Conditions for combining gene therapy with bone marrow transplantation in murine Krabbe disease.

Conclusion: These studies provide the conditions optimum for successfully treating the murine model of KD. There is some flexibility in dosing and timing to obtain a satisfactory outcome. These studies are critical to the planning of a human trial combining the "standard of care", HSCT, with a single iv injection of AAVrh10-GALC. PMID: 32363154 [PubMed]
Source: BioImpacts - Category: Research Tags: Bioimpacts Source Type: research