[HTML][HTML] Targeting platelet GPIbα transgene expression to human megakaryocytes and forming a complete complex with endogenous GPIbβ and GPIX
Q Shi, DA Wilcox, PA Morateck, SA Fahs… - Journal of Thrombosis …, 2004 - Elsevier
Summary Bernard–Soulier Syndrome (BSS) is a severe congenital platelet disorder that
results from a deficiency of the platelet membrane glycoprotein (GP) Ib/IX complex that is
composed of four subunits (GPIbα, GPIbβ, GPIX, and GPV). Mutations in either GPIbα,
GPIbβ, or GPIX can result in BSS with many of the known mutations occurring in GPIbα. In
this study, we have developed a gene therapy strategy to express hemagglutinin (HA)‐
tagged GPIbα in megakaryocytes and potentially correct a hereditary deficiency. To direct …
results from a deficiency of the platelet membrane glycoprotein (GP) Ib/IX complex that is
composed of four subunits (GPIbα, GPIbβ, GPIX, and GPV). Mutations in either GPIbα,
GPIbβ, or GPIX can result in BSS with many of the known mutations occurring in GPIbα. In
this study, we have developed a gene therapy strategy to express hemagglutinin (HA)‐
tagged GPIbα in megakaryocytes and potentially correct a hereditary deficiency. To direct …
