Gene therapy for rare haematological and neurometabolic paediatric diseases

Nowadays, gene therapy hast the potential to cure an increasingly greater number of monogenic inherited disorders with absent or limited treatment options, and radically change their natural history. Hematopoietic stem cells (HSCs) represent one of the preferred targets for gene therapy, as genetic...

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Bibliographic Details
Main Authors: Vera Gallo (Author), Alessandro Aiuti (Author)
Format: Book
Published: Elsevier, 2024-09-01T00:00:00Z.
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3rd Floor Main Library

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Call Number: A1234.567
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