Correction: Current advances in gene therapy of mitochondrial diseases

Summary of https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9906933/

• Mitochondrial diseases are a group of inherited disorders caused by mutations in mitochondrial DNA or nuclear DNA.
• Gene therapy is a promising approach for the treatment of mitochondrial diseases.
• Recent advances in gene therapy have enabled the delivery of therapeutic genes to the mitochondria.
• Gene therapy strategies include the use of viral vectors, non-viral vectors, and gene editing.
• Gene therapy has been used to treat a variety of mitochondrial diseases, including Leigh syndrome, Leber's hereditary optic neuropathy, and mitochondrial encephalomyopathy.
• Gene therapy has also been used to treat mitochondrial diseases in animal models.
• The safety and efficacy of gene therapy for mitochondrial diseases is still being evaluated.

Recent Advances in Gene Therapy for Mitochondrial Diseases

Gene therapy is a promising approach for the treatment of mitochondrial diseases, which are a group of inherited disorders caused by mutations in mitochondrial DNA or nuclear DNA. Recent advances in gene therapy have enabled the delivery of therapeutic genes to the mitochondria, which can be used to correct the underlying genetic defect. Gene therapy strategies include the use of viral vectors, non-viral vectors, and gene editing.

Gene therapy has been used to treat a variety of mitochondrial diseases, including Leigh syndrome, Leber's hereditary optic neuropathy, and mitochondrial encephalomyopathy. Gene therapy has also been used to treat mitochondrial diseases in animal models. The safety and efficacy of gene therapy for mitochondrial diseases is still being evaluated.

Viral Vectors for Gene Therapy

Viral vectors are the most commonly used method for delivering therapeutic genes to the mitochondria. Viral vectors are modified viruses that have been engineered to carry a therapeutic gene. The most commonly used viral vectors for gene therapy are adenoviruses, adeno-associated viruses, and retroviruses. These vectors can be used to deliver genes to the mitochondria, where they can be expressed and used to correct the underlying genetic defect.

Non-Viral Vectors for Gene Therapy

Non-viral vectors are also used for gene therapy. Non-viral vectors are typically composed of synthetic molecules, such as DNA or RNA, that have been engineered to carry a therapeutic gene. These vectors can be used to deliver genes to the mitochondria, where they can be expressed and used to correct the underlying genetic defect.

Gene Editing for Mitochondrial Diseases

Gene editing is a powerful tool for treating mitochondrial diseases. Gene editing techniques, such as CRISPR/Cas9, can be used to directly modify the mitochondrial DNA and correct the underlying genetic defect. Gene editing has been used to treat a variety of mitochondrial diseases, including Leigh syndrome, Leber's hereditary optic neuropathy, and mitochondrial encephalomyopathy.

In conclusion, gene therapy is a promising approach for the treatment of mitochondrial diseases. Recent advances in gene therapy have enabled the delivery of therapeutic genes to the mitochondria, which can be used to correct the underlying genetic defect. Gene therapy strategies include the use of viral vectors, non-viral vectors, and gene editing. The safety and efficacy of gene therapy for mitochondrial diseases is still being evaluated.

source of this article

published: 2023;
Correction: Current advances in gene therapy of mitochondrial diseases

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