User:Mhmd91/sandbox

AAV-based therapies was markedly utilized in the past few years for the treatment of several serious diseases such as Danon disease, a rare genetic disease caused by a mutation in LAMP2 gene resulting in downregulation of Lysosome-Associated Membrane Protein 2 (LAMP2) and thus deteriorated autophagy. One of the most demanding features of Danon disease is the rapidly progressive Hypertrophic cardiomyopathy which was the primary inducer of the ongoing extensive studies for possible treatments. The intravenous infusion of RP-A501 which is an AAV9 expressing LAMP2B was successful in the reversion of expression of LAMP2 in myocardial tissues leading to reduced thickness of the left ventrical wall and enhanced physical activity. Furthermore, RP-A501 was marked as an advanced regenerative medicine therapy by the US FDA, and its second phase clinical Trials commenced in the second quarter of 2023.