Current protocols for hematopoietic stem cell (HSC) gene therapy, involving the transplantation of ex vivo lentivirus vector-transduced HSCs into myeloablated recipients, are complex and not without risk for the patient. In vivo HSC gene therapy can be achieved by the direct modification of HSCs in the bone marrow after intraosseous injection of gene delivery vectors. A recently developed approach involves the mobilization of HSCs from the bone marrow into peripheral the blood circulation, intravenous vector injection, and re-engraftment of genetically modified HSCs in the bone marrow. We provide examples for in vivo HSC gene therapy and discuss advantages and disadvantages.
Key points
- •
Current protocols for hematopoietic stem gene therapy, involving the transplantation of ex vivo genetically modified hematopoietic stem cell (HSC), are complex and not without risk for the patient.
- •
HSCs in the bone marrow are intricately connected with the bone marrow stroma, which creates a physical barrier to transduction with intravenously injected gene transfer vectors.
- •
Intraosseal injection of viral vectors has been shown to result in in vivo transduction of HSCs in mice. It might be more feasible and efficient in large animals and humans.
- •
A new approach that involves the mobilization of HSCs from the bone marrow, their transduction in the periphery, and return to the bone marrow has shown first promising results.
Related posts:
Gene Editing
Therapeutic Gene Editing Safety and Specificity
Opening Marrow Niches in Patients Undergoing Autologous Hematopoietic Stem Cell Gene Therapy
Hematopoietic Gene Therapies for Metabolic and Neurologic Diseases
Gene Modified T Cell Therapies for Hematological Malignancies
Gene Therapy Approaches to Human Immunodeficiency Virus and Other Infectious Diseases
Stay updated, free articles. Join our Telegram channel
Full access? Get Clinical Tree
