“Sometimes real superheroes live in the hearts of small children fighting big battles.”
Peer-Reviewed Scientific Publications
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Gene Therapy and Genetic Medicines
Managing Immune Responses to AAV Gene Therapy
Safety and Efficacy of Gene Therapy for XLMTM: A multi-national, dose escalation trial
Biodistribution of AAV Gene Therapy following CNS administration
Gene Therapy Approaches for Monogenic Developmental Epilepsies
Gene Therapy for XLMTM: effects on muscle pathology
Gene Therapy Approaches for the SLC6A1 Deficiency
Systematic Reviews and Meta-analyses
The Burden of Disease of Crigler Najjar Syndrome
Neuropsychiatric Symptoms in PKU
Invited Discussions and Expert Guidelines