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 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...

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Autor principal:	Nikolova D.
Formato:	article
Lenguaje:	EN
Publicado:	Sciendo 2021
Materias:	gene therapy hematological diseases gene therapy drugs Medicine R
Acceso en línea:	https://doaj.org/article/8bfc36e42aee48cb9182a12ab93d3cc2
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spelling	oai:doaj.org-article:8bfc36e42aee48cb9182a12ab93d3cc22021-12-05T14:11:06ZCurrent Trends in the Gene Therapy of Hematologic Disorders2719-538410.2478/amb-2021-0048https://doaj.org/article/8bfc36e42aee48cb9182a12ab93d3cc22021-11-01T00:00:00Zhttps://doi.org/10.2478/amb-2021-0048https://doaj.org/toc/2719-5384Recent 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.Nikolova D.Sciendoarticlegene therapyhematological diseasesgene therapy drugsMedicineRENActa Medica Bulgarica, Vol 48, Iss 4, Pp 50-56 (2021)
institution	DOAJ
collection	DOAJ
language	EN
topic	gene therapy hematological diseases gene therapy drugs Medicine R
spellingShingle	gene therapy hematological diseases gene therapy drugs Medicine R Nikolova D. Current Trends in the Gene Therapy of Hematologic Disorders
description	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.
format	article
author	Nikolova D.
author_facet	Nikolova D.
author_sort	Nikolova D.
title	Current Trends in the Gene Therapy of Hematologic Disorders
title_short	Current Trends in the Gene Therapy of Hematologic Disorders
title_full	Current Trends in the Gene Therapy of Hematologic Disorders
title_fullStr	Current Trends in the Gene Therapy of Hematologic Disorders
title_full_unstemmed	Current Trends in the Gene Therapy of Hematologic Disorders
title_sort	current trends in the gene therapy of hematologic disorders
publisher	Sciendo
publishDate	2021
url	https://doaj.org/article/8bfc36e42aee48cb9182a12ab93d3cc2
work_keys_str_mv	AT nikolovad currenttrendsinthegenetherapyofhematologicdisorders
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Current Trends in the Gene Therapy of Hematologic Disorders

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