Stem Cells & Genome Therapies (BIO330) | 50+ Verified Q&As on iPSCs, Organoids, RPE Cells, GMP, AMD

This document presents over 50 expertly verified questions and answers from the Stem Cells and Genome Therapies curriculum (BIO330), offering a robust study guide for students of regenerative medicine, molecular biology, and advanced biomedical sciences. It explores stem cell biology, disease modeling, therapeutic development, and regulatory frameworks in depth. Topics include: Core properties of stem cells (self-renewal and potency) and their differentiation pathways via the three germ layers Induced pluripotent stem cells (iPSCs): how they are reprogrammed using transcription factors (Klf4, Oct4, Sox2, c-Myc) and applied for autologous transplantation and disease modeling Applications in sensorineural hearing loss: including stem cell-derived organoids, cochlear regeneration, and limbal stem cell therapy Retinal therapies: use of RPE cells from hESCs for macular degeneration and retinal organoid-based approaches for conditions like retinitis pigmentosa Manufacturing & regulation: Definitions of GMP, CMC, QMS, and their associated global regulatory bodies (FDA, MHRA, PMDA) Gene expression control: antisense oligonucleotides (ASOs), RNA toxicity, and epigenetic limitations of iPSCs Cutting-edge disease models using zebrafish retinal regeneration, organoids, and stem-cell-based cell replacement therapies The content reflects real-world application through examples like Auto-CLET, Allo-CLET, and SLET procedures for ocular regeneration, and includes updated insights on age-related macular degeneration (AMD), Fuchs endothelial corneal dystrophy (FECD), and the impact of CTG repeat expansion in the TCF4 gene. This resource is ideal for: Students in regenerative medicine, biomedical sciences, molecular genetics, and biotechnology Medical and pharmacy students studying stem cell therapies and advanced therapeutics Graduate students and research assistants working on organoids, gene expression modulation, or therapeutic cell delivery Professionals preparing for exams involving cell therapy protocols, translational research, or GMP-compliant production Keywords: stem cells, iPSCs, self-renewal, pluripotency, ectoderm, mesoderm, endoderm, organoids, RPE cells, retinal therapy, cochlear regeneration, AMD, ASO therapy, Fuchs dystrophy, TCF4 gene, GMP, QMS, CMC, limbal stem cell therapy, SLET, Auto-CLET, Allo-CLET