Techniques regarding the transportation of molecular cargo across the BBB are provided. For example, one or more embodiments described herein can comprise a chemical compound to facilitate molecular encapsulation of the molecular cargo. The chemical compound can comprise a diblock copolymer having a molecular backbone comprising a polycarbonate structure and a polyethylene glycol structure. Also, the polycarbonate structure can be functionalized with boronic acid.

The present invention refers to a GPR101 inhibitor, antagonist or inverse agonist or inverse agonist for use in preventive and/or therapeutic treatment of diseases selected from the group consisting of acromegaly and gigantism and to methods for preventive and/or therapeutic treatment of diseases selected from the group consisting of acromegaly and gigantism wherein to a subject GPR101 inhibitor, antagonist or inverse agonist is administered. Further, the present invention provides a GPR101 agonist for use in preventive and/or therapeutic treatment of disorders selected from the group consisting of dwarfism, short stature, hypopituitarism and a disease of low levels of pituitary hormone secretion and to methods for preventive and/or therapeutic treatment of diseases selected from the group consisting of dwarfism, short stature, hypopituitarism and a disease of low levels of pituitary hormone secretion wherein to a subject GPR101 agonist is administered. The present invention also provides GHRH inhibitors, antagonists or inverse agonists and GH antagonists for use in the therapeutic treatment of X-linked acrogigantism (X-LAG syndrome). In addition, the present invention refers to a method of increasing body mass and/or body size of livestock comprising administering to livestock an effective amount of GPR101 agonist. Further, the present invention is directed to a non-human transgenic animal, comprising as expressed transgene a gene encoding GPR101 or overexpressing endogenous GPR101 gene.

The present invention refers to a GPR101 inhibitor, antagonist or inverse agonist or inverse agonist for use in preventive and/or therapeutic treatment of diseases selected from the group consisting of acromegaly and gigantism and to methods for preventive and/or therapeutic treatment of diseases selected from the group consisting of acromegaly and gigantism wherein to a subject GPR101 inhibitor, antagonist or inverse agonist is administered. Further, the present invention provides a GPR101 agonist for use in preventive and/or therapeutic treatment of disorders selected from the group consisting of dwarfism, short stature, hypopituitarism and a disease of low levels of pituitary hormone secretion and to methods for preventive and/or therapeutic treatment of diseases selected from the group consisting of dwarfism, short stature, hypopituitarism and a disease of low levels of pituitary hormone secretion wherein to a subject GPR101 agonist is administered. The present invention also provides GHRH inhibitors, antagonists or inverse agonists and GH antagonists for use in the therapeutic treatment of X-linked acrogigantism (X-LAG syndrome). In addition, the present invention refers to a method of increasing body mass and/or body size of livestock comprising administering to livestock an effective amount of GPR101 agonist. Further, the present invention is directed to a non-human transgenic animal, comprising as expressed transgene a gene encoding GPR101 or overexpressing endogenous GPR101 gene.

A method of treating a leukemia comprising administering to a subject in need thereof a therapeutically effective amount of a composition comprising a compound of Formula (I) and/or (II) having the structure: OR2 R R1O n OR2 R R1O n I II wherein: 10 ---- represents a single or a double bond; R is OH when C----R is C—R, and R is O when C----R is C═R; n is 1, 3, 5 or 7; and R1 and R2 are independently hydrogen or acetyl, and/or isomers, stereoisomers or solvates thereof and/or mixtures thereof.

##STR00001##

A method of treating a leukemia comprising administering to a subject in need thereof a therapeutically effective amount of a composition comprising a compound of Formula (I) and/or (II) having the structure: OR2 R R1O n OR2 R R1O n I II wherein: 10 ---- represents a single or a double bond; R is OH when C----R is C—R, and R is O when C----R is C═R; n is 1, 3, 5 or 7; and R1 and R2 are independently hydrogen or acetyl, and/or isomers, stereoisomers or solvates thereof and/or mixtures thereof.

##STR00001##

Compositions and kits including an agent that inhibits the interaction between Disabled-2 and mutant CFTR proteins, optionally in combination with a CFTR corrector, CFTR potentiator, CAL inhibitor, mucolytic, anti inflammatory agent or a combination thereof are provided as are methods for preventing or treating cystic fibrosis.

Compositions and kits including an agent that inhibits the interaction between Disabled-2 and mutant CFTR proteins, optionally in combination with a CFTR corrector, CFTR potentiator, CAL inhibitor, mucolytic, anti inflammatory agent or a combination thereof are provided as are methods for preventing or treating cystic fibrosis.

N-Substituted indenoisoquinoline compounds, and pharmaceutical formulations of N-substituted indenoisoquinoline compounds are described. Also described are processes for preparing N-substituted indenoisoquinoline compounds. Also described are methods for treating cancer in mammals using the described N-substituted indenoisoquinoline compounds or pharmaceutical formulations thereof.

N-Substituted indenoisoquinoline compounds, and pharmaceutical formulations of N-substituted indenoisoquinoline compounds are described. Also described are processes for preparing N-substituted indenoisoquinoline compounds. Also described are methods for treating cancer in mammals using the described N-substituted indenoisoquinoline compounds or pharmaceutical formulations thereof.

In various implementations, berberine metabolites, such as dihydroberberine and/or tetrahydroberberine, may be administered to manage blood glucose levels, increase ketone levels (e.g., blood concentration of ketones), and/or for therapeutic purposes in humans. The administration of a pharmaceutically effective amount of berberine metabolites, such as dihydroberberine, may reduce fasting blood glucose levels, improve glucose tolerance, and/or improve blood ketone response. In some implementations, berberine metabolites may be administered with one or more other compounds.