Haematopoietic stem cell gene therapy in inborn errors of metabolism-Reference-Cited by-同舟云学术

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Haematopoietic stem cell gene therapy in inborn errors of metabolism

Published:2022-05-10 Issue:2 Volume:198 Page:227-243
ISSN:0007-1048
Container-title:British Journal of Haematology
language:en
Short-container-title:Br J Haematol

Author:

Chiesa Robert¹,Bernardo Maria Ester²³

Affiliation:

1. Bone Marrow Transplantation Department Great Ormond Street Hospital for Sick Children NHS Foundation Trust London UK

2. Pediatric Immunohematology and Bone Marrow Transplantation Unit San Raffaele Scientific Institute Milan Italy

3. “Vita Salute” San Raffaele University Milan Italy

Publisher

Wiley

Subject

Hematology

Link

https://onlinelibrary.wiley.com/doi/pdf/10.1111/bjh.18179

Reference86 articles.

1. Inborn Errors of Metabolism

2. Haematopoietic stem cell transplantation in inborn errors of metabolism

3. Autologous Stem-Cell-Based Gene Therapy for Inherited Disorders: State of the Art and Perspectives

4. Incidence of X-linked adrenoleukodystrophy and the relative frequency of its phenotypes

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1. Advanced Therapy Medicinal Products: Clinical, Non-clinical, and Quality Considerations;Pharmaceutical Biotechnology;2024

2. Preclinical lentiviral vector-mediated hematopoietic stem and progenitor cell gene therapy corrects Pompe disease-related muscle and neurological manifestations;2023-11-05

3. Gene and Cellular Therapies for Leukodystrophies;Pharmaceutics;2023-10-24

4. Development of analytics in newborn screening—from the Guthrie card to genetics;Bundesgesundheitsblatt - Gesundheitsforschung - Gesundheitsschutz;2023-10-12

5. Current and Future Perspective in Hematopoietic Stem Progenitor Cell-gene Therapy for Inborn Errors of Metabolism;HemaSphere;2023-09-12

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