Phenotyping of Human Ipsc-Derived Neurons: Patient-Driven Research

ISBN-13: 9780128222775 / Angielski / Miękka / 2022

Elizabeth D. Buttermore

Spis treści

Section I: Best practices and considerations when designing a new project 1. iPSC culture: best practices from sample procurement to reprogramming and differentiation 2. Phenotypic assay development with iPSC-derived neurons: technical 3. Derivation of cortical interneurons from human pluripotent stem cells to model neurodevelopmental disorders 4. Development of transcription factor-based strategies for neuronal differentiation from pluripotent stem cells 5. Differentiation of Purkinje cells from pluripotent stem cells for disease phenotyping in vitro 6. Brain organoids: models of cell type diversity, connectivity, and disease phenotypes

Section II: The use of iPSC-derived neurons to study neurological disorders 7. Human models as new tools for drug development and precision medicine 8. Use of cerebral organoids to model environmental and gene x environment interactions in the developing fetus and neurodegenerative disorders 9. iPSC-derived models of autism: Tools for patient phenotyping and assay-based drug discovery 10. Probing the electrophysiological properties of patient-derived neurons across neurodevelopmental disorders 11. Advantages and limitations of hiPSC-derived neurons for the study of neurodegeneration

Section III: New technology, industry perspective, and transitioning to the clinic 12. Developing clinically translatable screens using iPSC-derived neural cells 13. Gene editing hPSCs for modeling neurological disorders 14. Cell therapy and biomanufacturing using hiPSC-derived neurons 15. Ethical considerations for the use of stem cell-derived therapies

O autorze

Dr. Elizabeth Buttermore is currently the Director of Translational In Vitro Models in the Rosamund Stone Zander Translational Neuroscience Center at Boston Children's Hospital (BCH) where she leads the Human Neuron Core and Neurological Repository Core. Elizabeth has 10 years of experience in phenotypic assay development using high content imaging and multielectrode array approaches. One of her goals at BCH is to help standardize the way the field obtains and interprets phenotypic data and to help researchers across academia and industry move their research forward. She completed her postdoctoral work in Clifford Woolf's lab at BCH where she developed protocols for differentiating nociceptive neurons from human fibroblasts and iPSCs and used them to develop models for neuropathy and neuropathic pain. Prior to coming to BCH, Elizabeth completed her PhD in Neurobiology in 2012 at the University of North Carolina, in the lab of Manzoor Bhat, where she studied the organization and maintenance of molecular domains in myelinated axons.