Table 4.

Therapeutic interventions

	Human Barth Patients	TAZKD Mouse Model
Gene replacement	ModRNA (full-length human tafazzin cDNA) in Barth iPSC-derived cardiomyocytes AAV-mediated gene therapy tested in dermal fibroblasts	Cardiomyopathy reversible when DOX inducer is removed Des-TAZ vector gene therapy AAV9-TAZ gene therapy CL-Nanodisk as lipid replacement therapy
Pharmacological	Bezafibrate tested in lymphocytes and current clinical trials Resveratrol tested in lymphocytes Elamipretide clinical trials MitoTempo (antioxidant) tested in iPSC-derived cardiomyocytes Linoleic acid tested in fibroblasts and iPSC-derived cardiomyocytes	Bezafibrate ROS scavengers
Cardiovascular	Angiotensin converting enzyme inhibitors (ACEi), angiotensin receptor blockers (ARB), β-blockers Berlin EXCOR device Heart transplant Implantable cardioverter defibrillators
Skeletal myopathy	Exercise training	Endurance training
Neutropenia	Granulocyte colony stimulating factor (G-CSF)
References	13, 36, 65, 84, 85, 88, 92, 96, 99, 101	1, 32, 33, 71, 78, 88, 89, 92, 99

AAV, adeno-associated virus; CL, cardiolipin; DOX, doxycycline; iPSC, induced pluripotent stem cells; TAZ, tafazzin; TAZKD, knock down expression of tafazzin mRNA.