miRNA-embedded shRNAs for Lineage-specific BCL11A Knockdown and Hemoglobin F Induction

Mol Ther. 2015 Sep;23(9):1465-74. doi: 10.1038/mt.2015.113. Epub 2015 Jun 17.

Abstract

RNA interference (RNAi) technology using short hairpin RNAs (shRNAs) expressed via RNA polymerase (pol) III promoters has been widely exploited to modulate gene expression in a variety of mammalian cell types. For certain applications, such as lineage-specific knockdown, embedding targeting sequences into pol II-driven microRNA (miRNA) architecture is required. Here, using the potential therapeutic target BCL11A, we demonstrate that pol III-driven shRNAs lead to significantly increased knockdown but also increased cytotoxcity in comparison to pol II-driven miRNA adapted shRNAs (shRNA(miR)) in multiple hematopoietic cell lines. We show that the two expression systems yield mature guide strand sequences that differ by a 4 bp shift. This results in alternate seed sequences and consequently influences the efficacy of target gene knockdown. Incorporating a corresponding 4 bp shift into the guide strand of shRNA(miR)s resulted in improved knockdown efficiency of BCL11A. This was associated with a significant de-repression of the hemoglobin target of BCL11A, human γ-globin or the murine homolog Hbb-y. Our results suggest the requirement for optimization of shRNA sequences upon incorporation into a miRNA backbone. These findings have important implications in future design of shRNA(miR)s for RNAi-based therapy in hemoglobinopathies and other diseases requiring lineage-specific expression of gene silencing sequences.

Publication types

  • Research Support, N.I.H., Extramural
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Animals
  • Base Sequence
  • Carrier Proteins
  • Cell Line
  • Cell Lineage / genetics*
  • Erythroid Cells / cytology
  • Erythroid Cells / metabolism
  • Fetal Hemoglobin / biosynthesis*
  • Fetal Hemoglobin / genetics*
  • Gene Expression
  • Gene Knockdown Techniques
  • Gene Order
  • Gene Silencing*
  • Genetic Vectors / genetics
  • Humans
  • Mice
  • MicroRNAs / chemistry
  • MicroRNAs / genetics*
  • Nuclear Proteins
  • Nucleic Acid Conformation
  • Promoter Regions, Genetic
  • RNA Interference*
  • RNA, Small Interfering / chemistry
  • RNA, Small Interfering / genetics*
  • Repressor Proteins
  • Retroviridae / genetics
  • Transduction, Genetic
  • gamma-Globins / biosynthesis
  • gamma-Globins / genetics

Substances

  • BCL11A protein, human
  • Carrier Proteins
  • MicroRNAs
  • Nuclear Proteins
  • RNA, Small Interfering
  • Repressor Proteins
  • gamma-Globins
  • Fetal Hemoglobin