Due to their accessibility and role in a range of inherited and acquired diseases, genome editing in hematopoietic cells is a rapidly expanding field. Successful genome editing has been performed in many hematopoietic cell lineages from hematopoietic stem cells (HSCs) to terminally differentiated T cells. Different techniques and technologies have been employed to perform gene editing of different hematopoietic cell types. As our ability to successfully perform gene editing of hematopoietic cells has improved, so too has our knowledge of potential adverse on-target and off-target toxicities and how these differ between cell types and editing techniques.
Pre-clinical and clinical data of gene engineered hematopoietic cell approaches have demonstrated revolutionary therapeutic approaches for diseases such as the haemoglobinopathies, inborn errors of immunity and in cellular therapy approaches for cancer. To date, most genome editing is performed ex vivo and the technology to perform gene editing of hematopoietic cells to generate clinical products is a rapidly developing area. Recently proof-of-concept of in vivo editing of hematopoietic cells has been achieved which will pave the way for a new generation of hematopoietic cell therapies.
In this Research Topic we invite submissions that explore the current technologies, applications, recent developments, and future advances in genome editing of hematopoietic cells. Through this series we hope to explore the strategies for targeting different cell types, compare the different technologies and understand the synergies involved in targeting the cells of the blood to treat human disease.
Keywords:
in vivo editing, Haematopoietic Cells, gene therapy, blood cells, human diseases, CRISPR/Cas9
Important Note:
All contributions to this Research Topic must be within the scope of the section and journal to which they are submitted, as defined in their mission statements. Frontiers reserves the right to guide an out-of-scope manuscript to a more suitable section or journal at any stage of peer review.
Due to their accessibility and role in a range of inherited and acquired diseases, genome editing in hematopoietic cells is a rapidly expanding field. Successful genome editing has been performed in many hematopoietic cell lineages from hematopoietic stem cells (HSCs) to terminally differentiated T cells. Different techniques and technologies have been employed to perform gene editing of different hematopoietic cell types. As our ability to successfully perform gene editing of hematopoietic cells has improved, so too has our knowledge of potential adverse on-target and off-target toxicities and how these differ between cell types and editing techniques.
Pre-clinical and clinical data of gene engineered hematopoietic cell approaches have demonstrated revolutionary therapeutic approaches for diseases such as the haemoglobinopathies, inborn errors of immunity and in cellular therapy approaches for cancer. To date, most genome editing is performed ex vivo and the technology to perform gene editing of hematopoietic cells to generate clinical products is a rapidly developing area. Recently proof-of-concept of in vivo editing of hematopoietic cells has been achieved which will pave the way for a new generation of hematopoietic cell therapies.
In this Research Topic we invite submissions that explore the current technologies, applications, recent developments, and future advances in genome editing of hematopoietic cells. Through this series we hope to explore the strategies for targeting different cell types, compare the different technologies and understand the synergies involved in targeting the cells of the blood to treat human disease.
Keywords:
in vivo editing, Haematopoietic Cells, gene therapy, blood cells, human diseases, CRISPR/Cas9
Important Note:
All contributions to this Research Topic must be within the scope of the section and journal to which they are submitted, as defined in their mission statements. Frontiers reserves the right to guide an out-of-scope manuscript to a more suitable section or journal at any stage of peer review.