Some embodiments provided herein relate to methods, systems and kits for providing consistent intracoronary administration of a biologic to subjects having diverse coronary anatomies. In some embodiments, the biologic is an adeno-associated virus serotype 1 (AAV1) vector encoding sarcoplasmic/endoplasmic reticulum ATPase 2a (SERCA2a) protein.

The present disclosure provides proteins, nucleic acids, systems and methods for enhancing the synthesis of a protein.

Some embodiments provided herein relate to methods, systems and kits for providing consistent intracoronary administration of a biologic to subjects having diverse coronary anatomies. In some embodiments, the biologic is an adeno-associated virus serotype 1 (AAV1) vector encoding sarcoplasmic/endoplasmic reticulum ATPase 2a (SERCA2a) protein.

Compositions are described for direct protein delivery into multiple cell types in the mammalian inner ear. The compositions are used to deliver protein(s) (such as gene editing factors) editing of genetic mutations associated with deafness or associated disorders thereof. The delivery of genome editing proteins for gene editing and correction of genetic mutations protect or restore hearing from genetic deafness. Methods of treatment include the intracellular delivery of these molecules to a specific therapeutic target.

The present invention relates to a pharmaceutical composition for prevention and treatment of heart failure. Specifically, the present invention relates to a gene construct comprising a polynucleotide coding for SERCA2a protein or a fragment thereof and a polynucleotide coding for CCN5 protein or a fragment thereof, and a pharmaceutical composition comprising the same construct as an effective ingredient for preventing or treating heart failure. A pharmaceutical composition for prevention and treatment of heart failure according to the present invention is used in a method for co-expression of SERCA2a protein and CCN5 protein. Designed to exert a synergistic therapeutic effect through SERCA2a protein's function of preventing the loss of cardiomyocytes and increasing the activity of cardiomyocytes and CCN5 protein's function of suppressing the fibrosis of heart cells and tissues, the pharmaceutical composition can be useful for preventing or treating heart failure, which is a complex disorder induced by various etiological factors.

Some embodiments provided herein relate to methods, systems and kits for providing consistent intracoronary administration of a biologic to subjects having diverse coronary anatomies. In some embodiments, the biologic is an adeno-associated virus serotype 1 (AAV1) vector encoding sarcoplasmic/endoplasmic reticulum ATPase 2a (SERCA2a) protein.

The present invention includes compositions and methods for detecting, treating and preventing renal and pancreatic diseases and disorders.

A method of treating a glycosylation-defective protein associated disease or disorder in a subject, the method includes administering to the subject a therapeutically effective amount of a Sarco/ER ATPase (SERCA) inhibitor.

Aspects of the invention provide single stranded oligonucleotides for activating or enhancing expression of ATP2A2. Further aspects provide compositions and kits comprising single stranded oligonucleotides for activating or enhancing expression of ATP2A2. Methods for modulating expression of ATP2A2 using the single stranded oligonucleotides are also provided. Further aspects of the invention provide methods for selecting a candidate oligonucleotide for activating or enhancing expression of ATP2A2.

Compositions are described for direct protein delivery into multiple cell types in the mammalian inner ear. The compositions are used to deliver protein(s) (such as gene editing factors) editing of genetic mutations associated with deafness or associated disorders thereof. The delivery of genome editing proteins for gene editing and correction of genetic mutations protect or restore hearing from genetic deafness. Methods of treatment include the intracellular delivery of these molecules to a specific therapeutic target.