Advances of gene therapy for primary immunodeficiencies [version 1; referees: 2 approved]
In the recent past, the gene therapy field has witnessed a remarkable series of successes, many of which have involved primary immunodeficiency diseases, such as X-linked severe combined immunodeficiency, adenosine deaminase deficiency, chronic granulomatous disease, and Wiskott-Aldrich syndrome. Wh...
| Main Author: | |
|---|---|
| Format: | Article |
| Language: | English |
| Published: |
F1000 Research Ltd
2016-03-01
|
| Series: | F1000Research |
| Subjects: | |
| Online Access: | http://f1000research.com/articles/5-310/v1 |
| _version_ | 1818370440603631616 |
|---|---|
| author | Fabio Candotti |
| author_facet | Fabio Candotti |
| author_sort | Fabio Candotti |
| collection | DOAJ |
| description | In the recent past, the gene therapy field has witnessed a remarkable series of successes, many of which have involved primary immunodeficiency diseases, such as X-linked severe combined immunodeficiency, adenosine deaminase deficiency, chronic granulomatous disease, and Wiskott-Aldrich syndrome. While such progress has widened the choice of therapeutic options in some specific cases of primary immunodeficiency, much remains to be done to extend the geographical availability of such an advanced approach and to increase the number of diseases that can be targeted. At the same time, emerging technologies are stimulating intensive investigations that may lead to the application of precise genetic editing as the next form of gene therapy for these and other human genetic diseases. |
| first_indexed | 2024-12-13T23:39:46Z |
| format | Article |
| id | doaj.art-54f83b8dbce648b5bbdb3804f282c750 |
| institution | Directory Open Access Journal |
| issn | 2046-1402 |
| language | English |
| last_indexed | 2024-12-13T23:39:46Z |
| publishDate | 2016-03-01 |
| publisher | F1000 Research Ltd |
| record_format | Article |
| series | F1000Research |
| spelling | doaj.art-54f83b8dbce648b5bbdb3804f282c7502022-12-21T23:27:12ZengF1000 Research LtdF1000Research2046-14022016-03-01510.12688/f1000research.7512.18091Advances of gene therapy for primary immunodeficiencies [version 1; referees: 2 approved]Fabio Candotti0Division of Immunology and Allergy, University Hospital of Lausanne, Lausanne, SwitzerlandIn the recent past, the gene therapy field has witnessed a remarkable series of successes, many of which have involved primary immunodeficiency diseases, such as X-linked severe combined immunodeficiency, adenosine deaminase deficiency, chronic granulomatous disease, and Wiskott-Aldrich syndrome. While such progress has widened the choice of therapeutic options in some specific cases of primary immunodeficiency, much remains to be done to extend the geographical availability of such an advanced approach and to increase the number of diseases that can be targeted. At the same time, emerging technologies are stimulating intensive investigations that may lead to the application of precise genetic editing as the next form of gene therapy for these and other human genetic diseases.http://f1000research.com/articles/5-310/v1Genetics of the Immune SystemHematopoietic Stem CellsImmunomodulationImmunopharmacology & Hematologic PharmacologyMedical GeneticsMedical MicrobiologyNon-hematopoietic Stem CellsPediatric HematologyPediatric Infectious DiseasesPharmacokinetics & Drug DeliveryStem Cells & Regeneration |
| spellingShingle | Fabio Candotti Advances of gene therapy for primary immunodeficiencies [version 1; referees: 2 approved] F1000Research Genetics of the Immune System Hematopoietic Stem Cells Immunomodulation Immunopharmacology & Hematologic Pharmacology Medical Genetics Medical Microbiology Non-hematopoietic Stem Cells Pediatric Hematology Pediatric Infectious Diseases Pharmacokinetics & Drug Delivery Stem Cells & Regeneration |
| title | Advances of gene therapy for primary immunodeficiencies [version 1; referees: 2 approved] |
| title_full | Advances of gene therapy for primary immunodeficiencies [version 1; referees: 2 approved] |
| title_fullStr | Advances of gene therapy for primary immunodeficiencies [version 1; referees: 2 approved] |
| title_full_unstemmed | Advances of gene therapy for primary immunodeficiencies [version 1; referees: 2 approved] |
| title_short | Advances of gene therapy for primary immunodeficiencies [version 1; referees: 2 approved] |
| title_sort | advances of gene therapy for primary immunodeficiencies version 1 referees 2 approved |
| topic | Genetics of the Immune System Hematopoietic Stem Cells Immunomodulation Immunopharmacology & Hematologic Pharmacology Medical Genetics Medical Microbiology Non-hematopoietic Stem Cells Pediatric Hematology Pediatric Infectious Diseases Pharmacokinetics & Drug Delivery Stem Cells & Regeneration |
| url | http://f1000research.com/articles/5-310/v1 |
| work_keys_str_mv | AT fabiocandotti advancesofgenetherapyforprimaryimmunodeficienciesversion1referees2approved |