- High incidence of leukemia in large animals after stem cell gene therapy with a HOXB4-expressing retroviral vector
[作者:Zhang, XB; Beard, BC; Trobridge, GD; Wood, BL; Sale, GE; Sud, R; Humphries, RK; Kiem, HP,期刊:Journal of clinical investigation, 页码:1502-1510 , 文章类型: Article,,卷期:2008年118-4]
- Retroviral vector-mediated HSC gene therapy has been used to treat individuals with a number of life-threatening diseases. However, some patients with SCID-X1 developed retroviral vector-mediated leukemia after treatment...
- Mitotic spindle destabilization and genomic instability in Shwachman-Diamond syndrome
[作者:Austin, KM; Gupta, ML; Coats, SA; Tulpule, A; Mostoslavsky, G; Balazs, AB; Mulligan, RC; Daley, G; Pellman, D; Shimamura, A,期刊:Journal of clinical investigation, 页码:1511-1518 , 文章类型: Article,,卷期:2008年118-4]
- Deficiencies in the SBDS gene result in Shwachman-Diamond syndrome (SDS), an inherited bone marrow failure syndrome associated with leukemia predisposition. SBDS encodes a highly conserved protein previously implicated i...
- Premature termination codons in PRPF31 cause retinitis pigmentosa via haploinsufficiency due to nonsense-mediated mRNA decay
[作者:Frio, TR; Wade, NM; Ransijn, A; Berson, EL; Beckmann, JS; Rivolta, C,期刊:Journal of clinical investigation, 页码:1519-1531 , 文章类型: Article,,卷期:2008年118-4]
- Dominant mutations in the gene encoding the mRNA splicing factor PRPF31 cause retinitis pigmentosa, a hereditary form of retinal degeneration. Most of these mutations are characterized by DNA changes that lead to prematu...
- Reversal of axonal loss and disability in a mouse model of progressive multiple sclerosis
[作者:Basso, AS; Frenkel, D; Quintana, FJ; Costa-Pinto, FA; Petrovic-Stojkovic, S; Puckett, L; Monsonego, A; Bar-Shir, A; Engel, Y; Gozin, M; Weiner, HL,期刊:Journal of clinical investigation, 页码:1532-1543 , 文章类型: Article,,卷期:2008年118-4]
- Axonal degeneration is an important determinant of progressive neurological disability in multiple sclerosis (MS). Thus, therapeutic approaches promoting neuroprotection could aid the treatment of progressive MS. Here, w...
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