A nanoscale drug carrier capable of crossing the blood-brain barrier. Said carrier can target brain lesions (brain tumors or other neurodegenerative diseases). The targeting drug carrier capable of crossing the blood-brain barrier comprises all-heavy-chain human ferritin or a functional fragments reconstituted structure or a mutant thereof. The manner for crossing the blood-brain barrier of the drug carrier is receptor-mediated transcytosis. The drug carrier provides an effective nanoscale drug carrier for the treatment of brain tumors or other neurodegenerative diseases.

Methods and formulations are provided for treating spinobulbar muscular atrophy in a subject in need thereof. By administering a therapeutically effective amount of a selective androgen receptor modulator or a small molecule such as 1-[2-(4-methylphenoxy)ethyl]-2-[(2-phenoxyethyl)sulfanyl]-1H-benzimidazole or a derivative, prodrug, or pharmaceutically acceptable salt thereof, one or more symptoms of spinobulbar muscular atrophy can be ameliorated. The effective amount can be effective to prevent or delay loss of body weight, a loss of mobility, and/or a loss of physical strength in the subject; to prevent or delay neurogenic atrophy and/or to prevent a loss of spinal cord motor neurons in the subject; to restore the frequency of type I myofibers to normal levels for a healthy subject; and/or to reverse testicular atrophy in the subject.

Methods and formulations are provided for treating spinobulbar muscular atrophy in a subject in need thereof. By administering a therapeutically effective amount of a selective androgen receptor modulator or a small molecule such as 1-[2-(4-methylphenoxy)ethyl]-2-[(2-phenoxyethyl)sulfanyl]-1H-benzimidazole or a derivative, prodrug, or pharmaceutically acceptable salt thereof, one or more symptoms of spinobulbar muscular atrophy can be ameliorated. The effective amount can be effective to prevent or delay loss of body weight, a loss of mobility, and/or a loss of physical strength in the subject; to prevent or delay neurogenic atrophy and/or to prevent a loss of spinal cord motor neurons in the subject; to restore the frequency of type I myofibers to normal levels for a healthy subject; and/or to reverse testicular atrophy in the subject.

An ALS treatment method includes administering to an ALS patient an inhibitor for a target A or a promoter for a target B. The target A is one or more genes selected from the genes in Table 1-1 or a protein encoded by the gene, and the inhibitor for the target A is a substance that inhibits expression of the gene, or a substance that inhibits a function of the protein encoded by the gene. The target B is one or more genes selected from the genes in Table 1-2 or a protein encoded by the gene, and the promoter for the target B is a substance that promotes the expression of the gene, or a substance that promotes a function of the protein encoded by the gene.

An ALS treatment method includes administering to an ALS patient an inhibitor for a target A or a promoter for a target B. The target A is one or more genes selected from the genes in Table 1-1 or a protein encoded by the gene, and the inhibitor for the target A is a substance that inhibits expression of the gene, or a substance that inhibits a function of the protein encoded by the gene. The target B is one or more genes selected from the genes in Table 1-2 or a protein encoded by the gene, and the promoter for the target B is a substance that promotes the expression of the gene, or a substance that promotes a function of the protein encoded by the gene.

Topical compositions comprising cannabinoids in combination with one or more humectants and one or more penetration enhancers are provided. Also provided are methods comprising applying a topical cannabinoid composition provided herein to the skin areas of a subject affected by inflammation and/or pain associated with musculoskeletal disease or condition and uses of a topical cannabinoid formulation provided herein for the treatment of inflammation and/or pain associated with musculoskeletal disease or condition in a subject.

A method of obtaining a pluripotent-like multipotent cell, including providing a cell of a first type which is not a pluripotent-like multipotent cell; contacting the cell of a first type with an agent capable of remodeling the chromatin and/or DNA of the cell; transiently increasing expression of at least one pluripotent gene regulator in the cell of a first type, to a level at which the at least one pluripotent gene regulator is capable of driving transformation of the cell of a first type into the pluripotent-like multipotent cell; and placing or maintaining the cell in a differentiation medium and maintaining intracellular levels of the at least one pluripotent gene regulator for a sufficient period of time to allow a stable pluripotent-like multipotent cell to be obtained; wherein the pluripotent-like multipotent cell so obtained does not exhibit teratoma formation in vivo.

The present disclosure relates to methods for increasing telomere length in one or more human cells and/or increasing genome stability of one or more human cells, for example by contacting one or more human cells with an agent that increases expression of Zscan4 in the one or more human cells. Methods of treating a subject in need of telomere lengthening, treating a disease or condition associated with a genomic and/or chromosome abnormality, of rejuvenating one or more human cells, of rejuvenating tissues or organs, and of rejuvenating a subject in need thereof, for example by contacting one or more human cells in the subject with an agent that increases expression of Zscan4, or by administering to a subject in need thereof, an agent that increases expression of Zscan4 are also provided.

It is intended to provide MSCs for transplantation that have an improved post-transplantation cell survival rate and engraftment rate and are highly safe with fewer adverse reactions, and a method for conveniently producing MSCs for transplantation having a high cell survival rate and engraftment rate. As means therefor, the present invention provides a stem cell for transplantation comprising an MSC capable of overexpressing IL-10.

The disclosure provides gene therapy vectors, such as adeno-associated virus (AAV), optimized for delivering a transgene to muscles. The optimized vectors contain constitutive or a muscle-specific promoter to deliver whole body or skeletal/heart muscle-specific transgene expression, respectively, in combination with a transgene cDNA to replace the gene mutation found in a muscle disease with a normal copy of the gene, an internal ribosomal entry site (IRES) to allow for production of a second protein from the same transcript, and a muscle growth factor, to build new muscle growth and strength. For example, the invention provides The disclosure provides gene therapy vectors, such as recombinant adeno-associated vims (rAAV), designed for treatment of GNE myopathy in which the rAAV expresses UDP-GlcNAc-epimerase/ManNAc-6 alone or in combination with a muscle growth factor or muscle transdifferentation factor. The provided AAV replace the mutated GNE gene expression while expressing proteins that stimulate muscle growth.