Ontology highlight
ABSTRACT:
SUBMITTER: Sullivan SK
PROVIDER: S-EPMC3907760 | biostudies-literature | 2014 Jan
REPOSITORIES: biostudies-literature
Sullivan Spencer K SK Mills Jason A JA Koukouritaki Sevasti B SB Vo Karen K KK Lyde Randolph B RB Paluru Prasuna P Zhao Guoha G Zhai Li L Sullivan Lisa M LM Wang Yuhuan Y Kishore Siddharth S Gharaibeh Eyad Z EZ Lambert Michele P MP Wilcox David A DA French Deborah L DL Poncz Mortimer M Gadue Paul P
Blood 20131213 5
Megakaryocyte-specific transgene expression in patient-derived induced pluripotent stem cells (iPSCs) offers a new approach to study and potentially treat disorders affecting megakaryocytes and platelets. By using a Gp1ba promoter, we developed a strategy for achieving a high level of protein expression in human megakaryocytes. The feasibility of this approach was demonstrated in iPSCs derived from two patients with Glanzmann thrombasthenia (GT), an inherited platelet disorder caused by mutation ...[more]