- Hematopoietic Stem Cell Gene Therapy with a Lentiviral Vector in X-Linked Adrenoleukodystrophy
[作者:Cartier, N; Hacein-Bey-Abina, S; Bartholomae, CC; Veres, G; Schmidt, M; Kutschera, I; Vidaud, M; Abel, U; Dal-Cortivo, L; Caccavelli, L; Mahlaoui, N; Kiermer, V; Mittelstaedt, D; Bellesme, C; Lahlou, N; Lefrere, F; Blanche, S; Audit, M; Payen, E; Leboulch,期刊:Science, 页码:818-823 , 文章类型: Article,,卷期:2009年326-5954]
- X-linked adrenoleukodystrophy (ALD) is a severe brain demyelinating disease in boys that is caused by a deficiency in ALD protein, an adenosine triphosphate-binding cassette transporter encoded by the ABCD1 gene. ALD pro...
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