Gene Therapy for the Treatment of Primary Immune Deficiencies
Author:
Publisher
Springer Science and Business Media LLC
Subject
Pulmonary and Respiratory Medicine,Immunology and Allergy,Immunology
Link
http://link.springer.com/content/pdf/10.1007/s11882-016-0615-8.pdf
Reference36 articles.
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2. Pai S-Y, Logan BR, Griffith LM, et al. Transplantation outcomes for severe combined immunodeficiency, 2000–2009. N Engl J Med. 2014;371(5):434–46. doi: 10.1056/NEJMoa1401177 . A major retrospective study of the factors influencing outcomes for patients with SCID treated by allogeneic transplant in multiple major centers across North America, highlighting the benefits of early diagnosis and treatment.
3. Gennery AR, Slatter MA, Grandin L, et al. Transplantation of hematopoietic stem cells and long-term survival for primary immunodeficiencies in Europe: entering a new century, do we do better? J Allergy Clin Immunol. 2010;126(3):602–610.e11. doi: 10.1016/j.jaci.2010.06.015 .
4. Blaese RM, Culver KW, Miller AD, et al. T lymphocyte-directed gene therapy for ADA- SCID: initial trial results after 4 years. Science. 1995;270(5235):475–80. doi: 10.1126/science.270.5235.475 .
5. Chan B, Wara D, Bastian J, et al. Long-term efficacy of enzyme replacement therapy for adenosine deaminase (ADA)-deficient severe combined immunodeficiency (SCID). Clin Immunol. 2005;117(2):133–43. doi: 10.1016/j.clim.2005.07.006 .
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