在体内造血细胞的克隆追踪的目前的方法和未来的展望

Title:Current Approaches and Future Perspectives for In Vivo Clonal Tracking of Hematopoietic Cells

Volume: 16 Issue: 3

Author(s): Serena Scala, Lorena Leonardelli, Luca Biasco

Affiliation:

关键词: 克隆跟踪，造血干细胞，T细胞，整合位点分析。

摘要: 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.

Cite this article as:

Serena Scala, Lorena Leonardelli, Luca Biasco , Current Approaches and Future Perspectives for In Vivo Clonal Tracking of Hematopoietic Cells, Current Gene Therapy 2016; 16 (3) . https://dx.doi.org/10.2174/1566523216666160428104456