Advances of gene therapy for primary immunodeficiencies.

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Serval ID
serval:BIB_7FF778D09709
Type
Article: article from journal or magazin.
Publication sub-type
Review (review): journal as complete as possible of one specific subject, written based on exhaustive analyses from published work.
Collection
Publications
Institution
Title
Advances of gene therapy for primary immunodeficiencies.
Journal
F1000research
Author(s)
Candotti F.
ISSN
2046-1402 (Electronic)
ISSN-L
2046-1402
Publication state
Published
Issued date
2016
Peer-reviewed
Oui
Volume
5
Pages
31
Language
english
Abstract
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.
Pubmed
Open Access
Yes
Create date
11/08/2016 13:16
Last modification date
20/08/2019 14:40
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