Morpholino oligomers have great therapeutic potential for treatment of a broad range of
human diseases, including viral, bacterial, age-related, and genetic diseases, but they suffer
from poor systemic delivery into cells. Although various approaches have been undertaken
to address the delivery problem, it remains as the major barrier of morpholinos to be used as
effective therapeutics. This slow development is in part due to the cost of materials and the
animal models used for screening the efficacy and safety of those delivery approaches. The …

Antisense morpholino oligomers are synthetic macromolecules can modify gene expression.
While these compounds have great potential to treat a broad range of human diseases, they
suffer from poor cellular delivery. Conjugating cell-penetrating peptides onto these
morpholinos is one option of addressing delivery, however development remains slow due
to the cost of the animal models used to assess in vivo delivery and safety of peptide-
conjugated morpholinos. We have produced a novel transgenic zebrafish model that …