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Severe combined immunodeficiency due to adenosine deaminase deficiency

Subscribe to New Research on Severe combined immunodeficiency due to adenosine deaminase deficiency

Also Known As:

Adenosine deaminase deficiency; Adenosine deaminase-deficient severe combined immunodeficiency disease; Agammaglobulinemia, Swiss type; Bubble boy disease; Delayed-/Late-onset adenosine deaminase deficiency; Partial adenosine deaminase deficiency; SCID1; Severe combined immunodeficiency, alymphocytotic type

Networked: 130 relevant articles (5 outcomes, 10 trials/studies)

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Bio-Agent Context: Research Results

Experts

1.	Roifman, Chaim M: 5 articles (06/2012 - 07/2007)
2.	Grunebaum, Eyal: 5 articles (06/2012 - 07/2007)
3.	Aiuti, Alessandro: 4 articles (06/2011 - 07/2007)
4.	Candotti, Fabio: 2 articles (04/2014 - 10/2009)
5.	Kanegane, Hirokazu: 2 articles (05/2013 - 06/2012)
6.	Miyawaki, Toshio: 2 articles (05/2013 - 06/2012)
7.	Nomura, Keiko: 2 articles (05/2013 - 06/2012)
8.	Gaspar, H B: 2 articles (06/2012 - 11/2009)
9.	Cutz, Ernest: 2 articles (06/2012 - 10/2009)
10.	Hershfield, Michael S: 2 articles (06/2012 - 10/2009)

Related Diseases

1.	X-Linked Combined Immunodeficiency Diseases (X Linked Severe Combined Immunodeficiency) 07/01/2003 - "Advances in the understanding of the genetics of inherited immunologic diseases, gene transfer methodologies, and stem cell manipulation have all contributed to successes in treating X-linked severe combined immunodeficiency and adenosine deaminase deficiency. " 04/01/2014 - "Clinical results from the past 15 years have shown that significant improvement and even cure can be achieved for diseases such as X-linked severe combined immunodeficiency, adenosine deaminase deficiency, chronic granulomatous disease and Wiskott-Aldrich syndrome. " 05/01/2011 - "Retroviral vectors have been developed for hematopoietic stem cell (HSC) gene therapy and have successfully cured X-linked severe combined immunodeficiency (SCID-X1), adenosine deaminase deficiency (ADA-SCID), adrenoleukodystrophy, and Wiskott-Aldrich syndrome. " 01/01/2013 - "Retrovirus-based gene therapy was begun in 1990 for the patients of adenosine deaminase deficiency, followed by X-linked severe combined immunodeficiency, Wiskott-Aldrich syndrome and chronic granulomatous disease. " 12/01/2011 - "To date, HSC gene therapy has been successfully employed in the clinic for improving clinical outcomes in small numbers of patients with X-linked severe combined immunodeficiency (SCID-X1), adenosine deaminase deficiency (ADA-SCID), adrenoleukodystrophy (ALD), thalassemia, chronic granulomatous disease (CGD), and Wiskott-Aldrich syndrome (WAS). "
2.	Wiskott-Aldrich Syndrome (Syndrome, Wiskott-Aldrich) 04/01/2014 - "Clinical results from the past 15 years have shown that significant improvement and even cure can be achieved for diseases such as X-linked severe combined immunodeficiency, adenosine deaminase deficiency, chronic granulomatous disease and Wiskott-Aldrich syndrome. " 05/01/2011 - "Retroviral vectors have been developed for hematopoietic stem cell (HSC) gene therapy and have successfully cured X-linked severe combined immunodeficiency (SCID-X1), adenosine deaminase deficiency (ADA-SCID), adrenoleukodystrophy, and Wiskott-Aldrich syndrome. " 01/01/2013 - "Retrovirus-based gene therapy was begun in 1990 for the patients of adenosine deaminase deficiency, followed by X-linked severe combined immunodeficiency, Wiskott-Aldrich syndrome and chronic granulomatous disease. " 11/01/2009 - "For patients with specific disorders (severe combined immunodeficiency (SCID)-X1, adenosine deaminase deficiency (ADA)-SCID, X-chronic granulomatous disease (CGD) and Wiskott-Aldrich Syndrome (WAS)) who lack a suitable human leukocyte antigen (HLA)-matched donor, gene therapy has offered an important alternative treatment option. " 12/01/2011 - "To date, HSC gene therapy has been successfully employed in the clinic for improving clinical outcomes in small numbers of patients with X-linked severe combined immunodeficiency (SCID-X1), adenosine deaminase deficiency (ADA-SCID), adrenoleukodystrophy (ALD), thalassemia, chronic granulomatous disease (CGD), and Wiskott-Aldrich syndrome (WAS). "
3.	Severe Combined Immunodeficiency (Bare Lymphocyte Syndrome) 01/01/1999 - "Gene therapy for severe combined immunodeficiency caused by adenosine deaminase deficiency: improved retroviral vectors for clinical trials." 06/01/2007 - "In humans, adenosine deaminase deficiency is linked to severe combined immunodeficiency and as such the enzyme has been approved for the first gene therapy trial. " 01/01/1997 - "A study was made of fibroblast strains established from individuals with adenosine deaminase deficiency, T(-)B(-) SCID, Omenn's syndrome and a SCID heterozygote. " 10/01/2013 - "Due to underlying allelic heterogeneity, the disorder manifests as a spectrum, ranging from neonatal onset severe combined immunodeficiency to apparently normal partial adenosine deaminase deficiency. " 10/01/2013 - "Adenosine deaminase deficiency accounts for approximately 15-20% of severe combined immunodeficiency in humans. "
4.	Chronic Granulomatous Disease 01/01/1999 - "Retrovirus-mediated gene transfer has been tested for treatment of diseases that specifically affect the hematopoietic system, such as adenosine deaminase deficiency and chronic granulomatous disease. " 08/01/1996 - "Granulocyte colony-stimulating factor recruitment of CD34+ progenitors to peripheral blood: impaired mobilization in chronic granulomatous disease and adenosine deaminase--deficient severe combined immunodeficiency disease patients." 04/01/2014 - "Clinical results from the past 15 years have shown that significant improvement and even cure can be achieved for diseases such as X-linked severe combined immunodeficiency, adenosine deaminase deficiency, chronic granulomatous disease and Wiskott-Aldrich syndrome. " 01/01/2013 - "Retrovirus-based gene therapy was begun in 1990 for the patients of adenosine deaminase deficiency, followed by X-linked severe combined immunodeficiency, Wiskott-Aldrich syndrome and chronic granulomatous disease. " 11/01/2009 - "For patients with specific disorders (severe combined immunodeficiency (SCID)-X1, adenosine deaminase deficiency (ADA)-SCID, X-chronic granulomatous disease (CGD) and Wiskott-Aldrich Syndrome (WAS)) who lack a suitable human leukocyte antigen (HLA)-matched donor, gene therapy has offered an important alternative treatment option. "
5.	Thalassemia 10/01/1993 - "These range from accurate and simple screening for carriers of thalassemia (Old JM, Varawalla NY, Weatherall DJ: Lancet 2:834-837, 1990) to the use of preimplantation diagnosis of embryos at risk for untreatable congenital defects (Monk M, Holding C: Lancet 1:985-988, 1990) and the development of gene therapy for treatment of disorders such as adenosine deaminase deficiency (Sharp D: Lancet 1:1277-1278, 1991). " 11/01/2005 - "Transfer of drug resistance genes into hematopoietic stem cells (HSCs) has promise for the treatment of a variety of inherited, that is, X-linked severe combined immune deficiency, adenosine deaminase deficiency, thalassemia, and acquired disorders, that is, breast cancer, lymphomas, brain tumors, and testicular cancer. " 12/01/2011 - "To date, HSC gene therapy has been successfully employed in the clinic for improving clinical outcomes in small numbers of patients with X-linked severe combined immunodeficiency (SCID-X1), adenosine deaminase deficiency (ADA-SCID), adrenoleukodystrophy (ALD), thalassemia, chronic granulomatous disease (CGD), and Wiskott-Aldrich syndrome (WAS). "

Related Drugs and Biologics

1.	pegademase bovine (pegADA)
2.	purine
3.	Adenosine Deaminase
4.	Polyethylene Glycols (Polyethylene Glycol)
5.	Purine-Nucleoside Phosphorylase
6.	DNA (Deoxyribonucleic Acid)
7.	Adenosine
8.	Adenine
9.	3-Methyl-2-Oxobutanoate Dehydrogenase (Lipoamide) (Alpha-Keto Acid Dehydrogenase)
10.	Diamond

Related Therapies and Procedures

1.	Immunotherapy
2.	Enzyme Replacement Therapy
3.	Bone Marrow Transplantation (Transplantation, Bone Marrow)
4.	Transplants (Transplant)
5.	Hematopoietic Stem Cell Transplantation