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Ancestral Sequence Reconstruction as a Lead Optimization Approach in Gene Therapy Drug Development
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The development and functional testing of cellular therapies against protein tyrosine kinase 7 (PTK7) to treat high-risk neuroblastoma
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Pharmacological analysis of liver-directed AAV-fVIII gene therapy and anti-fVIII inhibitor incidence in a preclinical model of hemophilia A
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Non-genotoxic anti-CD117 conditioning for hematopoietic stem cell transplantation and gene therapy applications
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Developing novel cellular and gene therapies for pediatric malignancies
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