All participants reported normal or corrected-to-be-normal visual acuity and were right handed according to the Edinburgh Handedness Inventory [45].
Correcting Dlp levels by itself restored synaptic architecture, but both Dlp and Sdc had to be corrected to restore normal neurotransmission in dfmr1 null synapses.
In terms of inclusion criteria, all the participants had normal or corrected to normal vision, were native Spanish speakers and did not report cognitive or neurological disorders after a personal interview.
A total of 7 male and 10 female University of Glasgow students of normal or corrected to normal vision were paid to participate in the experiments.
Children with normal or corrected to normal vision were taken for the study.
All participants had normal or corrected to normal vision, were fluent English speakers, and gave informed written consent in accordance with the local research ethics committee.
Eighteen right-handed healthy native English speakers (5 male, 13 female, mean age = 22.5 years, and range 19 29), with normal or corrected to normal vision were screened using a comprehensive medical questionnaire and gave written informed consent before entering the MRI scanner.
Our RTM will trans-splice into both mutant and normal CD22 pre-mRNA, with the result that the mutant is corrected to encode a normal CD22 sequence, while the normal is trans-spliced into normal, although both forms will be tagged with the 3′ UTR sequence of the RTM.
As tamoxifen modulates molecular events downstream of myotubularin, not all pathogenic features were corrected to normal values as would be expected with gene-restoration therapies13.
Vision was normal or corrected to normal and we tested hearing in all subjects, which was within normal range for everyone (between 250 8000 Hz).
