Abstract: This disclosure features chemical entities (e.g., a compound or a pharmaceutically acceptable salt, and/or hydrate, and/or cocrystal, and/or drug combination and/or stereoisomers (e.g., enantiomers and diastereomers) of the compound) that inhibit (e.g., antagonize) Carnosine Dipeptidase 2 (CNDP2). Said chemical entities are useful, e.g., for treating a condition, disease or disorder in which increased (e.g., excessive) CNDP2 activation contributes to the pathology and/or symptoms and/or progression of the condition, disease or disorder (e.g., cancer) in a subject (e.g., a human). This disclosure also features compositions containing the same as well as methods of using and making the same.

Abstract: Compositions and methods are provided for the inhibition, treatment and/or prevention of fragile X syndrome and related disorders.

Abstract: CRISPR/Cas9-mediated genome editing holds clinical potential for treating genetic diseases, such as spinocerebellar ataxia type 2, which is caused by mutations in the ATXN2 gene. Here, compositions and methods for the treatment of spinocerebellar taxia type 2 are provided. In some embodiments, a composition providing a vector encoding a guide RNA of the disclosure and a vector encoding a Cas9 protein or nuclease domain thereof of the disclosure are provided for use in the treatment of spinocerebellar ataxia type 2.

Abstract: Compositions and methods are provided for the inhibition, treatment and/or prevention of fragile X syndrome and related disorders.