Abstract: The present invention includes methods for treating a disease or disorder that is associated with aberrant or absent dystrophin, by upregulating a brain isoform of dystrophin (e.g. purkinje and/or cortical) in a subject in need thereof. The method comprises administering to the subject a composition that upregulates a brain isoform of dystrophin.

Abstract: Compositions and methods for treating FSHD and for identifying agents useful for the treatment of FSHD.

Abstract: Compositions and methods for treating FSHD and for identifying agents useful for the treatment of FSHD.

Abstract: The present invention features compositions and methods that enhance cell extravasation and/or muscle cell fusion, and methods for identifying genes that enhance or inhibit extravasation and muscle cell fusion. One aspect of the present disclosure provides an isolated cell lacking a gene of Table 1 or expressing a reduced level of a gene of Table 1. In some embodiments, the isolated cell has a CRISPR-edited genome or expresses an inhibitory nucleic acid molecule targeting a gene of Table 1. In some embodiments, this inhibitory nucleic acid molecule is an antisense oligonucleotide molecule, a short interfering RNA (siRNA) molecule, or an small hairpin (shRNA) molecule. Another aspect of the present disclosure provides an isolated cell comprising an expression vector encoding a gene of Table 1.