Development of lentiviral gene therapy for Wiskott Aldrich syndrome.

ABSTRACT:

Background

Wiskott Aldrich syndrome (WAS) is a rare X-linked primary immunodeficiency. This complex disease is characterised by microthrombocytopenia, recurrent infections, eczema and is associated with a high incidence of autoimmunity and of lymphoid malignancies. WAS is attracting growing attention not only because it highlights the rich cellular and systems biology revolving around cytoskeletal regulation but also because it is candidate for a haematopoietic stem cell gene therapy indication.

Objectives

As several groups are developing this novel approach, this review discusses the state of the art and challenges in clinical development of gene therapy for WAS, with particular regard to biosafety.

Methods

In spite of the successes of haematopoietic gene therapy for

SUBMITTER: Galy A 

PROVIDER: S-EPMC2789278 | biostudies-literature | 2008 Feb

REPOSITORIES: biostudies-literature