Eintrag weiter verarbeiten
Development and Recent Progresses of Gene Therapy for β-Thalassemia
Gespeichert in:
| Zeitschriftentitel: | Thalassemia Reports |
|---|---|
| Personen und Körperschaften: | , , , , , |
| In: | Thalassemia Reports, 4, 2014, 2, S. 2925 |
| Format: | E-Article |
| Sprache: | Englisch |
| veröffentlicht: |
MDPI AG
|
| author_facet |
Acuto, Santina Baiamonte, Elena Di Stefano, Rosalia Spina, Barbara Barone, Rita Maggio, Aurelio Acuto, Santina Baiamonte, Elena Di Stefano, Rosalia Spina, Barbara Barone, Rita Maggio, Aurelio |
|---|---|
| author |
Acuto, Santina Baiamonte, Elena Di Stefano, Rosalia Spina, Barbara Barone, Rita Maggio, Aurelio |
| spellingShingle |
Acuto, Santina Baiamonte, Elena Di Stefano, Rosalia Spina, Barbara Barone, Rita Maggio, Aurelio Thalassemia Reports Development and Recent Progresses of Gene Therapy for β-Thalassemia |
| author_sort |
acuto, santina |
| spelling |
Acuto, Santina Baiamonte, Elena Di Stefano, Rosalia Spina, Barbara Barone, Rita Maggio, Aurelio 2039-4365 MDPI AG http://dx.doi.org/10.4081/thal.2014.2925 <jats:p>β-thalassemias are among the most common inherited monogenic disorders worldwide due to mutations in the β-globin gene that reduce or abolish the production of the β-globin chain resulting in transfusion-dependent chronic anemia. Currently, the only curative treatment is allogeneic hematopoietic stem cells (HSCs) transplantation, but this option is limited by the a vailability of HLA-matched donor. Gene therapy, based on autologous transplantation of genetically corrected HSCs, holds the promise to treat patients lacking a compati ble bone marrow donor. I nit ial attempts of gene transfer have been unsuccessful due to limitations of available vectors to stably transfer a globin gene in HSCs and reach high and regulated expression in the erythroid progeny. With the advent of lentiviral vectors (LVs), based on human immunodeficiency virus, many of the initial limitations have been overcome. Since 2000 when Sadelain and co-workers first demonstrated successful globin gene transfer in murine thalassemia models with improvement of the phenotype using a recombinant β globin/LV, several other groups have developed different vectors encoding either β, γ or mutated globin genes and confirmed these results in both murine models and erythroid progeny derived from patient’s HSCs. In light of these encouraging results, research has recently moved into clinical trials that are ongoing or soon to begin. One participant in an ongoing gene transfer trial for β-thalassemia has achieved clinical benefit with elimination of his transfusi on re quirement. Here , dev elopmen t and recent progress of gene therapy for β-thalassemia is reviewed.</jats:p> Development and Recent Progresses of Gene Therapy for β-Thalassemia Thalassemia Reports |
| doi_str_mv |
10.4081/thal.2014.2925 |
| facet_avail |
Online |
| format |
ElectronicArticle |
| fullrecord |
blob:ai-49-aHR0cDovL2R4LmRvaS5vcmcvMTAuNDA4MS90aGFsLjIwMTQuMjkyNQ |
| id |
ai-49-aHR0cDovL2R4LmRvaS5vcmcvMTAuNDA4MS90aGFsLjIwMTQuMjkyNQ |
| institution |
DE-Pl11 DE-Rs1 DE-105 DE-14 DE-Ch1 DE-L229 DE-D275 DE-Bn3 DE-Brt1 DE-D161 DE-Zwi2 DE-Gla1 DE-Zi4 DE-15 |
| imprint |
MDPI AG, 2014 |
| imprint_str_mv |
MDPI AG, 2014 |
| issn |
2039-4365 |
| issn_str_mv |
2039-4365 |
| language |
English |
| mega_collection |
MDPI AG (CrossRef) |
| match_str |
acuto2014developmentandrecentprogressesofgenetherapyforbthalassemia |
| publishDateSort |
2014 |
| publisher |
MDPI AG |
| recordtype |
ai |
| record_format |
ai |
| series |
Thalassemia Reports |
| source_id |
49 |
| title |
Development and Recent Progresses of Gene Therapy for β-Thalassemia |
| title_unstemmed |
Development and Recent Progresses of Gene Therapy for β-Thalassemia |
| title_full |
Development and Recent Progresses of Gene Therapy for β-Thalassemia |
| title_fullStr |
Development and Recent Progresses of Gene Therapy for β-Thalassemia |
| title_full_unstemmed |
Development and Recent Progresses of Gene Therapy for β-Thalassemia |
| title_short |
Development and Recent Progresses of Gene Therapy for β-Thalassemia |
| title_sort |
development and recent progresses of gene therapy for β-thalassemia |
| url |
http://dx.doi.org/10.4081/thal.2014.2925 |
| publishDate |
2014 |
| physical |
2925 |
| description |
<jats:p>β-thalassemias are among the most common inherited monogenic disorders worldwide due to mutations in the β-globin gene that reduce or abolish the production of the β-globin chain resulting in transfusion-dependent chronic anemia. Currently, the only curative treatment is allogeneic hematopoietic stem cells (HSCs) transplantation, but this option is limited by the a vailability of HLA-matched donor. Gene therapy, based on autologous transplantation of genetically corrected HSCs, holds the promise to treat patients lacking a compati ble bone marrow donor. I nit ial attempts of gene transfer have been unsuccessful due to limitations of available vectors to stably transfer a globin gene in HSCs and reach high and regulated expression in the erythroid progeny. With the advent of lentiviral vectors (LVs), based on human immunodeficiency virus, many of the initial limitations have been overcome. Since 2000 when Sadelain and co-workers first demonstrated successful globin gene transfer in murine thalassemia models with improvement of the phenotype using a recombinant β globin/LV, several other groups have developed different vectors encoding either β, γ or mutated globin genes and confirmed these results in both murine models and erythroid progeny derived from patient’s HSCs. In light of these encouraging results, research has recently moved into clinical trials that are ongoing or soon to begin. One participant in an ongoing gene transfer trial for β-thalassemia has achieved clinical benefit with elimination of his transfusi on re quirement. Here , dev elopmen t and recent progress of gene therapy for β-thalassemia is reviewed.</jats:p> |
| container_issue |
2 |
| container_start_page |
0 |
| container_title |
Thalassemia Reports |
| container_volume |
4 |
| format_de105 |
Article, E-Article |
| format_de14 |
Article, E-Article |
| format_de15 |
Article, E-Article |
| format_de520 |
Article, E-Article |
| format_de540 |
Article, E-Article |
| format_dech1 |
Article, E-Article |
| format_ded117 |
Article, E-Article |
| format_degla1 |
E-Article |
| format_del152 |
Buch |
| format_del189 |
Article, E-Article |
| format_dezi4 |
Article |
| format_dezwi2 |
Article, E-Article |
| format_finc |
Article, E-Article |
| format_nrw |
Article, E-Article |
| _version_ |
1792320992850214928 |
| geogr_code |
not assigned |
| last_indexed |
2024-03-01T10:54:56.56Z |
| geogr_code_person |
not assigned |
| openURL |
url_ver=Z39.88-2004&ctx_ver=Z39.88-2004&ctx_enc=info%3Aofi%2Fenc%3AUTF-8&rfr_id=info%3Asid%2Fvufind.svn.sourceforge.net%3Agenerator&rft.title=Development+and+Recent+Progresses+of+Gene+Therapy+for+%CE%B2-Thalassemia&rft.date=2014-09-29&genre=article&issn=2039-4365&volume=4&issue=2&pages=2925&jtitle=Thalassemia+Reports&atitle=Development+and+Recent+Progresses+of+Gene+Therapy+for+%CE%B2-Thalassemia&aulast=Maggio&aufirst=Aurelio&rft_id=info%3Adoi%2F10.4081%2Fthal.2014.2925&rft.language%5B0%5D=eng |
| SOLR | |
| _version_ | 1792320992850214928 |
| author | Acuto, Santina, Baiamonte, Elena, Di Stefano, Rosalia, Spina, Barbara, Barone, Rita, Maggio, Aurelio |
| author_facet | Acuto, Santina, Baiamonte, Elena, Di Stefano, Rosalia, Spina, Barbara, Barone, Rita, Maggio, Aurelio, Acuto, Santina, Baiamonte, Elena, Di Stefano, Rosalia, Spina, Barbara, Barone, Rita, Maggio, Aurelio |
| author_sort | acuto, santina |
| container_issue | 2 |
| container_start_page | 0 |
| container_title | Thalassemia Reports |
| container_volume | 4 |
| description | <jats:p>β-thalassemias are among the most common inherited monogenic disorders worldwide due to mutations in the β-globin gene that reduce or abolish the production of the β-globin chain resulting in transfusion-dependent chronic anemia. Currently, the only curative treatment is allogeneic hematopoietic stem cells (HSCs) transplantation, but this option is limited by the a vailability of HLA-matched donor. Gene therapy, based on autologous transplantation of genetically corrected HSCs, holds the promise to treat patients lacking a compati ble bone marrow donor. I nit ial attempts of gene transfer have been unsuccessful due to limitations of available vectors to stably transfer a globin gene in HSCs and reach high and regulated expression in the erythroid progeny. With the advent of lentiviral vectors (LVs), based on human immunodeficiency virus, many of the initial limitations have been overcome. Since 2000 when Sadelain and co-workers first demonstrated successful globin gene transfer in murine thalassemia models with improvement of the phenotype using a recombinant β globin/LV, several other groups have developed different vectors encoding either β, γ or mutated globin genes and confirmed these results in both murine models and erythroid progeny derived from patient’s HSCs. In light of these encouraging results, research has recently moved into clinical trials that are ongoing or soon to begin. One participant in an ongoing gene transfer trial for β-thalassemia has achieved clinical benefit with elimination of his transfusi on re quirement. Here , dev elopmen t and recent progress of gene therapy for β-thalassemia is reviewed.</jats:p> |
| doi_str_mv | 10.4081/thal.2014.2925 |
| facet_avail | Online |
| format | ElectronicArticle |
| format_de105 | Article, E-Article |
| format_de14 | Article, E-Article |
| format_de15 | Article, E-Article |
| format_de520 | Article, E-Article |
| format_de540 | Article, E-Article |
| format_dech1 | Article, E-Article |
| format_ded117 | Article, E-Article |
| format_degla1 | E-Article |
| format_del152 | Buch |
| format_del189 | Article, E-Article |
| format_dezi4 | Article |
| format_dezwi2 | Article, E-Article |
| format_finc | Article, E-Article |
| format_nrw | Article, E-Article |
| geogr_code | not assigned |
| geogr_code_person | not assigned |
| id | ai-49-aHR0cDovL2R4LmRvaS5vcmcvMTAuNDA4MS90aGFsLjIwMTQuMjkyNQ |
| imprint | MDPI AG, 2014 |
| imprint_str_mv | MDPI AG, 2014 |
| institution | DE-Pl11, DE-Rs1, DE-105, DE-14, DE-Ch1, DE-L229, DE-D275, DE-Bn3, DE-Brt1, DE-D161, DE-Zwi2, DE-Gla1, DE-Zi4, DE-15 |
| issn | 2039-4365 |
| issn_str_mv | 2039-4365 |
| language | English |
| last_indexed | 2024-03-01T10:54:56.56Z |
| match_str | acuto2014developmentandrecentprogressesofgenetherapyforbthalassemia |
| mega_collection | MDPI AG (CrossRef) |
| physical | 2925 |
| publishDate | 2014 |
| publishDateSort | 2014 |
| publisher | MDPI AG |
| record_format | ai |
| recordtype | ai |
| series | Thalassemia Reports |
| source_id | 49 |
| spelling | Acuto, Santina Baiamonte, Elena Di Stefano, Rosalia Spina, Barbara Barone, Rita Maggio, Aurelio 2039-4365 MDPI AG http://dx.doi.org/10.4081/thal.2014.2925 <jats:p>β-thalassemias are among the most common inherited monogenic disorders worldwide due to mutations in the β-globin gene that reduce or abolish the production of the β-globin chain resulting in transfusion-dependent chronic anemia. Currently, the only curative treatment is allogeneic hematopoietic stem cells (HSCs) transplantation, but this option is limited by the a vailability of HLA-matched donor. Gene therapy, based on autologous transplantation of genetically corrected HSCs, holds the promise to treat patients lacking a compati ble bone marrow donor. I nit ial attempts of gene transfer have been unsuccessful due to limitations of available vectors to stably transfer a globin gene in HSCs and reach high and regulated expression in the erythroid progeny. With the advent of lentiviral vectors (LVs), based on human immunodeficiency virus, many of the initial limitations have been overcome. Since 2000 when Sadelain and co-workers first demonstrated successful globin gene transfer in murine thalassemia models with improvement of the phenotype using a recombinant β globin/LV, several other groups have developed different vectors encoding either β, γ or mutated globin genes and confirmed these results in both murine models and erythroid progeny derived from patient’s HSCs. In light of these encouraging results, research has recently moved into clinical trials that are ongoing or soon to begin. One participant in an ongoing gene transfer trial for β-thalassemia has achieved clinical benefit with elimination of his transfusi on re quirement. Here , dev elopmen t and recent progress of gene therapy for β-thalassemia is reviewed.</jats:p> Development and Recent Progresses of Gene Therapy for β-Thalassemia Thalassemia Reports |
| spellingShingle | Acuto, Santina, Baiamonte, Elena, Di Stefano, Rosalia, Spina, Barbara, Barone, Rita, Maggio, Aurelio, Thalassemia Reports, Development and Recent Progresses of Gene Therapy for β-Thalassemia |
| title | Development and Recent Progresses of Gene Therapy for β-Thalassemia |
| title_full | Development and Recent Progresses of Gene Therapy for β-Thalassemia |
| title_fullStr | Development and Recent Progresses of Gene Therapy for β-Thalassemia |
| title_full_unstemmed | Development and Recent Progresses of Gene Therapy for β-Thalassemia |
| title_short | Development and Recent Progresses of Gene Therapy for β-Thalassemia |
| title_sort | development and recent progresses of gene therapy for β-thalassemia |
| title_unstemmed | Development and Recent Progresses of Gene Therapy for β-Thalassemia |
| url | http://dx.doi.org/10.4081/thal.2014.2925 |