<abstract xmlns="http://www.w3.org/1999/xhtml">

<p>Recent advances in molecular genetics and the invention of new technologies led to an advance in the development of gene therapy. Gene therapy is used to correct defective genes in order to cure a disease or help the body better fight a disease. It works by restoring or modifying cellular functions through the introduction of a functional gene into the target cell. The concept of gene therapy is simple, but introducing it to routine clinical practice is not. The main concerns are related to some safety issues as well as to the problem that maintaining a stable and prolonged expression in target cells may not be easily achieved. In spite of the difficulties, gene therapy remains a hope for many hematological disorders that cannot be effectively treated so far. This article reviews the current status of gene therapy with a focus on hematological disorders. In addition, clinically applied approaches are presented through particular examples of approved gene therapy drugs.</p>
</abstract>

Recent advances in molecular genetics and the invention of new technologies led to an advance in the development of gene therapy. Gene therapy is used to correct defective genes in order to cure a disease or help the body better fight a disease. It works by restoring or modifying cellular functions through the introduction of a functional gene into the target cell. The concept of gene therapy is simple, but introducing it to routine clinical practice is not. The main concerns are related to some safety issues as well as to the problem that maintaining a stable and prolonged expression in target cells may not be easily achieved. In spite of the difficulties, gene therapy remains a hope for many hematological disorders that cannot be effectively treated so far. This article reviews the current status of gene therapy with a focus on hematological disorders. In addition, clinically applied approaches are presented through particular examples of approved gene therapy drugs.

Current Trends in the Gene Therapy of Hematologic Disorders

University Hospital “Sv. Ivan Rilski” – Sofia

Acta Medica Bulgarica

This work is licensed under the Creative Commons Attribution-NonCommercial-NoDerivatives 4.0 International License.

{"article-title":"Current Trends in the Gene Therapy of Hematologic Disorders"}

Recent advances in molecular genetics and the invention of new technologies led to an advance in the development of gene therapy. Gene therapy is used to...

Current Trends in the Gene Therapy of Hematologic Disorders

Data publikacji: 20 lis 2021

Zakres stron: 50 - 56

Otrzymano: 24 mar 2021

Przyjęty: 18 kwi 2021

DOI: https://doi.org/10.2478/amb-2021-0048

Słowa kluczowe
gene therapy, hematological diseases, gene therapy drugs

© 2021 D. Nikolova, published by Sciendo

This work is licensed under the Creative Commons Attribution-NonCommercial-NoDerivatives 4.0 International License.

Current Trends in the Gene Therapy of Hematologic Disorders

Data publikacji: 20 lis 2021

Zakres stron: 50 - 56

Otrzymano: 24 mar 2021

Przyjęty: 18 kwi 2021

DOI: https://doi.org/10.2478/amb-2021-0048

Słowa kluczowegene therapy, hematological diseases, gene therapy drugs

© 2021 D. Nikolova, published by Sciendo

This work is licensed under the Creative Commons Attribution-NonCommercial-NoDerivatives 4.0 International License.

Słowa kluczowe
gene therapy, hematological diseases, gene therapy drugs