… Duchenne muscular dystrophy (DMD) following intramuscular and intravenous AAV-CRISPR
therapy. … the AAV8 CMV.micro-dystrophin vector at the dose of 1 × 10 12 vg/muscle to two 8-…

… Duchenne muscular dystrophy gene therapy efficacy using AAV9 in mdx mice; it increased
micro-dystrophin gene … In summary, during AAV-mediated DMD gene therapy, pre-treatment …

… A third approach is to combine DWORF overexpression with microdystrophin gene therapy.
Systemic AAV microdystrophin gene therapy is currently being tested in patients with DMD. …

… gene modelled on the micro-dystrophin gene is a compelling surrogate for dystrophin in
preventing pathology in the dog model of the disease [21]. In the context of gene therapy, AAV-…

… gene therapy for the permanent correction of DMD. Currently, the most widely used delivery
vector for gene therapy is recombinant AAV… after systemic AAV delivery, the skeletal muscles …

… of therapeutic approaches for Duchenne muscular dystrophy. Specifically, we focus on
strategies based on delivery of micro-dystrophin and micro-utrophin genes … on gene therapy and …

… Further results, supporting efficacy of AAV-mediated gene … a CK8 muscle-specific promoter
to express a micro-dystrophin … with the treatment, two systemic inflammatory responses and …

… Systemic human mini-dystrophin gene transfer has been … that is regulated by IRES to
monitor the efficiency of the AAV … A five-repeat micro-dystrophin gene ameliorated dystrophic …

… gene therapy with an AAV-delivered shortened version of dystrophin (µDys) for Duchenne
muscular dystrophy … in the context of systemic administration, as intramuscular AAV delivery is …

… (AAV) was the commonly used vector in DMD gene therapy,21 … that of fiverepeat micro-dystrophin
sequence constructed by … In conclusion, the LV could be applied for the gene therapy …