Sage Journals: Discover world-class research

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Research article

First published 1993-08

Transfer of the ADA Gene into Bone Marrow Cells and Peripheral Blood Lymphocytes for the Treatment of Patients Affected by ADA-Deficient SCID

Claudio Bordignon, Fulvio Mavilio, Giuliana Ferrari [...]

Human Gene Therapy

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Review article

First published 2017-11

Twenty-Five Years of Gene Therapy for ADA-SCID: From Bubble Babies to an Approved Drug

Francesca Ferrua, Alessandro Aiuti

Human Gene Therapy

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Research article

First published 2016-06

Investor Outlook: Rising from the Ashes;GSK's European Approval of Strimvelis for ADA-SCID

Joshua Schimmer, Steven Breazzano

Human Gene Therapy Clinical Development

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Research article

First published 2010-2

Telethon experience and its efforts towards the registration of new advanced therapies

Carla Paganin

Pharmaceuticals Policy and Law

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Research article

First published 1991-10

Pegademase Bovine: Replacement Therapy for Severe Combined Immunodeficiency Disease

Connie R. Lee, Constance A. McKenzie, Kathy D. Webster [...]

Annals of Pharmacotherapy

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Research article

First published 2006-03

High Incidence of Severe Combined Immune Deficiency in the Eastern Province of Saudi Arabia

Fawzi Suliaman, Abdulaziz Al-Ghonaium, Harb Harfi

Pediatric Allergy,Immunology,and Pulmonology

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Case report

First published 2015-06

Adenosine Deaminase-Deficient Severe Combined Immunodeficiency and Diffuse Large B-Cell Lymphoma

Ferah Genel, Erhan Ozbek, Gulcihan Ozek [...]

Pediatric Allergy,Immunology,and Pulmonology

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Review article

First published 2015-04

Clinical Applications of Gene Therapy for Primary Immunodeficiencies

Maria Pia Cicalese, Alessandro Aiuti

Human Gene Therapy

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Research article

First published 1993-08

Treatment of Severe Combined Immunodeficiency Disease (SCID) due to Adenosine Deaminase Deficiency with CD34 + Selected Autologous Peripheral Blood Cells Transduced with a Human ADA Gene (Amendment). National Institutes of Health

R. Michael Blaese, Kenneth W. Culver, W. French Anderson [...]

Human Gene Therapy

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Research article

First published 2017-03

Good Laboratory Practice Preclinical Safety Studies for GSK2696273 (MLV Vector-Based Ex Vivo Gene Therapy for Adenosine Deaminase Deficiency Severe Combined Immunodeficiency) in NSG Mice

Nicola Carriglio, Jan Klapwijk, Raisa Jofra Hernandez [...]

Human Gene Therapy Clinical Development

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