Abstract: An epigenetic silencer factor (ESF) comprising a transcription factor DNA-binding domain operably linked to at least one epigenetic effector domain, wherein the transcription factor is an oncogenic transcription factor or a cancer-associated transcription factor.

Abstract: The present disclosure provides novel methods for treating or preventing amyotrophic lateral sclerosis (ALS), methods for delaying the onset of neurological symptoms associated with ALS, increasing survival in subjects afflicted with ALS, and attenuating the decline of muscle strength associated with ALS in a subject in need thereof. The present disclosure also provides methods for treating or preventing ?-synucleinopathy or TDP-43 proteinopathy. The methods comprise administering to the subject an effective amount of a mitochondria-targeting peptidomimetic compound, such as (R)-2-amino-N-((S)-1-(((S)-5-amino-1-(3-benzyl-1,2,4-oxadiazol-5-yl)pentyl)amino)-3-(4-hydroxy-2,6-dimethylphenyl)-1-oxopropan-2-yl)-5-guanidinopentanamide, or a pharmaceutically acceptable salt, stereoisomer, tautomer, hydrate, and/or solvate thereof.

Abstract: A polynucleotide comprising a nucleotide sequence encoding methyl-CpG binding-protein 2 (MeCP2) operably linked to a strong promoter and a 3?-UTR, wherein the 3?-UTR is less than or equal to about 1000 bp in length.

Abstract: The present invention relates to a method for reprogramming a differentiated non neuronal cell into a dopaminergic neuron comprising the step of inducing the expression in the differentiated non neuronal cell of at least the protein encoded by the Mash1 human gene or orthologues thereof and the protein encoded by the Nurr1 human gene or orthologues thereof, expression vectors, reprogrammed dopaminergic neuron and uses thereof.