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Gene therapy for primary immunodeficiencies

Published:2020-12-18 Issue:6 Volume:193 Page:1044-1059
ISSN:0007-1048
Container-title:British Journal of Haematology
language:en
Short-container-title:Br. J. Haematol.

Author:

Fox Thomas A.¹²³^ORCID,Booth Claire³⁴^ORCID

Affiliation:

1. University College London (UCL) Institute of Immunity and Transplantation UCL LondonUK

2. Department of Clinical Haematology UCL Hospitals NHS Foundation Trust LondonUK

3. Molecular and Cellular Immunology Section UCL Great Ormond Street (GOS) Institute of Child Health LondonUK

4. Department of Paediatric Immunology GOS Hospital for Sick Children NHS Foundation Trust London UK

Publisher

Wiley

Subject

Hematology

Link

https://onlinelibrary.wiley.com/doi/pdf/10.1111/bjh.17269

Reference128 articles.

1. International Union of Immunological Societies: 2017 Primary Immunodeficiency Diseases Committee Report on Inborn Errors of Immunity

2. Primary immunodeficiencies

3. Human Inborn Errors of Immunity: 2019 Update of the IUIS Phenotypical Classification

4. Allogeneic HSCT in Adolescents and Young Adults With Primary Immunodeficiencies

5. Treatment of primary immunodeficiency with allogeneic transplant and gene therapy

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1. Recent advances and applications of the CRISPR-Cas system in the gene therapy of blood disorders;Gene;2024-12

2. Design and validation of a GMP stem cell manufacturing protocol for MPSII hematopoietic stem cell gene therapy;Molecular Therapy - Methods & Clinical Development;2024-06

3. Improving access to gene therapy for rare diseases;Disease Models & Mechanisms;2024-04-19

4. Challenges for gene editing in common variable immunodeficiency disorders: Current and future prospects;Clinical Immunology;2024-01

5. Editorial: Advances in therapeutic strategies of inborn errors of immunity;Frontiers in Immunology;2023-11-09

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