Targeting transgene expression to antigen-presenting cells derived from lentivirus-transduced engrafting human hematopoietic stem/progenitor cells

Dangerous liaisons: the role of “danger” signals in the immune response to gene therapy

“…SCs, unlike committed cells, begin to differentiate into various tissues, including cells involved in antigen presentation, such as DCs and macrophages. 88 Under steady-state, nondangerous, conditions, the transgenic antigen would be presented to naive T cells in the absence of costimulation, inducing T-cell anergy and resulting in antigen-specific tolerance.…”

Lentiviral vectors transcriptionally targeted to hematopoietic cells by WASP gene proximal promoter sequences

“…11 This has facilitated the development of transcriptionally regulated vectors. [12][13][14] The Wiskott-Aldrich syndrome (WAS) is a X-linked condition characterized clinically by profound and progressive immunodeficiency, eczema and severely decreased number of platelets (reviewed in Snapper and Rosen 15 and Thrasher 16 ). Patients with classical WAS have heterogeneous mutations in the WASP gene, [17][18][19] which usually result in the absence or very diminished expression of its encoded gene product (WASp), or otherwise give rise to non-functional truncated proteins.…”

Single particle high resolution spectral analysis flow cytometry

“…They were grown and harvested following the procedure described in Cui et al (32). The cells were fixed in 70% ethanol, resuspended in PBS, and stained with PI at a concentration of 5 lg/ml.…”