Preclinical Safety and Efficacy of Human CD34+ Cells Transduced With Lentiviral Vector for the Treatment of Wiskott-Aldrich Syndrome-Reference-Cited by-同舟云学术

Preclinical Safety and Efficacy of Human CD34+ Cells Transduced With Lentiviral Vector for the Treatment of Wiskott-Aldrich Syndrome

Published:2013-01 Issue:1 Volume:21 Page:175-184
ISSN:1525-0016
Container-title:Molecular Therapy
language:en
Short-container-title:Molecular Therapy

Author:

Scaramuzza Samantha,Biasco Luca,Ripamonti Anna,Castiello Maria C,Loperfido Mariana,Draghici Elena,Hernandez Raisa J,Benedicenti Fabrizio,Radrizzani Marina,Salomoni Monica,Ranzani Marco,Bartholomae Cynthia C,Vicenzi Elisa,Finocchi Andrea,Bredius Robbert,Bosticardo Marita,Schmidt Manfred,von Kalle Christof,Montini Eugenio,Biffi Alessandra,Roncarolo Maria G,Naldini Luigi,Villa Anna,Aiuti Alessandro

Publisher

Elsevier BV

Subject

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

Reference45 articles.

1. Ten years of gene therapy for primary immune deficiencies;Aiuti;Hematology Am Soc Hematol Educ Program,2009

2. 20 years of gene therapy for SCID;Fischer;Nat Immunol,2010

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

4. Efficacy of gene therapy for X-linked severe combined immunodeficiency;Hacein-Bey-Abina;N Engl J Med,2010

5. Gene therapy of X-linked severe combined immunodeficiency by use of a pseudotyped gammaretroviral vector;Gaspar;Lancet,2004

Cited by 73 articles. 订阅此论文施引文献订阅此论文施引文献，注册后可以免费订阅5篇论文的施引文献，订阅后可以查看论文全部施引文献

1. Systematic Review of Genetic Substrate Reduction Therapy in Lysosomal Storage Diseases: Opportunities, Challenges and Delivery Systems;BioDrugs;2024-08-23

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

3. Gene Therapy for Primary Immune Deficiency Diseases;Clinical Immunology;2023

4. A novel preclinical model of mucopolysaccharidosis type II for developing human hematopoietic stem cell gene therapy;Gene Therapy;2022-07-14

5. Catching Them Early: Framework Parameters and Progress for Prenatal and Childhood Application of Advanced Therapies;Pharmaceutics;2022-04-05