pDRIVE-WASP

WASP promoter (Hematopoietic cells) in pDRIVE expression plasmid

The Wiskott–Aldrich Syndrome protein (WASP) is a major cytoskeletal regulator expressed in hematopoietic cells. Mutations in the WASP gene are associated with Wiskott‑Aldrich X-linked immunodeficiency as well as isolated X-linked thrombocytopenia (XLT). In these patients, WASP is usually significantly reduced or absent.

The WASP gene is a candidate for hematopoietic stem cell gene therapy. Indeed, researchers have demonstrated that lentiviral vectors incorporating proximal promoter sequences from the WASP gene promoter confer hematopoietic-specific and physiological protein expression [1].

Specifically, a 500-bp fragment from the proximal WASP gene promoter was shown to direct expression of a transgene. This fragment contains four potential binding sites for hematopoietic transcription factors (c-Rel, PU.1, and two ets-1 motifs) [2].

References:

1. Martin F. et al., 2005. Lentiviral vectors transcriptionally targeted to hematopoietic cells by WASP gene proximal promoter sequences. Gene Ther. 12(8):715-23.
2. Ptrella A. et al., 1998. A 5' regulatory sequence containing two Ets motifs controls the expression of the Wiskott-Aldrich syndrome protein (WASP) gene in human hematopoietic cells. Blood. 91(12):4554-60.