In utero delivery of oligodeoxynucleotides for gene correction.
In utero delivery of oligodeoxynucleotides for gene correction.
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在子宫内递送寡脱氧核苷酸以进行基因校正。
DOI：
10.1007/978-1-62703-761-7_26
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发表时间：
2014
期刊：
影响因子：
--
通讯作者：
Clemens,PaulaR
中科院分区：
文献类型：
--
作者：
Cai,Lingzhi;Koppanati,BhanuMunil;Bertoni,Carmen;Clemens,PaulaR
Gene correction is attractive for single gene mutation disorders, such as Duchenne muscular dystrophy (DMD). Themdxmouse 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 ofmdxmice 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 fetalmdxmouse in utero at embryonic day 16 and to test correction of dystrophin deficiency at different ages after birth.
