Exploiting microRNA-regulation to improve the reach and safety of hematopoietic stem cell manipulation

Applicant Dr. Bernhard Gentner

Subject Area Hematology, Oncology

Term from 2006 to 2010

Project identifier Deutsche Forschungsgemeinschaft (DFG) - Project number 30450217

Final Report Year 2008

Final Report Abstract

No abstract available

American Society for Gene Therapy (ASGT) 2007 in Seattle, WA: MicroRNA - regulated lentiviral vectors drive robust tissue -, lineage - and developmental stage - specific gene expression in hematopoietic, neural and embryonic stem cells
Gentner B, Brown BD, Colleoni S, Gritti A, Galli C, Naldini L
Endogenous microRNA can be broadly exploited to regulate transgene expression according to tissue, lineage and differentiation state. Nat Biotechnol 25: 1457 - 1467, 2007
Brown BD, Gentner B, Cantore A, Colleoni S, Amendola M, Zingale A, Baccarini A, Lazzari G, Galli C, Naldini L
IVth Conference on Stem Cell Gene Therapy (2007) in Thessaloniki, Greece: Exploiting MicroRNA Expression Profiles for Lineage - and Differentiation State - SpecificTransgene Expression in Hematopoietic, Neural and Embryonic Stem Cells
American Society for Gene Therapy (ASGT) 2008 in Boston, MA: Identification of hematopoietic stem cell (HSC) specific microRNAs and design of lentiviral vectors that detarget transgene expression from HSC while driving it efficiently in differentiated progeny
B. Gentner, E. Lechman, G. Schira, J.E. Dick, L. Naldini
American Society for Gene Therapy (ASGT) 2008 in Boston, MA: Stable knock‐down of endogenous microRNA in vivo by overexpressing miRNA target sequences by lentiviral vectors
B. Gentner, G. Schira, A. Cantore, M. Amendola, L. Naldini