Provided herein, in some aspects, are compositions and methods for treating Barth syndrome (BTHS) using human tafazzin gene therapy or enzyme replacement therapy. The present disclosure, in some aspects, provides compositions and methods (e.g., gene therapy or enzyme replacement therapy) for treating Barth syndrome (BTHS). It was demonstrated herein that certain human Tafazzin (hTAZ) isoforms and the full length protein, as well as nucleic acids encoding them, are effective in treating BTHS.

An isolated antigen binding protein, which includes at least one CDR of a heavy chain variable region and at least one CDR of a light chain variable region and a method to encode an isolated nucleic acid molecule. A vector with the nucleic acid molecule. A cell with the nucleic acid molecule. A pharmaceutical composition with the isolated antigen binding protein. A method for preventing, alleviating or treating a CS-related disease or disorder. A method for detecting C5 in a sample.

The present disclosure relates to, inter alia, methods of treating mixed dyslipidemia with ethyl eicosapentaenoate.

Described herein is a discovery Platform Technology for analyzing a drug-induced toxicity condition, such as cardiotoxicity via model building.

A method for treating a subject suffering from non-compaction cardiomyopathy (NCC), by administering to the subject suffering from NCC a pharmaceutical composition having a therapeutically effective amount of a EZH2 downregulator including the statin.

A method for treating a subject suffering from non-compaction cardiomyopathy (NCC), by administering to the subject suffering from NCC a pharmaceutical composition having a therapeutically effective amount of a EZH2 downregulator including the statin.

The present invention relates to novel steroidal compounds of formula (I), process for preparation of the same and composition comprising these compounds. ##STR00001##

The present invention relates to novel steroidal compounds of formula (I), process for preparation of the same and composition comprising these compounds. ##STR00001##

Described herein are compositions and methods related to use of cardiosphere-derived cells and their extracellular vesicles, such as exosomes and microvesicles, for achieving anti-aging and rejuvenation. This includes discoveries for effects on heart structure, function, gene expression, and systemic parameters. For animal studies, intra-cardiac injections of neonatal rat CDCs was compared to in old and young rats including evaluation of blood, echocardiographic, haemodynamic and treadmill stress tests. For in vitro studies, human heart progenitors from older donors, or cardiomyocytes from aged rats were exposed to human CDCs or cardiosphere derived cell (CDC) derived exosomes (CDC-XO) from pediatric donors. CDCs and CDC-XOs were capable of effectuating youthful patterns of gene expression in the hearts of old, along with a variant of physiological and function benefits, including elongation of telomere length. Together, these results indicate capacity of CDCs and CDC-XO to ward off the effects of aging through rejuvenation.

Described herein are compositions and methods related to use of cardiosphere-derived cells and their extracellular vesicles, such as exosomes and microvesicles, for achieving anti-aging and rejuvenation. This includes discoveries for effects on heart structure, function, gene expression, and systemic parameters. For animal studies, intra-cardiac injections of neonatal rat CDCs was compared to in old and young rats including evaluation of blood, echocardiographic, haemodynamic and treadmill stress tests. For in vitro studies, human heart progenitors from older donors, or cardiomyocytes from aged rats were exposed to human CDCs or cardiosphere derived cell (CDC) derived exosomes (CDC-XO) from pediatric donors. CDCs and CDC-XOs were capable of effectuating youthful patterns of gene expression in the hearts of old, along with a variant of physiological and function benefits, including elongation of telomere length. Together, these results indicate capacity of CDCs and CDC-XO to ward off the effects of aging through rejuvenation.