Gene Therapy Approach with an Emphasis on Growth Factors: Theoretical and Clinical Outcomes in Neurodegenerative Diseases.

Autor: Parambi DGT; College of Pharmacy, Department of Pharmaceutical Chemistry, Jouf University, Al Jouf-2014, Sakaka, Saudi Arabia., Alharbi KS; College of Pharmacy, Department of Pharmaceutical Chemistry, Jouf University, Al Jouf-2014, Sakaka, Saudi Arabia., Kumar R; Kerala University of Health Sciences, Thrissur, Kerala, 680596, India., Harilal S; Kerala University of Health Sciences, Thrissur, Kerala, 680596, India., Batiha GE; Department of Pharmacology and Therapeutics, Faculty of Veterinary Medicine, Damanhour University, Damanhour, 22511, Al Beheira, Egypt., Cruz-Martins N; Faculty of Medicine, University of Porto, 4200-319, Porto, Portugal. ncmartins@med.up.pt.; Institute for Research and Innovation in Health (i3S), University of Porto, 4200-135, Porto, Portugal. ncmartins@med.up.pt.; Institute of Research and Advanced Training in Health Sciences and Technologies (CESPU), Rua Central de Gandra, 1317, 4585-116, Gandra PRD, Portugal. ncmartins@med.up.pt., Magdy O; Department of Clinical Pharmacology, College of Pharmacy, Jouf University, Sakaka, Al Jouf-2014, Kingdom of Saudi Arabia.; Pharmacognosy Department, College of Pharmacy, Jouf University, Sakaka, Aljouf, 72341, Kingdom of Saudi Arabia., Musa A; Pharmacognosy Department, College of Pharmacy, Jouf University, Sakaka, Aljouf, 72341, Kingdom of Saudi Arabia.; Pharmacognosy Department, Faculty of Pharmacy, Al-Azhar University, Cairo, 11371, Egypt., Panda DS; Department of Pharmaceutics, College of Pharmacy, Jouf University, Al Jouf, Sakaka, 72341, Kingdom of Saudi Arabia., Mathew B; Department of Pharmaceutical Chemistry, Amrita School of Pharmacy, Amrita Vishwa Vidyapeetham, AIMS Health Sciences Campus, Kochi, 682 041, India. bijomathew@aims.amrita.edu.
Jazyk: angličtina
Zdroj: Molecular neurobiology [Mol Neurobiol] 2022 Jan; Vol. 59 (1), pp. 191-233. Date of Electronic Publication: 2021 Oct 15.
DOI: 10.1007/s12035-021-02555-y
Abstrakt: The etiology of many neurological diseases affecting the central nervous system (CNS) is unknown and still needs more effective and specific therapeutic approaches. Gene therapy has a promising future in treating neurodegenerative disorders by correcting the genetic defects or by therapeutic protein delivery and is now an attraction for neurologists to treat brain disorders, like Alzheimer's disease, Parkinson's disease, amyotrophic lateral sclerosis, spinal muscular atrophy, spinocerebellar ataxia, epilepsy, Huntington's disease, stroke, and spinal cord injury. Gene therapy allows the transgene induction, with a unique expression in cells' substrate. This article mainly focuses on the delivering modes of genetic materials in the CNS, which includes viral and non-viral vectors and their application in gene therapy. Despite the many clinical trials conducted so far, data have shown disappointing outcomes. The efforts done to improve outcomes, efficacy, and safety in the identification of targets in various neurological disorders are also discussed here. Adapting gene therapy as a new therapeutic approach for treating neurological disorders seems to be promising, with early detection and delivery of therapy before the neuron is lost, helping a lot the development of new therapeutic options to translate to the clinic.
(© 2021. The Author(s), under exclusive licence to Springer Science+Business Media, LLC, part of Springer Nature.)
Databáze: MEDLINE
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