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Friday, July 4, 2014

In Utero Delivery of Oligodeoxynucleotides for Gene Correction

I In Utero Delivery of Oligodeoxynucleotides for Gene Correction
Lingzhi Cai, M.D., Ph.D. (et al.)
Lacy was contacted by her previous supervisor in the UPMC Department of Neurology where she had conducted genetic research on muscular dystrophy to first author a chapter on the above subject. That text has now been published by Springer as "Gene Correction Methods and Protocols." And you thought all she could do was cook chicken and cashews!

"Gene correction is attractive for single gene mutation disorders, such as Duchenne muscular dystrophy (DMD). The mdx mouse model of DMD is dystrophin deficient due to a premature chain-terminating point mutation in exon 23 of the dystrophin gene. Gene editing of genomic DNA using single-stranded oligodeoxynucleotides (ssODNs) offers the potential to change the DNA sequence to alter mRNA and protein expression in defined ways. When applied to fetal skeletal muscle of mdx mice in utero, this technology leads to restoration of dystrophin protein expression, thus providing a valid gene-based therapeutic application at the earliest developmental stage. Here, we describe detailed methods for gene editing using muscle delivery of ssODNs to the fetal mdx mouse in utero at embryonic day 16 and to test correction of dystrophin deficiency at different ages after birth."