At the Samaranch Laboratory, we are committed to developing cutting-edge gene therapies for inherited disorders using recombinant AAV-based technology. Our core strength lies in the precise direct delivery of therapeutic agents into the central nervous system. We use stereotactic and microsurgical techniques, including intracranial convection-enhanced delivery, cerebrospinal fluid injections, and in-utero gene transfer. Our research includes capsid development, cell targeting and axonal transport optimization, and immune response assessment to provide disease-specific gene transfer needed to maximize the efficacy and safety of these treatments.
Our current preclinical studies include the therapeutic development for lysosomal storage disorders (Niemann-Pick Type A and GM1 gangliosidosis), neurotransmitter-related diseases (AADC and MAO deficiencies), and neurodegenerative diseases (Parkinson's and Alzheimer's diseases).