Autologous haematopoietic stem cell gene therapy (HSC-GT) is an emerging therapy for treatment-refractory neurodegenerative lysosomal storage disorders (LSD). Mucopolysaccharidosis IIIA (MPSIIIA) is a devastating LSD where pathogenic variants in the SGSH gene lead to toxic heparan sulfate accumulation, inflammation and...
J. Kinsella, Simon A. Jones, K. Buckland et al.· Molecular Therapy· 0 citations
BG147 promises to transform ex vivo and in vivo gene therapies by transiently inhibiting intrinsic immune barriers mediated by IFITM3 to enhance a wide range of protocols.
Dara Annett, Bethan J. Critchley, Kate L. Morling et al.· Molecular Therapy· 0 citations
Efficient and precise correction of STAT1 GOF mutations by base editing is demonstrated, with maintenance of the correction through long-term engraftment in vivo, which represents the first application of gene editing to correct a dominant gain-of-function mutation causing immunodeficiency.
Robert Torrance, K. Orf, Nathan White et al.· Blood· 0 citations
A clinical-compatible platform enabling large-scale manufacturing while preserving HSPC viability, stemness, and multilineage functionality is established and a robust and adaptable roadmap for future HDR-based gene editing platforms is offered.
A. Naseem, W. Vetharoy, T. E. Whittaker et al.· Molecular therapy. Advances· 1 citation
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