Gene therapy – Principles and applications
The concept of gene therapy dates back to the 1960s and 1970s and it is yet to come of age, as there is a paucity of reliable, long-term data on the safety and efficacy of this novel therapy. It is fast emerging as a promising approach to treat, cure or prevent genetic based diseases by correction o...
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| Format: | Article |
| Language: | English |
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Wolters Kluwer Medknow Publications
2022-01-01
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| Series: | Global Journal of Transfusion Medicine |
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| Online Access: | http://www.gjtmonline.com/article.asp?issn=2468-8398;year=2022;volume=7;issue=1;spage=3;epage=6;aulast=Pathak |
| _version_ | 1811243250361565184 |
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| author | Sangeeta Pathak |
| author_facet | Sangeeta Pathak |
| author_sort | Sangeeta Pathak |
| collection | DOAJ |
| description | The concept of gene therapy dates back to the 1960s and 1970s and it is yet to come of age, as there is a paucity of reliable, long-term data on the safety and efficacy of this novel therapy. It is fast emerging as a promising approach to treat, cure or prevent genetic based diseases by correction of defective genes that are responsible for disease development. Gene therapy is a key treatment strategy for disorders caused by a missing or faulty gene. It involves introduction of functional genes into appropriate cells so that they can produce sufficient amounts of proteins, encoded by the transferred gene or turn off genes that are causing problems and eventually resulting in permanent correction of the disorder. While the concept of gene replacement therapy is mostly suitable for recessive diseases, novel strategies have been suggested that are capable of also treating conditions with a dominant pattern of inheritance. Gene therapy treatments are of broadly two types, Germline gene therapy, & Somatic Germline gene therapy. There are two methods for administering gene therapy -Ex vivo & in-vivo. There are two main parts to gene replacement: Genes and Vectors. Gene therapy is useful for hematologic conditions as Hemoglobinopathies, Hemophilia and Inherited bone marrow failure. Future Prospective of Gene Therapy in Transfusion Medicine ,work is being done to explore ex vivo generation of RBCs utilizing various cell fractions, including hematopeitic progenitor cells from cord blood and adult sources. Cost of therapy and ethical issues are the limiting factors for the use of Gene therapy. |
| first_indexed | 2024-04-12T14:04:09Z |
| format | Article |
| id | doaj.art-56f9863e95014970879a6f426f789e4e |
| institution | Directory Open Access Journal |
| issn | 2468-8398 2455-8893 |
| language | English |
| last_indexed | 2024-04-12T14:04:09Z |
| publishDate | 2022-01-01 |
| publisher | Wolters Kluwer Medknow Publications |
| record_format | Article |
| series | Global Journal of Transfusion Medicine |
| spelling | doaj.art-56f9863e95014970879a6f426f789e4e2022-12-22T03:30:07ZengWolters Kluwer Medknow PublicationsGlobal Journal of Transfusion Medicine2468-83982455-88932022-01-01713610.4103/gjtm.gjtm_87_21Gene therapy – Principles and applicationsSangeeta PathakThe concept of gene therapy dates back to the 1960s and 1970s and it is yet to come of age, as there is a paucity of reliable, long-term data on the safety and efficacy of this novel therapy. It is fast emerging as a promising approach to treat, cure or prevent genetic based diseases by correction of defective genes that are responsible for disease development. Gene therapy is a key treatment strategy for disorders caused by a missing or faulty gene. It involves introduction of functional genes into appropriate cells so that they can produce sufficient amounts of proteins, encoded by the transferred gene or turn off genes that are causing problems and eventually resulting in permanent correction of the disorder. While the concept of gene replacement therapy is mostly suitable for recessive diseases, novel strategies have been suggested that are capable of also treating conditions with a dominant pattern of inheritance. Gene therapy treatments are of broadly two types, Germline gene therapy, & Somatic Germline gene therapy. There are two methods for administering gene therapy -Ex vivo & in-vivo. There are two main parts to gene replacement: Genes and Vectors. Gene therapy is useful for hematologic conditions as Hemoglobinopathies, Hemophilia and Inherited bone marrow failure. Future Prospective of Gene Therapy in Transfusion Medicine ,work is being done to explore ex vivo generation of RBCs utilizing various cell fractions, including hematopeitic progenitor cells from cord blood and adult sources. Cost of therapy and ethical issues are the limiting factors for the use of Gene therapy.http://www.gjtmonline.com/article.asp?issn=2468-8398;year=2022;volume=7;issue=1;spage=3;epage=6;aulast=Pathakapplicationsgene therapyprinciplessteps in gene therapy gene alterations |
| spellingShingle | Sangeeta Pathak Gene therapy – Principles and applications Global Journal of Transfusion Medicine applications gene therapy principles steps in gene therapy gene alterations |
| title | Gene therapy – Principles and applications |
| title_full | Gene therapy – Principles and applications |
| title_fullStr | Gene therapy – Principles and applications |
| title_full_unstemmed | Gene therapy – Principles and applications |
| title_short | Gene therapy – Principles and applications |
| title_sort | gene therapy principles and applications |
| topic | applications gene therapy principles steps in gene therapy gene alterations |
| url | http://www.gjtmonline.com/article.asp?issn=2468-8398;year=2022;volume=7;issue=1;spage=3;epage=6;aulast=Pathak |
| work_keys_str_mv | AT sangeetapathak genetherapyprinciplesandapplications |