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Description
In conclusion, this pivotal work described by Kwiatkowski and colleagues represents the first phase 3 clinical study using lentiviral gene therapy to introduce functional copies of a modified form of the -globin gene ( A-T87Q-globin gene) into HSC isolated from patients with severe genetic forms of -thalassemia
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[DOI] [PubMed] [Google Scholar] 7.Treasure Island (FL): StatPearls Publishing
