Current advances in gene therapy of mitochondrial diseases.

Autor: Soldatov VO; Core Facility Centre, Institute of Gene Biology, Russian Academy of Sciences, Moscow, Russia. pharmsoldatov@gmail.com.; Department of Pharmacology and Clinical Pharmacology, Belgorod State National Research University, Belgorod, Russia. pharmsoldatov@gmail.com.; Laboratory of Genome Editing for Biomedicine and Animal Health, Belgorod State National Research University, Belgorod, Russia. pharmsoldatov@gmail.com., Kubekina MV; Core Facility Centre, Institute of Gene Biology, Russian Academy of Sciences, Moscow, Russia., Skorkina MY; Department of Biochemistry, Belgorod State National Research University, Belgorod, Russia.; Laboratory of Genome Editing for Biomedicine and Animal Health, Belgorod State National Research University, Belgorod, Russia., Belykh AE; Dioscuri Centre for Metabolic Diseases, Nencki Institute of Experimental Biology, Polish Academy of Sciences, Warsaw, Poland., Egorova TV; Laboratory of Modeling and Gene Therapy of Hereditary Diseases, Institute of Gene Biology, Russian Academy of Sciences, Moscow, Russia., Korokin MV; Department of Pharmacology and Clinical Pharmacology, Belgorod State National Research University, Belgorod, Russia., Pokrovskiy MV; Department of Pharmacology and Clinical Pharmacology, Belgorod State National Research University, Belgorod, Russia., Deykin AV; Department of Pharmacology and Clinical Pharmacology, Belgorod State National Research University, Belgorod, Russia.; Laboratory of Genome Editing for Biomedicine and Animal Health, Belgorod State National Research University, Belgorod, Russia., Angelova PR; Department of Clinical and Movement Neurosciences, UCL Queen Square Institute of Neurology, London, UK.
Jazyk: angličtina
Zdroj: Journal of translational medicine [J Transl Med] 2022 Dec 05; Vol. 20 (1), pp. 562. Date of Electronic Publication: 2022 Dec 05.
DOI: 10.1186/s12967-022-03685-0
Abstrakt: Mitochondrial diseases (MD) are a heterogeneous group of multisystem disorders involving metabolic errors. MD are characterized by extremely heterogeneous symptoms, ranging from organ-specific to multisystem dysfunction with different clinical courses. Most primary MD are autosomal recessive but maternal inheritance (from mtDNA), autosomal dominant, and X-linked inheritance is also known. Mitochondria are unique energy-generating cellular organelles designed to survive and contain their own unique genetic coding material, a circular mtDNA fragment of approximately 16,000 base pairs. The mitochondrial genetic system incorporates closely interacting bi-genomic factors encoded by the nuclear and mitochondrial genomes. Understanding the dynamics of mitochondrial genetics supporting mitochondrial biogenesis is especially important for the development of strategies for the treatment of rare and difficult-to-diagnose diseases. Gene therapy is one of the methods for correcting mitochondrial disorders.
(© 2022. The Author(s).)
Databáze: MEDLINE
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