Gene therapy for adenosine deaminase-deficient severe combined immune deficiency: clinical comparison of retroviral vectors and treatment plans. (Q36370817)

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English	Gene therapy for adenosine deaminase-deficient severe combined immune deficiency: clinical comparison of retroviral vectors and treatment plans.	scientific article

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13 February 2020

Gene therapy for adenosine deaminase-deficient severe combined immune deficiency: clinical comparison of retroviral vectors and treatment plans (English)

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13 February 2020

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13 February 2020

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Shepherd H Schurman

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Elizabeth Garabedian

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Elizabeth Garabedian

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Kit L Shaw

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Linda Muul

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Denise Carbonaro

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Robert Sokolic

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Christopher Choi

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G Jayashree Jagadeesh

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Pei-Yu Fu

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Eric Gschweng

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John F Tisdale

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Kenneth I Weinberg

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Neena Kapoor

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Ami Shah

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Hisham Abdel-Azim

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Xiao-Jin Yu

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Alan S Wayne

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Howard M Rosenblatt

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Carla M Davis

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Radha G Rishi

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Otto O Yang

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Gerhard Bauer

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Joanna A Ireland

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Barbara C Engel

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Gregory M Podsakoff

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Michael S Hershfield

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R Michael Blaese

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11 September 2012

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3635-3646

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13 February 2020

Myeloid dysplasia and bone marrow hypocellularity in adenosine deaminase-deficient severe combined immune deficiency.

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Genomic instability and myelodysplasia with monosomy 7 consequent to EVI1 activation after gene therapy for chronic granulomatous disease

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Prolonged pancytopenia in a gene therapy patient with ADA-deficient SCID and trisomy 8 mosaicism: a case report

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Successful reconstitution of immunity in ADA-SCID by stem cell gene therapy following cessation of PEG-ADA and use of mild preconditioning

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Long-term efficacy of enzyme replacement therapy for adenosine deaminase (ADA)-deficient severe combined immunodeficiency (SCID).

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The many faces of IL-7: from lymphopoiesis to peripheral T cell maintenance

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30 September 2017

Adenosine deaminase deficiency: genotype-phenotype correlations based on expressed activity of 29 mutant alleles

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Multiple modifications in cis elements of the long terminal repeat of retroviral vectors lead to increased expression and decreased DNA methylation in embryonic carcinoma cells.

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Effects of retroviral vector design on expression of human adenosine deaminase in murine bone marrow transplant recipients engrafted with genetically modified cells

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Hematopoietic stem cell gene therapy for adenosine deaminase-deficient severe combined immunodeficiency leads to long-term immunological recovery and metabolic correction

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21 September 2018

Gene therapy for immunodeficiency due to adenosine deaminase deficiency.

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21 September 2018

Adenosine-deaminase deficiency in two patients with severely impaired cellular immunity

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https://pubmed.ncbi.nlm.nih.gov/22968453

12 December 2020

based on heuristic

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Reference intervals for serum IgG, IgA, IgM, C3, and C4 as determined by rate nephelometry

1 reference

https://pubmed.ncbi.nlm.nih.gov/22968453

12 December 2020

based on heuristic

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Treatment of patients with severe combined immunodeficiency due to adenosine deaminase (ADA) deficiency by autologous transplantation of genetically modified bone marrow cells

1 reference

https://pubmed.ncbi.nlm.nih.gov/22968453

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Brief report: hepatic dysfunction as a complication of adenosine deaminase deficiency

1 reference

https://pubmed.ncbi.nlm.nih.gov/22968453

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

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10.1182/BLOOD-2012-02-400937

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13 February 2020

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13 February 2020

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13 February 2020

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