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Updated: Feb 13, 2026

CRISPR/Cas9 Gene Editing of Hematopoietic Stem and Progenitor Cells for Gene Therapy Applications
Published on: August 9, 2022
[Gene therapy of blood disorders]
1Zakładu Immunologii CSK WAM w Warszawie.
Abstract:
In the article the basic knowledge concerning genes and genetic disorders necessary to understand the methods utilized in contemporary attempts of gene therapy has been reviewed. Then, a classification of gene therapy methods was presented and approaches to somatic gene therapy were discussed with particular attention to the utilization of retroviral vectors and packaging cell lines. Other vector systems such as adenovirus, vaccinia virus, and herpes virus derived have also been mentioned as well as antisense strategy. Reviewed applications concerned mainly inborn gene errors, but strategy of gene-directed, gene-forced suicide of leukemic cells has also been mentioned.
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