Gene transfer into hematopoietic stem cells as treatment for primary immunodeficiency diseases

Gene transfer into hematopoietic stem cells as treatment for primary immunodeficiency diseases.

Gene transfer into the hematopoietic stem cell has shown curative potential for a variety of hematological disorders. Primary immunodeficiency diseases have led to the way in this field of gene therapy as an example and a model. Clinical results from the past 15 years have shown that significant improvement and even cure can be achieved for diseases such as X-linked severe combined immunodefici...

I. Gene Transfer into Hematopoietic Stem Cells

This article reviews 1) the use of gene transfer methods to genetically manipulate hematopoietic stem cell targets, 2) recent advances in technology that are addressing problems that have prevented widespread successful translation of gene transfer approaches for the cure of disease, and 3) recent regulatory issues related to human gene therapy trials. In Section I, Dr. Nienhuis describes the u...

Differentiation of Mouse Embryonic Stem Cells into Hematopoietic Cells

Purpose: Differentiation of Mouse embryonicstem cells into Hematopoietic cells. Materials and Methods: In this study, we used EB formation system for Hematopoietic differentiation of mouse embryonic stem cell (Royan B1) in suspension culture. EBs cultured in medium with Hematopoietic inducer cytokines (SCF, TPO, GMCSF, IL3, Flt3 and EPO) .presence of hematopoietic differentiated cell assessed ...

Side effects of retroviral gene transfer into hematopoietic stem cells.

Recent conceptual and technical improvements have resulted in clinically meaningful levels of gene transfer into repopulating hematopoietic stem cells. At the same time, evidence is accumulating that gene therapy may induce several kinds of unexpected side effects, based on preclinical and clinical data. To assess the therapeutic potential of genetic interventions in hematopoietic cells, it wil...

European concerted action on human hematopoietic stem cell: gene transfer into hematopoietic stem cells.