The present invention provides methods of generating a recombinant AAV vector with reduced immunogenicity, comprising: providing eukaryotic cells with a nucleic acid comprising a sequence of interest that is flanked by AAV inverted terminal repeats, wherein the nucleic acid comprises CpG dinucleotide sites, wherein at least a portion of the CpG dinucleotide sites are methylated, wherein the eukaryotic cell expresses one or more other components necessary to achieve recombinant AAV biosynthesis, whereby the recombinant AAV vector is generated by the eukaryotic cell, wherein the generated recombinant AAV vector comprises nucleic acid wherein at least a portion of the CpG dinucleotide sites are methylated.

The present specification provides a drug that causes highly-efficient skipping of exon 51 in the human dystrophin gene. The present specification provides an antisense oligomer having an activity to induce skipping of exon 51 in the human dystrophin gene.

This invention relates to polynucleotides encoding mini-dystrophin proteins, viral vectors comprising the same, and methods of using the same for delivery of mini-dystrophin to a cell or a subject.

The present application provides materials and methods for treating a patient with Duchenne Muscular Dystrophy (DMD) both ex vivo and in vivo. In addition, the present application provides materials and methods for editing a dystrophin gene in a cell by genome editing.

The invention provide for recombinant AAV vectors comprising a miniaturized human micro-dystrophin gene and methods of using the recombinant vectors to reduce or prevent fibrosis in subjects suffering from muscular dystrophy.

Disclosed herein are transcription activator-like effector nuclease (TALEN)-related compositions and methods of using said TALENs for correcting mutant genes.

The present disclosure provides a method of modifying a mutant dystrophin gene in the genome of a cell. The present disclosure further provides compositions and kits for modifying a mutant dystrophin gene in the genome of a cell.

A method of treating fibrosis in a subject in need thereof is provided. The method comprising administering to the subject a polypeptide comprising a propeptide of lysyl oxidase (LOX), the polypeptide being devoid of LOX catalytic activity, thereby treating the fibrosis in the subject, wherein the method does not comprise administration of D-penicillamine. Also provided are polypeptide compositions for use in therapy.

There are provided compositions and methods for modulating stem cell division, in particular, division symmetry. It has been demonstrated that Wnt7a polypeptide fragments promoting symmetrical expansion of stem cells. The compositions and methods of the invention are useful, for example, in modulating stem cell division symmetry in vitro, ex vivo, and in vivo, in replenishing and expanding the stem cell pool, and in promoting the formation, maintenance, repair and regeneration of tissue.

The invention described herein provides gene therapy vectors, such as adeno-associated virus (AAV) vectors, that co-express a functional protein (such as a miniaturized human micro-dystrophin gene product) and one or more additional coding sequences for an RNAi sequence (siRNA, shRNA, miRNA), an antisense sequence, a guide sequence for a gene editing enzyme (such as an sgRNA for CRISPR/Cas9, or a crRNA for CRISPR/Casl2a), and/or a micro RNA, and methods of using the vectors to treat subjects suffering from a muscular dystrophy such as DMD/BMD.