Sickle Cell Disease

NaPro's gene editing demonstrates gene alteration in human cells

Published in Gene Therapy Weekly, July 3rd, 2003

NaPro BioTherapeutics, Inc., (NPRO) announced the development of a clinically applicable protocol to achieve specific gene alteration in human hematopoietic stem/progenitor cells.

These results, presented at the American Society of Gene Therapy (ASGT) annual meeting, achieve a milestone in the development of its procedures for potential treatment of sickle cell disease. Specifically, these data demonstrate successful alteration of a single base pair in the beta-globin gene from normal to sickle.

NaPro is now investigating the use of the same approach to correct mutant sickle cells to a normal state with the ultimate hope of providing effective...

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Source: Gene Therapy Weekly (2003-07-03)

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