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
More News: Bone Marrow Transplant | Brain | Gene Therapy | Genetics | Liver | Liver Transplant | Neurology | Pathology | Peripheral Neuropathy | Research | Stem Cell Therapy | Stem Cells | Study | Transplants | Urology & Nephrology