The Treatment of Genetic Disease
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[1] S. Ellard,et al. Treatment of genetic disease , 2007 .
[2] Edward A Copelan,et al. Hematopoietic stem-cell transplantation. , 2006, The New England journal of medicine.
[3] Ronald G. Crystal,et al. Genetic medicines: treatment strategies for hereditary disorders , 2006, Nature Reviews Genetics.
[4] J. Rasko,et al. Successful transduction of liver in hemophilia by AAV-Factor IX and limitations imposed by the host immune response , 2006, Nature Medicine.
[5] W. Hauswirth,et al. Long-term restoration of rod and cone vision by single dose rAAV-mediated gene transfer to the retina in a canine model of childhood blindness. , 2005, Molecular therapy : the journal of the American Society of Gene Therapy.
[6] K. Weinberg,et al. Long-term efficacy of enzyme replacement therapy for adenosine deaminase (ADA)-deficient severe combined immunodeficiency (SCID). , 2005, Clinical immunology.
[7] K. Weinberg. Early use of drastic therapy. , 2005, New England Journal of Medicine.
[8] S. Schrier,et al. New strategies in the treatment of the thalassemias. , 2005, Annual review of medicine.
[9] A. Fischer,et al. Gene therapy for severe combined immunodeficiency. , 2005, Annual review of medicine.
[10] Christine Kinnon,et al. Mutations in TNFRSF13B Encoding TACI Are Associated With Common Variable Immunodeficiency in Humans , 2006, Pediatrics.
[11] M. Stevenson. Therapeutic potential of RNA interference , 2004, The New England journal of medicine.
[12] R. Nagel,et al. Sickle-cell disease , 2004, The Lancet.
[13] R. Sandhaus. α1-Antitrypsin deficiency · 6: New and emerging treatments for α1-antitrypsin deficiency , 2004, Thorax.
[14] G. Pellegrini. Changing the cell source in cell therapy? , 2004, The New England journal of medicine.
[15] Y. Saunthararajah,et al. DNA hypo‐methylating agents and sickle cell disease , 2004, British journal of haematology.
[16] P. Zeitlin. Can curcumin cure cystic fibrosis? , 2004, The New England journal of medicine.
[17] H. Paulson,et al. RNAi suppresses polyglutamine-induced neurodegeneration in a model of spinocerebellar ataxia , 2004, Nature Medicine.
[18] J. Muenzer,et al. Advances in the treatment of mucopolysaccharidosis type I. , 2004, The New England journal of medicine.
[19] M. Poe,et al. Cord-blood transplants from unrelated donors in patients with Hurler's syndrome. , 2004, The New England journal of medicine.
[20] R. Desnick,et al. Enzyme replacement and enhancement therapies for lysosomal diseases , 2004, Journal of Inherited Metabolic Disease.
[21] G. Daley,et al. Nuclear transplantation, embryonic stem cells and the potential for cell therapy. , 2003, The hematology journal : the official journal of the European Haematology Association.
[22] G. Lukács,et al. Pharmacologic approaches to correcting the basic defect in cystic fibrosis. , 2003, The New England journal of medicine.
[23] Z. Estrov,et al. Adult stem cells for tissue repair - a new therapeutic concept? , 2003, The New England journal of medicine.
[24] Mark A. Kay,et al. Progress and problems with the use of viral vectors for gene therapy , 2003, Nature Reviews Genetics.
[25] K. High. The risks of germline gene transfer. , 2003, The Hastings Center report.
[26] P. Kaplan,et al. Effectiveness of enzyme replacement therapy in 1028 patients with type 1 Gaucher disease after 2 to 5 years of treatment: a report from the Gaucher Registry. , 2002, The American journal of medicine.
[27] A. Mortellaro,et al. Correction of ADA-SCID by Stem Cell Gene Therapy Combined with Nonmyeloablative Conditioning , 2002, Science.
[28] Jean Bennett,et al. Gene therapy restores vision in a canine model of childhood blindness , 2001, Nature Genetics.
[29] I. Weissman,et al. Translating stem and progenitor cell biology to the clinic: barriers and opportunities. , 2000, Science.
[30] T. Hollon. Researchers and regulators reflect on first gene therapy death , 2000, Nature Medicine.
[31] D. Kelly,et al. Current results and evolving indications for liver transplantation in children. , 1998, Journal of pediatric gastroenterology and nutrition.
[32] J. Wagner,et al. Hematopoietic stem-cell transplantation in globoid-cell leukodystrophy. , 1998, The New England journal of medicine.
[33] P. Lane. Sickle cell disease. , 1996, Pediatric clinics of North America.
[34] B. Childs,et al. Response to treatment in hereditary metabolic disease: 1993 survey and 10-year comparison. , 1995, American journal of human genetics.
[35] T. Starzl,et al. Liver transplantation: a 31-year perspective. Part I. , 1990, Current problems in surgery.
[36] B. Childs,et al. The effect of Mendelian disease on human health. II: Response to treatment. , 1985, American journal of medical genetics.