of Michigan, Ann Arbor, MI 48109, USA; NeuroNetwork for Emerging Therapies, University of Michigan, Ann Arbor, MI 48109, USA; Department of Neurosurgery, University of Michigan, Ann Arbor, MI 48109, USA., Koubek EJ; Department of Neurology, University of Michigan, Ann Arbor, MI 48109, USA; NeuroNetwork for Emerging Therapies, University of Michigan, Ann Arbor, MI 48109, USA., Sakowski SA; Department of Neurology, University of Michigan, Ann Arbor, MI 48109, USA; NeuroNetwork for Emerging Therapies, University of Michigan, Ann Arbor, MI 48109, USA., Feldman EL; Department of Neurology, University of Michigan, Ann Arbor, MI 48109, USA; NeuroNetwork for Emerging Therapies, University of Michigan, Ann Arbor, MI 48109, USA. Electronic address: efeldman@med.umich.edu.
Jazyk:	angličtina
Zdroj:	Neurotherapeutics : the journal of the American Society for Experimental NeuroTherapeutics [Neurotherapeutics] 2024 Jul; Vol. 21 (4), pp. e00427. Date of Electronic Publication: 2024 Aug 02.
DOI:	10.1016/j.neurot.2024.e00427
Abstrakt:	Rapid advances in biological knowledge and technological innovation have greatly advanced the fields of stem cell and gene therapies to combat a broad spectrum of neurologic disorders. Researchers are currently exploring a variety of stem cell types (e.g., embryonic, progenitor, induced pluripotent) and various transplantation strategies, each with its own advantages and drawbacks. Similarly, various gene modification techniques (zinc finger, TALENs, CRISPR-Cas9) are employed with various delivery vectors to modify underlying genetic contributors to neurologic disorders. While these two individual fields continue to blaze new trails, it is the combination of these technologies which enables genetically engineered stem cells and vastly increases investigational and therapeutic opportunities. The capability to culture and expand stem cells outside the body, along with their potential to correct genetic abnormalities in patient-derived cells or enhance cells with extra gene products, unleashes the full biological potential for innovative, multifaceted approaches to treat complex neurological disorders. In this review, we provide an overview of stem cell and gene therapies in the context of neurologic disorders, highlighting recent advances and current shortcomings, and discuss prospects for future therapies in clinical settings. Competing Interests: Declaration of competing interest The authors declare the following financial interests/personal relationships which may be considered as potential competing interests: Eva L. Feldman reports financial support was provided by National Institutes of Health. Kevin S. Chen reports financial support was provided by Alzheimer's Association. Kevin S. Chen reports financial support was provided by Frances and Kenneth Eisenberg Emerging Scholar Fund. Eva L. Feldman reports financial support was provided by Robert and Katherine Jacobs Environmental Health Initiative. Eva L. Feldman reports financial support was provided by Andrea and Lawrence A. Wolfe Brain Health Initiative. Eva L. Feldman reports financial support was provided by Richard and Jane Manoogian Foundation. Eva L. Feldman reports financial support was provided by Frank L. and Helen Gofrank Foundation Research Program in Alzheimer's Disease and Brain Health. Eva L. Feldman reports financial support was provided by Kiriluk Family Fund for Brain Health Research. Eva L. Feldman reports financial support was provided by Taubman Foundation. Eva L. Feldman reports financial support was provided by NeuroNetwork for Emerging Therapies. If there are other authors, they declare that they have no known competing financial interests or personal relationships that could have appeared to influence the work reported in this paper. (Copyright © 2024 The Authors. Published by Elsevier Inc. All rights reserved.)
Databáze:	MEDLINE
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