• With very low systemic doses, we demonstrate that B-MSP-PMO restores high-level, uniform dystrophin protein expression in multiple peripheral muscle groups, yielding functional correction and improvement of the mdx dystrophic phenotype. (ox.ac.uk)
  • We also report that the frequency of genetic repair of the mdx mutation can be enhanced if RNAi is used to suppress expression of the recombinase inhibitor protein Msh2 in cultures containing myoblasts but not in those heavily enriched in myoblasts. (nih.gov)
  • The donor splice site in the DMD gene in mdx5cv mice causes a frameshift which results in a translation of truncated dystrophin protein (10 of the 79 exons). (nih.gov)
  • Here, we tested the hypothesis that SERCA2a over-expression may mitigate electrocardiography (ECG) abnormalities in old female mdx mice, a murine model of DMD cardiomyopathy. (biomedsearch.com)
  • Control mice were compared to mdx using a combination of in vivo and in vitro 1H-MRS methods to determine whether neural necrosis or developmental abnormalities occur in dystrophic brain. (ox.ac.uk)
  • Immunofluorescence staining and western blot confirmed SERCA2a over-expression in the mdx heart. (biomedsearch.com)
  • Using primary myoblasts from mdx5cv mice, single-stranded DNA oligonucleotides were designed to correct this DNA mutation. (nih.gov)
  • This method may also be useful for evaluating the therapeutic effects of drug administration, gene transfer, and myoblast transfer in the mdx mouse. (nih.gov)
  • E ) Genotyping results of mdx -C mice with mosaicism of 2 to 100% corrected Dmd gene. (nih.gov)
  • B ) Schematic of the 20-nucleotide sgRNA target sequence of the mdx allele (blue) and the PAM (red).The red arrowhead indicates the Cas9 cleavage site. (nih.gov)
  • NAA levels were normal in mdx brain compared to controls suggesting minor, if any, neuronal necrosis in dystrophic brain. (ox.ac.uk)
  • Home cage physical activity of mice, quantified from the force plate signal, was decreased by sActRIIB-Fc the whole 7-wk treatment in sedentary mice. (lu.se)
  • An in vivo and in vitro H-magnetic resonance spectroscopy study of mdx mouse brain: abnormal development or neural necrosis? (ox.ac.uk)
  • To understand what happens during the first weeks after sActRIIB-Fc administration, when mice are less active, healthy mice were injected with sActRIIB-Fc or PBS for 2 wk. (lu.se)