|
ИСТИНА |
Войти в систему Регистрация |
Интеллектуальная Система Тематического Исследования НАукометрических данных |
||
Engineering Liver-Specific Promoters: A Comprehensive Review of Design, Mechanisms, and Clinical Applications in Gene Therapyстатья Электронная публикация
- Авторы: Artemyev Valentin, Paremskaia Anastasiia Iu, Dzhioeva Amina A., Mishina Daria, Bogdanov Viktor, Krupinova Julia, Mazloum Ali, Feoktistova Sofya G., Mityaeva Olga N., Volchkov Pavel Yu
- Журнал: Cells
- Том: 15
- Номер: 1
- Год издания: 2026
- Издательство: MDPI
- Местоположение издательства: Basel, Switzerland
- Номер статьи: 14
- DOI: 10.3390/cells15010014
- Аннотация: The liver is a primary metabolic hub and a pivotal target for gene therapy, owing to its capacity for protein secretion, role in metabolic homeostasis and immune tolerance. Liver-directed gene therapies are used to treat numerous inherited metabolic disorders and coagulation factor deficiencies including hemophilia (A and B), Crigler–Najjar syndrome, mucopolysaccharidoses, phenylketonuria, Fabry, Gaucher, Wilson and Pompe diseases. The efficacy and safety of liver-directed gene therapy rely on the use of strong tissue-specific promoters. To date, there are many different liver-specific promoters used in preclinical and clinical studies, including novel completely synthetic promoters. This review provides a comprehensive analysis of the design, engineering and application of liver-specific promoters. Furthermore, we discuss fundamental principles of gene expression regulation in the liver and the physiological and immunological characteristics that make it a suitable target organ for gene therapy delivery.
- Добавил в систему: Паремская Анастасия Юрьевна