Gene therapy for the treatment of adenosine deaminase-deficient severe combined immune deficiency-Reference-Cited by-同舟云学术

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Gene therapy for the treatment of adenosine deaminase-deficient severe combined immune deficiency

Published:2017-05-22 Issue:6 Volume:5 Page:477-485
ISSN:2167-8707
Container-title:Expert Opinion on Orphan Drugs
language:en
Short-container-title:Expert Opinion on Orphan Drugs

Author:

Sokolic Robert¹²,Candotti Fabio³

Affiliation:

1. Office of Tissues and Advanced Therapies, Center for Biologics Evaluation and Research, Food and Drug Administration, Silver Spring, MD, United States of America

2. Division of Hematology/Oncology, University Medicine Foundation, Providence, RI

3. Immunology and Allergy Service, Department of Medicine Centre Hospitalier, Universitaire Vaudois, Lausanne, Switzerland

Funder

UNIL-CHUV

U.S. Food and Drug Administration

Publisher

Informa UK Limited

Subject

Pharmacology (medical),Health Policy,Pharmacology, Toxicology and Pharmaceutics (miscellaneous)

Link

https://www.tandfonline.com/doi/pdf/10.1080/21678707.2017.1325360

Reference58 articles.

1. Adenosine deaminase deficiency in a child with severe combined immunodeficiency

2. Adenosine Deaminase Deficiency – More Than Just an Immunodeficiency

3. Effect of Polyethylene Glycol-Modified Adenosine Deaminase (PEG-ADA) Therapy in Two ADA-Deficient Children: Measurement of Erythrocyte Deoxyadenosine Triphosphate as a Useful Tool

4. Outcome of hematopoietic stem cell transplantation for adenosine deaminase–deficient severe combined immunodeficiency

5. Mismatched Related and Unrelated Donors for Allogeneic Hematopoietic Cell Transplantation for Adults with Hematologic Malignancies

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