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...

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Bibliographic Details
Main Author: Fabio Candotti
Format: Article
Language:English
Published: F1000 Research Ltd 2016-03-01
Series:F1000Research
Subjects:
Online Access:http://f1000research.com/articles/5-310/v1
Description
Summary: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.
ISSN:2046-1402