Lentiviral vector ALS20 yields high hemoglobin levels with low genomic integrations for treatment of beta-globinopathies-Reference-Cited by-同舟云学术

Lentiviral vector ALS20 yields high hemoglobin levels with low genomic integrations for treatment of beta-globinopathies

Published:2021-04 Issue:4 Volume:29 Page:1625-1638
ISSN:1525-0016
Container-title:Molecular Therapy
language:en
Short-container-title:Molecular Therapy

Author:

Breda Laura,Ghiaccio Valentina,Tanaka Naoto,Jarocha Danuta,Ikawa Yasuhiro,Abdulmalik Osheiza,Dong Alisa,Casu Carla,Raabe Tobias D.,Shan Xiaochuan,Danet-Desnoyers Gwenn A.,Doto Aoife M.,Everett John,Bushman Frederic D.,Radaelli Enrico,Assenmacher Charles A.,Tarrant James C.,Hoepp Natalie,Kurita Ryo,Nakamura Yukio,Guzikowski Virginia,Smith-Whitley Kim,Kwiatkowski Janet L.,Rivella Stefano

Funder

Pennsylvania Department of Health

Children's Hospital of Philadelphia

Publisher

Elsevier BV

Subject

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

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4. Northstar-2: Updated Safety and Efficacy Analysis of Lentiglobin Gene Therapy in Patients with Transfusion-Dependent β-Thalassemia and Non-β0/β0 Genotypes;Thompson;Blood,2019

5. Northstar-3: Interim Results from a Phase 3 Study Evaluating Lentiglobin Gene Therapy in Patients with Transfusion-Dependent β-Thalassemia and Either a β0 or IVS-I-110 Mutation at Both Alleles of the HBB Gene;Lal;Blood,2019

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