A new species of leafhopper, Amphigonalia bispinosa, is described and is the replacement vector species for A. severini (DeLong) which had been previously misidentified. Distribution of both species ...
Figure 1: Insertion of a traffic light reporter into the AAVS1 locus of HEK293 cells and suppression of the NHEJ pathway. To assess whether a point mutation can be corrected in a tumor cell line, we ...
L444P KI mice were generated using a loxP replacement vector, resulting in the insertion of a point mutation of leucine to proline (L444P) in exon 10 of the Gba1 gene (Ginns et al., 2014). These Gba1 ...
Onasemnogene abeparvovec-brve is the first gene replacement therapy approved for SMA patients aged 2 years and older, expanding access beyond infants. Phase 3 trials demonstrated significant motor ...