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ArtikelAdeno - Associated Virus Serotype 8 Efficiently Delivers Genes to Muscle and Heart  
Oleh: Wang, Zhong ; Tong, Zhu ; Chunping Qiao ; Liqiao, Zhou ; Bing, Wang ; Zhang, Jian ; Chunlian Chen ; Juan Li ; Xiao, Xiao
Jenis: Article from Journal - ilmiah internasional
Dalam koleksi: Nature Biotechnology: The Science and Business of Biotechnology vol. 23 no. 3 (Mar. 2005), page 321-328.
Topik: GENES; adeno associated virus; serotype 8; genes; muscle and heart
Ketersediaan
  • Perpustakaan Pusat (Semanggi)
    • Nomor Panggil: NN9.2
    • Non-tandon: 1 (dapat dipinjam: 0)
    • Tandon: tidak ada
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Isi artikelSystemic gene delivery into muscle has been a major challenge for muscular dystrophy gene therapy, with capillary blood vessels posing the principle barrier and limiting vector dissemination. Previous efforts to deliver genes into multiple muscles have relied on isolated vessel perfusion or pharmacological interventions to enforce broad vector distribution. We compared the efficiency of multiple adeno associated virus (AAV) vectors after a single injection via intraperitoneal or intravenous routes without additional intervention. We show that AAV8 is the most efficient vector for crossing the blood vessel barrier to attain systemic gene transfer in both skeletal and cardiac muscles of mice and hamsters. Serotypes such as AAV1 and AAV6 which demonstrate robust infection in skeletal muscle cells, were less effective in crossing the blood vessel barrier. Gene expression persisted in muscle and heart, but diminished in tissue undergoing rapid cell division, such as neonatal liver. This technology should prove useful fors muscle directed systemic gene therapy.
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