ALPL-1 was never assigned as a target for antibody-based therapy, probably because it is found in different healthy tissues and its cellular location remains controversial being reported both at the plasma membrane and in the cytosol29. Membrane ALPL expression has previously been described in hea...
There are also a number of metabolic disorders that have enzyme-replacement therapy (ERT) treatments such as Gaucher's disease, Fabry disease, and Wolman disease (10). Although there are many successful protein therapeutics on the market, their success relies on both their clinical utility and ...
According to the minimal criteria proposed by the International Society for Cellular Therapy [21], human MSCs are in vitro defined by the following proper- ties: spindle-shape fibroblast-like morphology, the capacity to grow in adhesion on plastic surfaces, and to expand under appropriate ...
This gene therapy involves the intramuscular delivery of a recombinant AAV1 that facilitates muscle-specific expression of LPL. It has been found to be both feasible and well tolerated, showing promising clinical outcomes in LPLD patients [94], [95]. A significant obstacle, however, is the ...
, which demonstrate an evolutionary conserved role of this enzyme, we consider zebrafish as a promising species for modeling HPP in order to discover new potential therapy strategies in the long-term. Similar content being viewed by others