Promoterless Gene Targeting Approach Combined to CRISPR/Cas9 Efficiently Corrects Hemophilia B Phenotype in Neonatal Mice.

Front Genome Ed · 2022 · PMC8962648 · PMID 35359664

Europe PMC DOI

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did not reach statistical significanceno p-value reported

In fact, the levels of plasmatic hFIX in the HDR-L group of animals reached ∼10,000 ng/ml at 10 months of age, while they were ∼6,000 ng/ml in the HDR-H group, although these differences did not reach statistical significance.

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