Current approaches and future perspectives for in vivo clonal tracking of hematopoietic cells

Serena Scala, Lorella Leonardelli, Luca Biasco

Research output: Contribution to journalArticle


Over the past years, clonal tracking has gained the center stage as a unique technology capable to unveil population dynamics and hierarchical relationships in vivo. We here highlighted the main open questions related to the in vivo clonal behavior of hematopoietic cells with a particular focus on hematopoietic stem and progenitor cells and T cells as main targets of cell- and gene-therapies. We walked through the current methods applied for tracing in vivo dynamics and functions of hematopoietic cells in animal models and we described the results of early studies conducted on humans. We specifically focused our attention on the recent use of retroviral/lentiviral vector Integration Site (IS) analyses to follow stably marked clones and their progeny in vivo. We showed how this molecular tracking method can be successfully employed in human studies to unveil the clonal behavior of hematopoietic cells, describing pioneering works conducted on samples from gene therapy treated patients. Clonal tracking through IS identification still comes with a complex wet-experimental protocol and technical/analytical constraints. In this regard, we reviewed the features of the available computational tools for the identification and quantification of ISs and we highlighted the potential future improvements of IS-based tracking, as this technology is becoming a major source of information on in vivo fate and survival of engineered cells in humans.

Original languageEnglish
Pages (from-to)184-193
Number of pages10
JournalCurrent Gene Therapy
Issue number3
Publication statusPublished - Jun 1 2016


  • Clonal tracking
  • Hematopoietic stem cells
  • Integration site analysis
  • T cells

ASJC Scopus subject areas

  • Genetics
  • Molecular Biology
  • Molecular Medicine
  • Genetics(clinical)
  • Drug Discovery

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