The present disclosure is related to compositions and methods for the regulated and controlled expression of proteins.

The present disclosure is related to compositions and methods for the regulated and controlled expression of proteins.

The present invention provides an eco-friendly smart photosensitizer comprising a conjugate of hydroxypropyl methylcellulose and porfimer sodium photosensitizer, and a photo-stem cell therapy product comprising the photosensitizer. The photosensitizer of the present invention can be advantageously used in various fields including anticancer therapy, stem cell therapy, and the like, without side effects.

The present invention relates to RNAi constructs for reducing expression of the SLC30A8 gene. Methods of using such RNAi constructs to treat or prevent disease, such as pre-diabetes or diabetes are also described.

The present invention relates to RNAi constructs for reducing expression of the SLC30A8 gene. Methods of using such RNAi constructs to treat or prevent disease, such as pre-diabetes or diabetes are also described.

Methods of treating a disease characterized by mRNA instability or nonsense-mediated decay of an mRNA of a disease-associated gene in a subject by administering a pharmaceutical composition comprising at least one agent that decreases FTO expression, function or both are provided. Kits and pharmaceutical compositions comprising an agent that decreases FTO expression, function or both and a read-through promoting agent are also provided, as are methods of determining suitability of a subject to be treated with an agent that decreases FTO expression, function or both.

Methods of treating a disease characterized by mRNA instability or nonsense-mediated decay of an mRNA of a disease-associated gene in a subject by administering a pharmaceutical composition comprising at least one agent that decreases FTO expression, function or both are provided. Kits and pharmaceutical compositions comprising an agent that decreases FTO expression, function or both and a read-through promoting agent are also provided, as are methods of determining suitability of a subject to be treated with an agent that decreases FTO expression, function or both.

A method of making an erythroid cell comprising elevated levels of a target protein or polypeptide, the method comprising: a) provision of an erythroid progenitor which is able to express the target protein or polypeptide; b) expression of the target protein or polypeptide; and c) maturation of the erythroid progenitor into the erythroid cell; wherein during maturation of the erythroid progenitor into the erythroid cell, the target protein or polypeptide is configured and/or inhibited such that ubiquitination of the target protein or polypeptide is hindered or prevented. Erythroid cells, pharmaceutical compositions and methods of use related thereto, and a method of screening for proteins or polypeptides degraded by ubiquitination during maturation of an erythroid progenitor are also provided.

A method of making an erythroid cell comprising elevated levels of a target protein or polypeptide, the method comprising: a) provision of an erythroid progenitor which is able to express the target protein or polypeptide; b) expression of the target protein or polypeptide; and c) maturation of the erythroid progenitor into the erythroid cell; wherein during maturation of the erythroid progenitor into the erythroid cell, the target protein or polypeptide is configured and/or inhibited such that ubiquitination of the target protein or polypeptide is hindered or prevented. Erythroid cells, pharmaceutical compositions and methods of use related thereto, and a method of screening for proteins or polypeptides degraded by ubiquitination during maturation of an erythroid progenitor are also provided.

The disclosure features novel lipids and compositions involving the same. Lipid nanoparticles (e.g., empty LNPs or loaded LNPs) include a novel lipid as well as additional lipids such as phospholipids, structural lipids, and PEG lipids. Lipid nanoparticles (e.g., empty LNPs or loaded LNPs) further including therapeutic and/or prophylactics such as RNA are useful in the delivery of therapeutic and/or prophylactics to mammalian cells or organs to, for example, regulate polypeptide, protein, or gene expression.