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The field of HSCT gene therapy has advanced from proof-of-concept studies to the treatment of humans with acquired and genetic diseases. Several clinical trials have resulted in life-saving successes, and the curative potential of genetically-modified HSCs is now a reality with the number of disease applications growing rapidly, including chronic granulomatous disease, Wiscott-Aldrich disease, Fanconi anemia, β-thalassemia, and sickle cell disease. Hemophilia A remains a prime candidate for HSCT gene therapy. Advancements in fVIII transgene design, viral vector engineering, and immunological conditioning have cured this disease in animal models and show promise for upcoming clinical trials. Additional studies continue to elucidate methods and technologies to identify and isolate HSCs and improve transduction of this important gene therapy target. In addition, overcoming the current limitations of fVIII expression, as well as reducing the risks of the gene transfer procedure, such as insertional mutagenesis, is possible. Further advancements in the field of HSCT gene therapy have included novel mechanisms to target site-specific gene insertions or corrections using engineered nucleases or PNA-complexes. Trough ongoing intensive research, the field of HSCT gene therapy is progressing towards safer, more efficient, and cost effective treatment options.

Engineered Hematopoietic Stem Cells as Therapeutics for Hemophilia A