Safety and efficacy of Igk-TATk-CDKL5 gene therapy in mosaic CDKL5 deficiency.

Additionally, Cdkl5 +/− female mice showed a deficit in locomotor habituation during the 15 min of the open field testing session, with a clear trend toward improvement (p = 0.07) following GT treatment.

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2A ), and a positive trend in the hippocampus ( Fig. 6 E and F) and cerebellum ( Supplementary Fig. 2B ).

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Although the comparison did not reach statistical significance due to low sample size and high intra-group variability in the treated group, a clear biological trend was observed.

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