TY  - JOUR
N2  - 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.
VL  - 20
ID  - discovery10162546
N1  - This article is licensed under a Creative Commons Attribution 4.0 International License, which permits use, sharing, adaptation, distribution and reproduction in any medium or format, as long as you give appropriate credit to the original author(s) and the source, provide a link to the Creative Commons licence, and indicate if changes were made. The images or other third party material in this article are included in the article's Creative Commons licence, unless indicated otherwise in a credit line to the material. If material is not included in the article's Creative Commons licence and your intended use is not permitted by statutory regulation or exceeds the permitted use, you will need to obtain permission directly from the copyright holder. To view a copy of this licence, visit http://creativecommons.org/licenses/by/4.0/. The Creative Commons Public Domain Dedication waiver (http://creativecommons.org/publicdomain/zero/1.0/) applies to the data made available in this article, unless otherwise stated in a credit line to the data.
SN  - 1479-5876
EP  - 23
JF  - Journal of Translational Medicine
AV  - public
UR  - https://doi.org/10.1186/s12967-022-03685-0
KW  - Mitochondrial diseases
KW  -  Gene therapy
KW  -  Energy metabolism
KW  -  Mitochondrial DNA
KW  -  Heteroplasmy
TI  - Current advances in gene therapy of mitochondrial diseases
IS  - 1
PB  - BioMed Central
Y1  - 2022/12/05/
A1  - Soldatov, Vladislav
A1  - Deykin, Alexey
A1  - Skorkina, Marina
A1  - Kubekina, Marina
A1  - Belykh, Andrei
A1  - Egorova, Tatiana
A1  - Korokin, Mikhail
A1  - Pokrovskiy, Mikhail
A1  - Stroh, Plamena R
ER  -