C. Kupatt

Gene Therapy for Cardiomyopathy associated with Duchenne Muscular Dystrophy in a Pig Model

This study shows that AAV1.SERCA2a gene therapy improves heart function and reduces arrhythmia in a pig model of Duchenne muscular dystrophy.

Increased pulmonary monocyte infiltration and attenuated phagocytosis defines perinatal dysfunction of innate immunity in Cystic Fibrosis

The paper identifies increased monocyte infiltration and reduced phagocytosis in CF lungs at birth, suggesting innate immune dysfunction and the need for early treatment.

Immature human engineered heart tissues engraft in a guinea pig chronic injury model

This study shows that transplanting immature engineered heart tissue patches improves cardiomyocyte engraftment in a chronic injury guinea pig model.

A scalable, clinically severe pig model for Duchenne muscular dystrophy

This paper develops a scalable pig model for Duchenne muscular dystrophy by breeding DMD carriers, enabling large cohorts for disease mechanism studies and therapeutic testing.

Systemic gene therapy with thymosin β4 alleviates glomerular injury in mice

This study shows that systemic TB4 gene therapy prevents podocyte injury and albuminuria in mice with Adriamycin-induced glomerular disease.

Primate heart regeneration via migration and fibroblast repulsion by human heart progenitors

This study shows human heart progenitors can regenerate primate hearts by migrating to injury sites, repelling fibroblasts, and replacing damaged tissue through CXCL12/CXCR4 and SLIT2/ROBO1 signaling.

Agrin promotes coordinated therapeutic processes leading to improved cardiac repair in pigs

This study shows that Agrin improves cardiac repair in pigs after myocardial infarction by reducing injury and enhancing function.