Seminar SeriesDeciphering SPTAN1-associated disease mechanism and potential therapy in patient-derived cell lines
Seminar SeriesTowards a divalent siRNA therapy for prion disease: an academic, patient-scientist led effort
Seminar SeriesAdvancing human hematopoietic stem cell therapies: from sickle cell disease to personalized treatments for bone marrow failure disorders
Seminar SeriesPreclinical development of personalized ASO therapeutics using patient-derived organoid systems
Seminar SeriesTowards a platform for in vivo corrective editing of ultra-rare and N-of-1 metabolic disorders
Seminar SeriesPersonalized Splice-modulating Antisense Oligonucleotide Therapy for PEX1-related Zellweger Spectrum Disorder
Seminar SeriesThe Most Important Aspects of the Guidelines for N=1 Exon Skipping Design and in vitro Development
