Gene therapy for ADA‐SCID, the first marketing approval of an ex vivo gene therapy in Europe: paving the road for the next generation of advanced therapy medicinal products
Author:
Affiliation:
1. San Raffaele Telethon Institute for Gene Therapy (SR‐Tiget) IRCCS San Raffaele Scientific Institute Milan Italy
2. Vita Salute San Raffaele University Milan Italy
Publisher
EMBO
Subject
Molecular Medicine
Link
https://onlinelibrary.wiley.com/doi/pdf/10.15252/emmm.201707573
Reference15 articles.
1. Correction of ADA-SCID by Stem Cell Gene Therapy Combined with Nonmyeloablative Conditioning
2. Multilineage hematopoietic reconstitution without clonal selection in ADA-SCID patients treated with stem cell gene therapy
3. Gene Therapy for Immunodeficiency Due to Adenosine Deaminase Deficiency
4. T Lymphocyte-Directed Gene Therapy for ADA − SCID: Initial Trial Results After 4 Years
5. Gene Therapy in Peripheral Blood Lymphocytes and Bone Marrow for ADA − Immunodeficient Patients
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