Sustained improvement of clinical CNS and somatic features of Gaucher disease type 3 after haematopoietic stem cell (HSC) gene therapy: A first-in-world report

Aimee Donald,Jane Potter,Heather J. Church,Bahareh Amirloo,James Cooper, Christine Egerton,Kelly E. Watts, Rebekah A. Hutton,Stewart Rust,Rebecca Bromley, Stavros M. Stivaros, Mathews Adera, Essra Ridha, Milena Veselinovic,Simon A. Jones,Robert Wynn,Alexander Broomfield

Molecular Genetics and Metabolism(2023)

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gaucher disease type,haematopoietic stem cell,gene therapy,clinical cns,first-in-world
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