In utero delivery of oligodeoxynucleotides for gene correction

Methods in Molecular Biology

Volumn 1114, Issue , 2014, Pages 399-411

  Cai, Lingzhi ^a   Koppanati, Bhanu Munil ^b   Bertoni, Carmen ^c   Clemens, Paula R ^b,d  

^a UNIVERSITY OF PITTSBURGH   (United States)

^b UNIVERSITY OF PITTSBURGH SCHOOL OF MEDICINE   (United States)

^c UNIVERSITY OF CALIFORNIA   (United States)

^d VETERANS AFFAIRS MEDICAL CENTER   (United States)

Author keywords

Duchenne; Gene correction; In utero; Muscular dystrophy; Oligodeoxynucleotide

Indexed keywords

COLLAGEN TYPE 4; DYSTROPHIN; GENOMIC DNA; MESSENGER RNA; OLIGODEOXYNUCLEOTIDE; OLIGODEOXYNUCLEOTIDE DERIVATIVE; SINGLE STRAND OLIGODEOXYNUCLEOTIDE; UNCLASSIFIED DRUG;

ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; ARTICLE; CONSENSUS SEQUENCE; CONTROLLED STUDY; DNA SEQUENCE; DONOR SITE; DUCHENNE MUSCULAR DYSTROPHY; EMBRYO; EXON; FEMALE; FETUS; FETUS DEVELOPMENT; GENE DELIVERY SYSTEM; GENE THERAPY; IMMUNOHISTOCHEMISTRY; IN UTERO GENE DELIVERY SYSTEM; INTRON; MOUSE; NONHUMAN; NONSENSE MUTATION; POINT MUTATION; POSTOPERATIVE ANALGESIA; PRIORITY JOURNAL; PROTEIN EXPRESSION; RNA EDITING; RNA SPLICING; SKELETAL MUSCLE; UTERUS HORN; X CHROMOSOME LINKED MUSCULAR DYSTROPHIC MOUSE; CARBOXY TERMINAL SEQUENCE; DNA MODIFICATION; GENE EDITING; GENE EXPRESSION; NONVIRAL GENE DELIVERY SYSTEM;

ANIMALS; DNA, SINGLE-STRANDED; DYSTROPHIN; GENE EXPRESSION; GENE TRANSFER TECHNIQUES; GENETIC THERAPY; MICE; MICE, INBRED MDX; MUSCLE, SKELETAL; MUSCULAR DYSTROPHY, DUCHENNE; OLIGODEOXYRIBONUCLEOTIDES;

MUS;

EID: 84922056967     PISSN: 10643745     EISSN: None     Source Type: Book Series    
DOI: 10.1007/978-1-62703-761-7_26     Document Type: Article

References (12)

1. Engel AG, Ozawa E (2004) Dystrophinopathies. In: Engel AG, Franzini-Armstrong C (eds) Myology, 3rd edn. McGraw-Hill, New York, pp 961-1025
2. Zellweger H, Antonik A (1975) Newborn screening for Duchenne muscular dystrophy. Pediatrics 55:30-34
3. Koppanati BM, Li J, Reay DP, Wang B, Daood M, Zheng H, Xiao X, Watchko JF, Clemens PR (2010) Improvement of the mdx mouse dystrophic phenotype by systemic in utero AAV8 delivery of a minidystrophin gene. Gene Ther 17:1355-1362
4. Reay DP, Bilbao R, Koppanati BM, Cai L, O'Day TL, Jiang Z, Zheng H, Watchko JF, Clemens PR (2008) Full-length dystrophin gene transfer to the mdx mouse in utero. Gene Ther 15:531-536
5. Bertoni C (2008) Clinical approaches in the treatment of Duchenne muscular dystrophy (DMD) using oligonucleotides. Front Biosci 13:517-527
6. Bartlett RJ, Stockinger S, Denis MM, Bartlett WT, Inverardi L, Le TT, Man N, Morris GE, Bogan DJ, Metcalf-Bogan J, Kornegay JN (2000) In vivo targeted repair of a point mutation in the canine dystrophin gene by a chimeric RNA/DNA oligonucleotide. Nat Biotechnol 18:615-622
7. Bertoni C, Rando TA (2002) Dystrophin gene repair in mdx muscle precursor cells in vitro and in vivo mediated by RNA-DNA chimeric oligonucleotides. Hum Gene Ther 13: 707-718
8. Bertoni C, Lau C, Rando TA (2003) Restoration of dystrophin expression in mdx muscle cells by chimeraplast-mediated exon skipping. Hum Mol Genet 12:1087-1099
9. Bertoni C, Morris GE, Rando TA (2005) Strand bias in oligonucleotide- mediated dystrophin gene editing. Hum Mol Genet 14:221-233
10. Bertoni C, Rustagi A, Rando TA (2009) Enhanced gene repair mediated by methyl-CpGmodifi ed single-stranded oligonucleotides. Nucleic Acids Res 37:7468-7482
11. Kayali R, Bury F, Ballard M, Bertoni C (2010) Site-directed gene repair of the dystrophin gene mediated by PNA-ssODNs. Hum Mol Genet 19:3266-3281
12. Rando TA, Disatnik MH, Zhou LZ (2000) Rescue of dystrophin expression in mdx mouse muscle by RNA/DNA oligonucleotides. Proc Natl Acad Sci USA 97: 5363-5368 and Skin Diseases