Immunity and AAV-mediated gene therapy for muscular dystrophies in large animal models and human trials
Adeno-associated viral (AAV) vector mediated gene replacement for the treatment of muscular dystrophy represents a promising therapeutic strategy in modern medicine. One major obstacle in using AAV vectors for in vivo gene delivery is the development of host immune responses to the viral capsid prot...
| Main Authors: | , , , |
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| Format: | Article |
| Language: | English |
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Frontiers Media S.A.
2011-09-01
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| Series: | Frontiers in Microbiology |
| Subjects: | |
| Online Access: | http://journal.frontiersin.org/Journal/10.3389/fmicb.2011.00201/full |
| _version_ | 1811258869788180480 |
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| author | Zejing eWang Stephen J Tapscott Stephen J Tapscott Jeffrey S Chamberlain Jeffrey S Chamberlain Rainer eStorb Rainer eStorb |
| author_facet | Zejing eWang Stephen J Tapscott Stephen J Tapscott Jeffrey S Chamberlain Jeffrey S Chamberlain Rainer eStorb Rainer eStorb |
| author_sort | Zejing eWang |
| collection | DOAJ |
| description | Adeno-associated viral (AAV) vector mediated gene replacement for the treatment of muscular dystrophy represents a promising therapeutic strategy in modern medicine. One major obstacle in using AAV vectors for in vivo gene delivery is the development of host immune responses to the viral capsid protein and transgene products as evidenced in animal models and human trials for a range of genetic diseases. Here, we review immunity against AAV vector and transgene in the context of gene delivery to muscles for treating muscular dystrophies, and immune modulatory strategies to prevent unwanted immune responses and induce tolerance for a successful gene therapy. |
| first_indexed | 2024-04-12T18:21:59Z |
| format | Article |
| id | doaj.art-b48724f4c77449c684b00f5a56e3bbb4 |
| institution | Directory Open Access Journal |
| issn | 1664-302X |
| language | English |
| last_indexed | 2024-04-12T18:21:59Z |
| publishDate | 2011-09-01 |
| publisher | Frontiers Media S.A. |
| record_format | Article |
| series | Frontiers in Microbiology |
| spelling | doaj.art-b48724f4c77449c684b00f5a56e3bbb42022-12-22T03:21:25ZengFrontiers Media S.A.Frontiers in Microbiology1664-302X2011-09-01210.3389/fmicb.2011.0020113158Immunity and AAV-mediated gene therapy for muscular dystrophies in large animal models and human trialsZejing eWang0Stephen J Tapscott1Stephen J Tapscott2Jeffrey S Chamberlain3Jeffrey S Chamberlain4Rainer eStorb5Rainer eStorb6Fred Hutchinson Cancer Research CenterFred Hutchinson Cancer Research CenterUniversity of WashingtonFred Hutchinson Cancer Research CenterUniversity of WashingtonFred Hutchinson Cancer Research CenterUniversity of Washington School of MedicineAdeno-associated viral (AAV) vector mediated gene replacement for the treatment of muscular dystrophy represents a promising therapeutic strategy in modern medicine. One major obstacle in using AAV vectors for in vivo gene delivery is the development of host immune responses to the viral capsid protein and transgene products as evidenced in animal models and human trials for a range of genetic diseases. Here, we review immunity against AAV vector and transgene in the context of gene delivery to muscles for treating muscular dystrophies, and immune modulatory strategies to prevent unwanted immune responses and induce tolerance for a successful gene therapy.http://journal.frontiersin.org/Journal/10.3389/fmicb.2011.00201/fullImmunitymuscular dystrophyreviewImmune Modulationaav |
| spellingShingle | Zejing eWang Stephen J Tapscott Stephen J Tapscott Jeffrey S Chamberlain Jeffrey S Chamberlain Rainer eStorb Rainer eStorb Immunity and AAV-mediated gene therapy for muscular dystrophies in large animal models and human trials Frontiers in Microbiology Immunity muscular dystrophy review Immune Modulation aav |
| title | Immunity and AAV-mediated gene therapy for muscular dystrophies in large animal models and human trials |
| title_full | Immunity and AAV-mediated gene therapy for muscular dystrophies in large animal models and human trials |
| title_fullStr | Immunity and AAV-mediated gene therapy for muscular dystrophies in large animal models and human trials |
| title_full_unstemmed | Immunity and AAV-mediated gene therapy for muscular dystrophies in large animal models and human trials |
| title_short | Immunity and AAV-mediated gene therapy for muscular dystrophies in large animal models and human trials |
| title_sort | immunity and aav mediated gene therapy for muscular dystrophies in large animal models and human trials |
| topic | Immunity muscular dystrophy review Immune Modulation aav |
| url | http://journal.frontiersin.org/Journal/10.3389/fmicb.2011.00201/full |
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