SUMMARY Ex vivo hematopoietic stem cell (HSC) gene therapy is effective for non-malignant blood disorders including sickle cell disease (SCD), but requires hospitalization, ex vivo cell manipulation, and conditioning. Direct in vivo gene delivery could remove these barriers and widen access. Here, we explore phagocytos...
Denise Klatt, Adele Mucci, Bo-Ya Liu et al.· Cell Stem Cell· 0 citations
The main goal of the German National Strategy for GCT is to ensure patient access to advanced therapies while strengthening Germany’s position as an international hub for biomedical innovation.
Christian Gallus, F. Ayuk, P. Beckhove et al.· Human Gene Therapy· 0 citations
Autologous HSC gene therapy is supported as an effective alternative to alloHSCT with a favorable risk-benefit profile for severe LAD-I, and markedly lower annualized incidences of prespecified serious infections, infection-related hospitalizations, and prolonged infection-related hospitalizations after RP-L201 treatme...
C. Booth, J. Sevilla, E. Almarza et al.· Journal of Human Immunity· 0 citations
Lentiviral vectors have revolutionized gene therapy by efficient and stable transduction of dividing and non-dividing cells, their large packaging capacity, and their compatibility with pseudotyping to alter viral tropism. The vesicular stomatitis virus glycoprotein (VSV-G) is widely used as a viral envelope protein of...
Anjali Shrivastava, Felix L. Warnecke, J. Schott et al.· Molecular Therapy· 0 citations
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