Dyah Karjosukarso

Cell and gene therapy for thymus-related diseases The main objective is to develop cell and gene therapy to target thymus-related diseases. • Optimized iPSC differentiation into thymus organoids and lentiviral-based gene delivery • Designed, executed, and interpreted cellular and molecular assays • Performed bioinformatics analysis of (single cell) RNA-seq data • Co-authored study reports, patent, and ethical permit applications • Managed research projects and supervised laboratory technicians • Coordinated collaborations with academic partners and CRO

Antisense oligonucleotides for Stargardt disease Principal Investigator: Prof. Dr. Rob Collin, in collaboration with Astherna BV The main objective of this project is to provide evidence of safety and efficacy of antisense oligonucleotides, targeting some variants in Stargardt disease, to be approved for clinical trial. A lead molecule was identified and further investigated by Astherna BV. • Led the development of antisense oligonucleotides-based therapies for Stargardt disease • Generated iPSC-derived retinal organoids and performed cellular and molecular assays • Implemented an AON screening pipeline to identify lead molecule • Designed, executed and analysed experiments within the pipeline • Managed research projects and supervised laboratory technicians • Co-authored SOP, study reports, ethical permit applications, and peer-reviewed publications • Coordinated collaborations with academic partners and CRO

Application of single cell technologies in the investigation of therapeutic resistance in head and neck cancer Principal Investigator: Dr. Klaas Mulder In this project, single cell RNA-seq combined with immunodetection of signaling pathways by sequencing were utilized to study the response and resistance of head and neck squamous cell carcinoma towards given therapy. • Applied single-cell RNA-seq and molecular technologies to study therapeutic resistance in head and neck cancer • Performed bioinformatics analysis in Linux and R on the generated sequencing data • Designed and executed in vivo, cellular and molecular assays • Managed research projects and collaborations with academic partners • Co-authored ethical permit application and peer-reviewed publications • Provided teaching and supervision to students

Thesis title: "Towards deeper molecular insight into familial exudative vitreoretinopathy: Zinc-ing deep into the vessels" Promotors: Prof. Dr. Rob Collin and Prof. Dr. Frans Cremers Co-promotor: Dr. Huiqing Zhou The thesis comprises of characterization of ZNF408, a gene that is mutated in a inherited blindness termed familial exudative vitreoretinopathy (FEVR). In FEVR patients, the blood vessels supporting the retina are damaged, which cause visual impairment. The study includes investigation of ZNF408 function and how its defect lead to vision loss, both at in vitro and in vivo level. • Designed and conducted in vitro and in vivo studies using endothelial cell culture, iPSC differentiation toward the endothelial lineage, and zebrafish models (CRISPR/Cas9, visuomotor assays) • Applied molecular techniques including DNA/RNA isolation, cloning, gene expression analysis, Sanger sequencing, RNA-seq, ChIP-seq, Western blot, and immunofluorescence • Performed bioinformatics analyses of sequencing data to elucidate gene function and pathogenic mechanisms • Co-authored manuscripts and presented findings at international conferences

Lineage Primed State Pluripotency

Identification of ZNF408 Transcriptional Targets

Candidate Genes Approach in Elucidation of Genetic Defect in Pontine Tegmental Cap Dysplasia and Axonal Charcot-Marie-Tooth Disease

Understanding miRNA-mediated Regulation of CD247 in the Pathogenesis of Systemic Scleroderma and Methylation Analysis of IRX3 in association with Type 1 Diabetes

Graduated cum laude

Graduated cum laude