Maria-Veronica Muñoz-Rojas's research while affiliated with Cambridge and other places

... ASMD is a life-threatening disorder associated with significant morbidity and mortality, due to both central nervous system (CNS) and non-CNS manifestations [3][4][5] . Non-CNS manifestations include splenomegaly, impaired pulmonary function limiting diffusion capacity, and hepatomegaly leading to liver failure 6,7 . ASMD represents a significant burden on both patients and caregivers, affecting both physical and emotional well-being 2 . ...

... For example, ADEPT and ERT clinical trials have used cyclosporine to broadly suppress the immune system during dosing. 53,113,114 Another approach is to tolerize the immune system against the single novel agent rather than broadly suppressing it. For example, Selecta Biosciences developed rapamycin-encapsulated nanoparticles (ImmTOR) that when coadministered with immunogenic biologic therapies, induce tolerogenic dendritic cells and antigenspecific regulatory T-cells. ...

... ERT has also been able to stabilize the visceral manifestations of MPS IH, MPS II, MPS IIIA, MPS IV, and MPS VI, but its lack of ability to cross the blood-brain barrier (BBB) has prevented successful treatment by intravenous infusions of enzyme of the neurologic aspects of these diseases (Cox-Brinkman et al., 2007;Muenzer, 2004;Valayannopoulos & Wijburg, 2011). The delivery of these enzymes through an intrathecal route was investigated as a means of improving neurological outcomes (Auclair et al., 2010;Dickson et al., 2007Dickson et al., , 2008Dickson et al., , 2010Kakkis et al., 2004;Munoz-Rojas et al., 2008. Clinical trials of gene therapy-based approaches for MPS IH, II, and IIIA have shown some efficacy and encouraging results (de Castro, Del Toro, Giugliani, & Couce, 2021). ...