Disclosed herein are novel lipids and liposomal compositions prepared using such compounds and related methods of neutralizing or otherwise modifying such liposomal compositions. The lipids described herein are useful for example, as liposomal vehicles to facilitate the delivery of encapsulated polynucleotides to target cells and the subsequent transfection of such target cells. In certain embodiments, one or more of the compounds that comprise the liposomal delivery vehicle may be neutralized or further modified such that the properties of the liposomal delivery vehicle are modified.

Disclosed herein are novel lipids and liposomal compositions prepared using such compounds and related methods of neutralizing or otherwise modifying such liposomal compositions. The lipids described herein are useful for example, as liposomal vehicles to facilitate the delivery of encapsulated polynucleotides to target cells and the subsequent transfection of such target cells. In certain embodiments, one or more of the compounds that comprise the liposomal delivery vehicle may be neutralized or further modified such that the properties of the liposomal delivery vehicle are modified.

Disclosed herein is an agent that modulates a cis interaction between Repulsive Guidance Molecule A (RGMa) and Neogenin or lipid rafts. Modulation by the agent may include blocking the cis interaction between RGMa and Neogenin and/or disrupting lipid rafts. In turn, this promotes neuronal cell survival and axon growth and/or regeneration. Also disclosed herein is a method of treating a disease in a subject in need thereof. The method may include administering the agent to the subject. Further disclosed herein is a method of identifying an agent that modulates the cis interaction between RGMa and Neogenin.

The present invention provides methods and materials useful delivering liquids, including liquids comprising nucleic acid molecules into cells. In particular, the present invention provides methods for delivering saline solution, exogenous compositions, and isolated vectors to kidney cells, using the renal vein as a guide and under hydrodynamic pressure. The delivery methods and materials herein are useful to research, prognose, ameliorate symptoms of kidney injury, and treat kidney pathologies.

The present invention provides, among other things, methods of treating phenylketonuria (PKU), including administering to a subject in need of treatment a composition comprising an mRNA encoding phenylalanine hydroxylase (PAH) at an effective dose and an administration interval such that at least one symptom or feature of PKU is reduced in intensity, severity, or frequency or has delayed in onset. In some embodiments, the mRNA is encapsulated in a liposome comprising one or more cationic lipids, one or more non-cationic lipids, one or more cholesterol-based lipids and one or more PEG-modified lipids.

The present invention provides, among other things, methods of treating phenylketonuria (PKU), including administering to a subject in need of treatment a composition comprising an mRNA encoding phenylalanine hydroxylase (PAH) at an effective dose and an administration interval such that at least one symptom or feature of PKU is reduced in intensity, severity, or frequency or has delayed in onset. In some embodiments, the mRNA is encapsulated in a liposome comprising one or more cationic lipids, one or more non-cationic lipids, one or more cholesterol-based lipids and one or more PEG-modified lipids.

The present invention relates to extracellular vesicles derived from ginger (Zingiber officinale) and a use thereof, and provides: extracellular vesicles which are derived from ginger and have anticancer activity, a pharmaceutical composition and a health functional food composition which is for preventing or treating cancer and contains the extracellular vesicles as an active ingredient. The ginger-derived extracellular vesicles can penetrate into cancer cells, have excellent anticancer activity, such as inhibiting the proliferation, infiltration and migration of cancer cells, and thus may be provided as an effective anticancer composition.

The present invention relates to extracellular vesicles derived from ginger (Zingiber officinale) and a use thereof, and provides: extracellular vesicles which are derived from ginger and have anticancer activity, a pharmaceutical composition and a health functional food composition which is for preventing or treating cancer and contains the extracellular vesicles as an active ingredient. The ginger-derived extracellular vesicles can penetrate into cancer cells, have excellent anticancer activity, such as inhibiting the proliferation, infiltration and migration of cancer cells, and thus may be provided as an effective anticancer composition.

Pharmaceutical compositions including a mitochondria-targeting polypeptide and a therapeutic agent coupled to the mitochondria-targeting polypeptide are provided. Methods of treating a subject in need thereof including administering a therapeutically effective amount of the pharmaceutical composition to the subject are provided. The subject may have an aging-related condition, heart failure, diabetes, myocardial infarction, acquired mitochondrial disorder, and/or inherited mitochondrial disorder. Methods of delivering compound to a mitochondrion and of preparing a compound for delivery to a mitochondrion are also provided. Furthermore, biological tracers including a mitochondria-targeting polypeptide and a label coupled to the mitochondria-targeting polypeptide are provided.

Pharmaceutical compositions including a mitochondria-targeting polypeptide and a therapeutic agent coupled to the mitochondria-targeting polypeptide are provided. Methods of treating a subject in need thereof including administering a therapeutically effective amount of the pharmaceutical composition to the subject are provided. The subject may have an aging-related condition, heart failure, diabetes, myocardial infarction, acquired mitochondrial disorder, and/or inherited mitochondrial disorder. Methods of delivering compound to a mitochondrion and of preparing a compound for delivery to a mitochondrion are also provided. Furthermore, biological tracers including a mitochondria-targeting polypeptide and a label coupled to the mitochondria-targeting polypeptide are provided.