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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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Molecular Composition and Chemical Data The molecular composition of these research compounds show fundamental differences that affect their biological activity and research applications