AAV6-huWT-PLN

AAV6-huWT-PLN is an investigational adeno-associated virus serotype 6 (AAV6) gene therapy designed for the treatment of phospholamban (PLN) deficiency, specifically dilated or arrhythmogenic cardiomyopathies caused by the monoallelic Arg14del (R14del) mutation in the PLN gene. The therapy utilizes an AAV6 vector to deliver and overexpress human wild-type phospholamban (huWT-PLN) in cardiomyocytes. This supplementary gene therapy approach aims to decrease the proportion of mutated PLN mRNA transcripts and proteins, thereby restoring normal calcium homeostasis and alleviating the pathogenic effects of the R14del mutation. It is being developed in collaboration between the Technical University of Munich and the University of Eastern Finland.

Other names

AAV6-WT-PLNAAV-6-WT-PLNAAV 6-WT-PLNAAV6-huWT-PLN gene therapyAAV-6-huWT-PLN gene therapyAAV 6-huWT-PLN gene therapy