Safety and efficacy studies of CRISPR-Cas9 treatment of sickle cell disease highlights disease-specific responses-Reference-Cited by-同舟云学术

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Safety and efficacy studies of CRISPR-Cas9 treatment of sickle cell disease highlights disease-specific responses

Published:2024-07 Issue: Volume: Page:
ISSN:1525-0016
Container-title:Molecular Therapy
language:en
Short-container-title:Molecular Therapy

Author:

Frati Giacomo,Brusson Megane,Sartre Gilles,Mlayah Bochra,Felix Tristan,Chalumeau Anne,Antoniou Panagiotis,Hardouin Giulia,Concordet Jean-Paul,Romano Oriana,Turchiano Giandomenico,Miccio Annarita^ORCID

Funder

Institute Imagine Institute of Genetic Diseases

Publisher

Elsevier BV

Reference67 articles.

1. Global epidemiology of sickle haemoglobin in neonates: a contemporary geostatistical model-based map and population estimates;Piel;Lancet,2013

2. The cure of thalassemia by bone marrow transplantation;Lucarelli;Blood Rev.,2002

3. Interim Results from a Phase 1/2 Clinical Study of Lentiglobin Gene Therapy for Severe Sickle Cell Disease;Kanter;Blood,2016

4. Gene Therapy for Sickle Cell Anemia Using a Modified Gamma Globin Lentivirus Vector and Reduced Intensity Conditioning Transplant Shows Promising Correction of the Disease Phenotype;Malik;Blood,2018

5. Gene Therapy in a Patient with Sickle Cell Disease;Ribeil;N. Engl. J. Med.,2017

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