Synthetic Promoters in Gene Therapy: Design Approaches, Features and Applications
Gene therapy is a promising approach to the treatment of various inherited diseases, but its development is complicated by a number of limitations of the natural promoters used. The currently used strong ubiquitous natural promoters do not allow for the specificity of expression, while natural tissu...
Saved in:
| Main Authors: | , , , , , , , |
|---|---|
| Format: | Article |
| Language: | English |
| Published: |
MDPI AG
2024-11-01
|
| Series: | Cells |
| Subjects: | |
| Online Access: | https://www.mdpi.com/2073-4409/13/23/1963 |
| Tags: |
Add Tag
No Tags, Be the first to tag this record!
|
| _version_ | 1850260052804370432 |
|---|---|
| author | Valentin Artemyev Anna Gubaeva Anastasiia Iu. Paremskaia Amina A. Dzhioeva Andrei Deviatkin Sofya G. Feoktistova Olga Mityaeva Pavel Yu. Volchkov |
| author_facet | Valentin Artemyev Anna Gubaeva Anastasiia Iu. Paremskaia Amina A. Dzhioeva Andrei Deviatkin Sofya G. Feoktistova Olga Mityaeva Pavel Yu. Volchkov |
| author_sort | Valentin Artemyev |
| collection | DOAJ |
| description | Gene therapy is a promising approach to the treatment of various inherited diseases, but its development is complicated by a number of limitations of the natural promoters used. The currently used strong ubiquitous natural promoters do not allow for the specificity of expression, while natural tissue-specific promoters have lowactivity. These limitations of natural promoters can be addressed by creating new synthetic promoters that achieve high levels of tissue-specific target gene expression. This review discusses recent advances in the development of synthetic promoters that provide a more precise regulation of gene expression. Approaches to the design of synthetic promoters are reviewed, including manual design and bioinformatic methods using machine learning. Examples of successful applications of synthetic promoters in the therapy of hereditary diseases and cancer are presented, as well as prospects for their clinical use. |
| format | Article |
| id | doaj-art-90aeefefeb5e451faaa87baa86e77071 |
| institution | OA Journals |
| issn | 2073-4409 |
| language | English |
| publishDate | 2024-11-01 |
| publisher | MDPI AG |
| record_format | Article |
| series | Cells |
| spelling | doaj-art-90aeefefeb5e451faaa87baa86e770712025-08-20T01:55:44ZengMDPI AGCells2073-44092024-11-011323196310.3390/cells13231963Synthetic Promoters in Gene Therapy: Design Approaches, Features and ApplicationsValentin Artemyev0Anna Gubaeva1Anastasiia Iu. Paremskaia2Amina A. Dzhioeva3Andrei Deviatkin4Sofya G. Feoktistova5Olga Mityaeva6Pavel Yu. Volchkov7Federal Research Center for Innovator and Emerging Biomedical and Pharmaceutical Technologies, 125315 Moscow, RussiaFederal Research Center for Innovator and Emerging Biomedical and Pharmaceutical Technologies, 125315 Moscow, RussiaFederal Research Center for Innovator and Emerging Biomedical and Pharmaceutical Technologies, 125315 Moscow, RussiaMoscow Center for Advanced Studies, Kulakova Str. 20, 123592 Moscow, RussiaFederal Research Center for Innovator and Emerging Biomedical and Pharmaceutical Technologies, 125315 Moscow, RussiaFederal Research Center for Innovator and Emerging Biomedical and Pharmaceutical Technologies, 125315 Moscow, RussiaFederal Research Center for Innovator and Emerging Biomedical and Pharmaceutical Technologies, 125315 Moscow, RussiaFederal Research Center for Innovator and Emerging Biomedical and Pharmaceutical Technologies, 125315 Moscow, RussiaGene therapy is a promising approach to the treatment of various inherited diseases, but its development is complicated by a number of limitations of the natural promoters used. The currently used strong ubiquitous natural promoters do not allow for the specificity of expression, while natural tissue-specific promoters have lowactivity. These limitations of natural promoters can be addressed by creating new synthetic promoters that achieve high levels of tissue-specific target gene expression. This review discusses recent advances in the development of synthetic promoters that provide a more precise regulation of gene expression. Approaches to the design of synthetic promoters are reviewed, including manual design and bioinformatic methods using machine learning. Examples of successful applications of synthetic promoters in the therapy of hereditary diseases and cancer are presented, as well as prospects for their clinical use.https://www.mdpi.com/2073-4409/13/23/1963synthetic promotersgene therapypromoter designeukaryotic promotersgene expression |
| spellingShingle | Valentin Artemyev Anna Gubaeva Anastasiia Iu. Paremskaia Amina A. Dzhioeva Andrei Deviatkin Sofya G. Feoktistova Olga Mityaeva Pavel Yu. Volchkov Synthetic Promoters in Gene Therapy: Design Approaches, Features and Applications Cells synthetic promoters gene therapy promoter design eukaryotic promoters gene expression |
| title | Synthetic Promoters in Gene Therapy: Design Approaches, Features and Applications |
| title_full | Synthetic Promoters in Gene Therapy: Design Approaches, Features and Applications |
| title_fullStr | Synthetic Promoters in Gene Therapy: Design Approaches, Features and Applications |
| title_full_unstemmed | Synthetic Promoters in Gene Therapy: Design Approaches, Features and Applications |
| title_short | Synthetic Promoters in Gene Therapy: Design Approaches, Features and Applications |
| title_sort | synthetic promoters in gene therapy design approaches features and applications |
| topic | synthetic promoters gene therapy promoter design eukaryotic promoters gene expression |
| url | https://www.mdpi.com/2073-4409/13/23/1963 |
| work_keys_str_mv | AT valentinartemyev syntheticpromotersingenetherapydesignapproachesfeaturesandapplications AT annagubaeva syntheticpromotersingenetherapydesignapproachesfeaturesandapplications AT anastasiiaiuparemskaia syntheticpromotersingenetherapydesignapproachesfeaturesandapplications AT aminaadzhioeva syntheticpromotersingenetherapydesignapproachesfeaturesandapplications AT andreideviatkin syntheticpromotersingenetherapydesignapproachesfeaturesandapplications AT sofyagfeoktistova syntheticpromotersingenetherapydesignapproachesfeaturesandapplications AT olgamityaeva syntheticpromotersingenetherapydesignapproachesfeaturesandapplications AT pavelyuvolchkov syntheticpromotersingenetherapydesignapproachesfeaturesandapplications |