A new age of precision gene therapy

Author:

Schambach Axel,Buchholz Christian J,Torres-Ruiz Raul,Cichutek Klaus,Morgan Michael,Trapani Ivana,Büning Hildegard

Funder

European Research Council

AFM-Téléthon

European Innovation Council

Publisher

Elsevier BV

Subject

General Medicine

Reference138 articles.

1. The Human BioMolecular Atlas Program,2023

2. Gene therapy returns to centre stage;Naldini;Nature,2015

3. Gene therapy to be authorised for first time in EU;Watts;BMJ,2012

4. Determinants of enhancer and promoter activities of regulatory elements;Andersson;Nat Rev Genet,2020

5. Vertex and CRISPR Therapeutics announce authorization of the first CRISPR/Cas9 gene-edited therapy, CASGEVY™ (exagamglogene autotemcel), by the United Kingdom MHRA for the treatment of sickle cell disease and transfusion-dependent beta thalassemia,2023

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