Gene therapy for Wiskott-Aldrich syndrome: History, new vectors, future directions-Reference-Cited by-同舟云学术

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Gene therapy for Wiskott-Aldrich syndrome: History, new vectors, future directions

Published:2020-08 Issue:2 Volume:146 Page:262-265
ISSN:0091-6749
Container-title:Journal of Allergy and Clinical Immunology
language:en
Short-container-title:Journal of Allergy and Clinical Immunology

Author:

Ferrua Francesca,Marangoni Francesco,Aiuti Alessandro,Roncarolo Maria Grazia

Funder

National Institutes of Health

National Institute of Arthritis and Musculoskeletal and Skin Diseases

Publisher

Elsevier BV

Subject

Immunology,Immunology and Allergy

Reference10 articles.

1. Clinical manifestations and pathophysiological mechanisms of the Wiskott-Aldrich syndrome;Candotti;J Clin Immunol,2018

2. Excellent outcomes following hematopoietic cell transplantation for Wiskott-Aldrich syndrome: a PIDTC report;Burroughs;Blood,2020

3. Evidence for long-term efficacy and safety of gene therapy for Wiskott–Aldrich syndrome in preclinical models;Marangoni;Mol Ther,2009

4. Gene therapy targeting haematopoietic stem cells for inherited diseases: progress and challenges;Cavazzana;Nat Rev Drug Discov,2019

5. Outcomes following gene therapy in patients with severe Wiskott-Aldrich syndrome;Hacein-Bey Abina;JAMA,2015

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1. Gene therapy in pediatrics – Clinical studies and approved drugs (as of 2023);Life Sciences;2024-07

2. Wiskott-Aldrich syndrome: a study of 577 patients defines the genotype as a biomarker for disease severity and survival;Blood;2024-06-13

3. International Society for Cell & Gene Therapy Stem Cell Engineering Committee report on the current state of hematopoietic stem and progenitor cell–based genomic therapies and the challenges faced;Cytotherapy;2024-06

4. What a Clinician Needs to Know About Genome Editing: Status and Opportunities for Inborn Errors of Immunity;The Journal of Allergy and Clinical Immunology: In Practice;2024-05

5. Generation of stable suspension producer cell lines for serum‐free lentivirus production;Biotechnology Journal;2024-05

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