Gene Therapy
Reference19 articles.
1. Aartsma-Rus A, van Ommen GJ (2007) Antisense-mediated exon skipping: a versatile tool with therapeutic and research applications. RNA 13:1609–1624
2. Aiuti A, Cattaneo F, Galimberti S, Benninghoff U, Cassani B, Callegaro L, Scaramuzza S, Andolfi G, Mirolo M, Brigida I (2009) Gene therapy for immunodeficiency due to adenosine deaminase deficiency. N Engl J Med 360(5):447–458
3. Buchschacher GL (2001) Introduction to retroviruses and retroviral vectors. Somat Cell Mol Genet 26:1–11
4. Bushman FD (2007) Retroviral integration and human gene therapy. J Clin Invest 117(8):2083–2086
5. Cavazzana-Calvo M, Hacein-Bey S, de Saint Basile G, Gross F, Yvon E, Nusbaum P, Selz F, Hue C, Certain S, Casanova JL (2000) Gene therapy of human severe combined immunodeficiency (SCID)-X1 disease. Science 288(5466):669–672