In Vivo and Ex Vivo Gene Therapy for Inherited and Non-Inherited Disorders
Ongoing advances in pharmaceutical biotechnology have paved the way to ground-breaking new biological therapeutic modalities, offering the possibility of a durable curative approach for a number of life-threatening diseases, for which the medical need is as yet unmet. Over the past decades, gene the...
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| 格式: | Online |
|---|---|
| 語言: | 英语 |
| 出版: |
IntechOpen
2023
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| 主題: | |
| 在線閱讀: | ONIX_20231201_9781789857184_1892 |
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| _version_ | 1869519075277799424 |
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| collection | Directory of Open Access Books |
| description | Ongoing advances in pharmaceutical biotechnology have paved the way to ground-breaking new biological therapeutic modalities, offering the possibility of a durable curative approach for a number of life-threatening diseases, for which the medical need is as yet unmet. Over the past decades, gene therapy has seen a massive transformation from a proof-of-concept approach to a clinical reality culminating in the regulatory approval of state-of-the-art products in the European Union and in the United States. This book captures some of the scientific progresses notably in gene transfer technologies and translational development of in vivo and ex vivo gene therapy interventions in the treatment of a broad range of complex and debilitating non-inherited and inherited disorders such as: human immunodeficiency virus 1 (HIV-1) infection, cancer, cystic fibrosis, hereditary retinopathies, haemophilia B, cardiac diseases, and chronic liver fibrosis. |
| format | Online |
| id | doab-20.500.12854ir-130783 |
| institution | Directory of Open Access Books |
| language | eng |
| publishDate | 2023 |
| publishDateRange | 2023 |
| publishDateSort | 2023 |
| publisher | IntechOpen |
| publisherStr | IntechOpen |
| record_format | ojs |
| spelling | doab-20.500.12854ir-1307832024-03-31T22:45:22Z In Vivo and Ex Vivo Gene Therapy for Inherited and Non-Inherited Disorders Bachtarzi, Houria nanoparticles, heart failure, drug delivery, vaccine, hiv, cardiomyopathy thema EDItEUR::M Medicine and Nursing::MF Pre-clinical medicine: basic sciences::MFN Medical genetics Ongoing advances in pharmaceutical biotechnology have paved the way to ground-breaking new biological therapeutic modalities, offering the possibility of a durable curative approach for a number of life-threatening diseases, for which the medical need is as yet unmet. Over the past decades, gene therapy has seen a massive transformation from a proof-of-concept approach to a clinical reality culminating in the regulatory approval of state-of-the-art products in the European Union and in the United States. This book captures some of the scientific progresses notably in gene transfer technologies and translational development of in vivo and ex vivo gene therapy interventions in the treatment of a broad range of complex and debilitating non-inherited and inherited disorders such as: human immunodeficiency virus 1 (HIV-1) infection, cancer, cystic fibrosis, hereditary retinopathies, haemophilia B, cardiac diseases, and chronic liver fibrosis. 2023-12-01T18:15:08Z 2023-12-01T18:15:08Z 2019 book ONIX_20231201_9781789857184_1892 9781789857184 9781789857177 9781839620829 https://directory.doabooks.org/handle/20.500.12854/130783 eng image/jpeg n/a https://www.intechopen.com/books/6788 https://mts.intechopen.com/storage/books/6788/authors_book/authors_book.pdf IntechOpen IntechOpen 10.5772/intechopen.72962 10.5772/intechopen.72962 78a36484-2c0c-47cb-ad67-2b9f5cd4a8f6 9781789857184 9781789857177 9781839620829 IntechOpen 198 open access |
| spellingShingle | nanoparticles, heart failure, drug delivery, vaccine, hiv, cardiomyopathy thema EDItEUR::M Medicine and Nursing::MF Pre-clinical medicine: basic sciences::MFN Medical genetics In Vivo and Ex Vivo Gene Therapy for Inherited and Non-Inherited Disorders |
| title | In Vivo and Ex Vivo Gene Therapy for Inherited and Non-Inherited Disorders |
| title_full | In Vivo and Ex Vivo Gene Therapy for Inherited and Non-Inherited Disorders |
| title_fullStr | In Vivo and Ex Vivo Gene Therapy for Inherited and Non-Inherited Disorders |
| title_full_unstemmed | In Vivo and Ex Vivo Gene Therapy for Inherited and Non-Inherited Disorders |
| title_short | In Vivo and Ex Vivo Gene Therapy for Inherited and Non-Inherited Disorders |
| title_sort | in vivo and ex vivo gene therapy for inherited and non inherited disorders |
| topic | nanoparticles, heart failure, drug delivery, vaccine, hiv, cardiomyopathy thema EDItEUR::M Medicine and Nursing::MF Pre-clinical medicine: basic sciences::MFN Medical genetics |
| topic_facet | nanoparticles, heart failure, drug delivery, vaccine, hiv, cardiomyopathy thema EDItEUR::M Medicine and Nursing::MF Pre-clinical medicine: basic sciences::MFN Medical genetics |
| url | ONIX_20231201_9781789857184_1892 |