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“All three patients showed stable engraftment of WASP-expressing cells and improvements in platelet counts, immune functions, and clinical score,” Dr. Naldini et al. report. They add vector integration analyses indicated that gene-corrected HSPCs resulted in highly polyclonal and multilineage hematopoiesis. Importantly, the researchers note that “lentiviral gene therapy did not induce selection of integrations near oncogenes,” and that they did not observe aberrant clonal expansion 20 to 32 months post-therapy.
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"Elvin" (2019-05-27)
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“All three patients showed stable engraftment of WASP-expressing cells and improvements in platelet counts, immune functions, and clinical score,” Dr. Naldini et al. report. They add vector integration analyses indicated that gene-corrected HSPCs resulted in highly polyclonal and multilineage hematopoiesis. Importantly, the researchers note that “lentiviral gene therapy did not induce selection of integrations near oncogenes,” and that they did not observe aberrant clonal expansion 20 to 32 months post-therapy.
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