Update on Clinical Ex Vivo Hematopoietic Stem Cell Gene Therapy for Inherited Monogenic Diseases-Reference-Cited by-同舟云学术

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Update on Clinical Ex Vivo Hematopoietic Stem Cell Gene Therapy for Inherited Monogenic Diseases

Published:2021-02 Issue:2 Volume:29 Page:489-504
ISSN:1525-0016
Container-title:Molecular Therapy
language:en
Short-container-title:Molecular Therapy

Author:

Tucci Francesca,Scaramuzza Samantha,Aiuti Alessandro^ORCID,Mortellaro Alessandra

Funder

European Commission

Else Kröner-Fresenius-Stiftung

Jeffrey Modell Foundation

Fondazione Telethon

Ministero della Salute

Publisher

Elsevier BV

Subject

Drug Discovery,Pharmacology,Genetics,Molecular Biology,Molecular Medicine

Reference130 articles.

1. Hematopoietic cell transplantation in primary immunodeficiency–conventional and emerging indications;Slatter;Expert Rev. Clin. Immunol.,2018

2. Hematopoietic stem cell transplantation in inborn errors of metabolism;Tan;Front Pediatr.,2019

3. Hematopoietic stem cell gene therapy for adenosine deaminase-deficient severe combined immunodeficiency leads to long-term immunological recovery and metabolic correction;Gaspar;Sci. Transl. Med.,2011

4. Gene therapy for immunodeficiency due to adenosine deaminase deficiency;Aiuti;N. Engl. J. Med.,2009

5. LMO2-associated clonal T cell proliferation in two patients after gene therapy for SCID-X1;Hacein-Bey-Abina;Science,2003

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1. Gene therapy for polygenic or complex diseases;Biomarker Research;2024-09-04

2. The Role of the Conditioning Regimen for Autologous and Ex Vivo Genetically Modified Hematopoietic Stem Cell-based Therapies: Recommendations from the ISCT Stem Cell Engineering Committee;Cytotherapy;2024-09

3. Advances in Pompe Disease Treatment: From Enzyme Replacement to Gene Therapy;Molecular Diagnosis & Therapy;2024-08-12

4. Phase I clinical trial of intracerebral injection of lentiviral-ABCD1 for the treatment of cerebral adrenoleukodystrophy;Science Bulletin;2024-08

5. CNS-wide repopulation by hematopoietic-derived microglia-like cells corrects progranulin deficiency in mice;Nature Communications;2024-07-05

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