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Current Opinion in Biotechnology, ISSN 0958-1669, 12/2019, Volume 60, pp. 39 - 45
Genetic diseases affecting proteins and cells composing the blood may be treated by gene therapy using gene addition or gene editing methods. Protein... 
Journal Article
Blood, ISSN 0006-4971, 09/2017, Volume 130, Issue 11, pp. 1278 - 1279
In this issue of Blood, Morris et al describe application of autologous transplant/ gene therapy for Wiskott-Aldrich syndrome (WAS), first demonstrated to be... 
HEMATOLOGY | Animals, Newborn | Genetic Therapy | Animals | Humans
Journal Article
Current opinion in biotechnology, 12/2018, Volume 60, p. 39
Genetic diseases affecting proteins and cells composing the blood may be treated by gene therapy using gene addition or gene editing methods. Protein... 
Journal Article
Cytotherapy, ISSN 1465-3249, 2018, Volume 21, Issue 3, pp. 358 - 366
Journal Article
Journal Article
Molecular Therapy - Methods & Clinical Development, ISSN 2329-0501, 06/2019, Volume 13, pp. 390 - 398
Lentiviral vector (LV)-based hematopoietic stem and progenitor cell (HSPC) gene therapy is becoming a promising alternative to allogeneic stem cell... 
gene therapy | HSCs | lentivirus | sickle cell disease | SYSTEM | MEDICINE, RESEARCH & EXPERIMENTAL | GENE-THERAPY | SICKLE-CELL-DISEASE | HUMAN HEMATOPOIETIC STEM | EFFICIENCY
Journal Article
Blood, ISSN 0006-4971, 06/2015, Volume 125, Issue 23, pp. 3521 - 3522
In this issue of Blood, Touzot et al report that autologous gene therapy/hematopoietic stem cell transplantation (HSCT) for infants with X-linked severe... 
HEMATOLOGY | SEVERE COMBINED IMMUNODEFICIENCY | Genetic Therapy | Humans | Hematopoietic Stem Cell Transplantation | X-Linked Combined Immunodeficiency Diseases - therapy | Female | Male | T-Lymphocytes - immunology | Thymus Gland - immunology | Interleukin Receptor Common gamma Subunit - immunology
Journal Article
Science Translational Medicine, ISSN 1946-6234, 08/2011, Volume 3, Issue 97
Journal Article
by Kwan, Antonia and Abraham, Roshini S and Currier, Robert and Brower, Amy and Andruszewski, Karen and Abbott, Jordan K and Baker, Mei and Ballow, Mark and Bartoshesky, Louis E and Bonilla, Francisco A and Brokopp, Charles and Brooks, Edward and Caggana, Michele and Celestin, Jocelyn and Church, Joseph A and Comeau, Anne Marie and Connelly, James A and Cowan, Morton J and Cunningham-Rundles, Charlotte and Dasu, Trivikram and Dave, Nina and De La Morena, Maria T and Duffner, Ulrich and Fong, Chin-To and Forbes, Lisa and Freedenberg, Debra and Gelfand, Erwin W and Hale, Jaime E and Hanson, I. Celine and Hay, Beverly N and Hu, Diana and Infante, Anthony and Johnson, Daisy and Kapoor, Neena and Kay, Denise M and Kohn, Donald B and Lee, Rachel and Lehman, Heather and Lin, Zhili and Lorey, Fred and Abdel-Mageed, Aly and Manning, Adrienne and McGhee, Sean and Moore, Theodore B and Naides, Stanley J and Notarangelo, Luigi D and Orange, Jordan S and Pai, Sung-Yun and Porteus, Matthew and Rodriguez, Ray and Romberg, Neil and Routes, John and Ruehle, Mary and Rubenstein, Arye and Saavedra-Matiz, Carlos A and Scott, Ginger and Scott, Patricia M and Secord, Elizabeth and Seroogy, Christine and Shearer, William T and Siegel, Subhadra and Silvers, Stacy K and Stiehm, E. Richard and Sugerman, Robert W and Sullivan, John L and Tanksley, Susan and Tierce, Millard L and Verbsky, James and Vogel, Beth and Walker, Rosalyn and Walkovich, Kelly and Walter, Jolan E and Wasserman, Richard L and Watson, Michael S and Weinberg, Geoffrey A and Weiner, Leonard B and Wood, Heather and Yates, Anne B and Puck, Jennifer M
JAMA, ISSN 0098-7484, 08/2014, Volume 312, Issue 7, pp. 729 - 738
Journal Article
Nature Biotechnology, ISSN 1087-0156, 07/2016, Volume 34, Issue 7, pp. 695 - 697
Journal Article
Journal Article
Nature Biotechnology, ISSN 1087-0156, 08/2010, Volume 28, Issue 8, pp. 839 - 847
Journal Article