The Path to Progress Preclinical Studies of Age-Related Neurodegenerative Diseases: A Perspective on Rodent and hiPSC-Derived Models

Scientists advocate for a paradigm shift towards the application of human iPSC-derived systems for Alzheimer’s and Parkinson’s disease modeling and the development of improved human-based models in a dish for drug discovery and preclinical assessment of therapeutic targets.
[Molecular Therapy]
MacDougall, G., Brown, L. Y., Kantor, B., & Chiba-Falek, O. (2021). The path to progress preclinical studies of age-related neurodegenerative diseases: A perspective on rodent and hiPSC-derived models. Molecular Therapy, 0(0). https://doi.org/10.1016/j.ymthe.2021.01.001 Cite
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Mitochondrial DNA Dynamics in Reprogramming to Pluripotency

The authors examine potential mechanisms for maintenance or shifts in heteroplasmy that occur in iPSCs generated by cellular reprogramming, and further discuss manipulations that can alter heteroplasmy to impact stem and differentiated cell performance.
[Trends in Cell Biology]
Sercel, A. J., Carlson, N. M., Patananan, A. N., & Teitell, M. A. (2021). Mitochondrial DNA Dynamics in Reprogramming to Pluripotency. Trends in Cell Biology, 0(0). https://doi.org/10.1016/j.tcb.2020.12.009 Cite
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BlueRock Therapeutics in Collaboration with Memorial Sloan Kettering Cancer Center Receives IND Clearance for DA01 in Parkinson’s Disease

BlueRock Therapeutics, in collaboration with Memorial Sloan Kettering Cancer Center, announced that the US FDA has cleared their Investigational New Drug application to proceed with a Phase I study in patients with advanced Parkinson’s disease (PD). This is the first trial in the US to study pluripotent stem cell-derived dopaminergic neurons in patients with Parkinson’s disease.
[BlueRock Therapeutics (PR Newswire, Inc.)]
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Single Cell Transcriptomic Analysis of Human Pluripotent Stem Cell Chondrogenesis

Scientists combined bulk RNA sequencing, single cell RNA sequencing, and bioinformatic analyses, including weighted gene co-expression analysis, to investigate the gene regulatory networks regulating human iPSC differentiation under chondrogenic conditions.
[Nature Communications]
Wu, C.-L., Dicks, A., Steward, N., Tang, R., Katz, D. B., Choi, Y.-R., & Guilak, F. (2021). Single cell transcriptomic analysis of human pluripotent stem cell chondrogenesis. Nature Communications, 12(1), 362. https://doi.org/10.1038/s41467-020-20598-y Cite
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Culture Surface Protein Coatings Affect the Barrier Properties and Calcium Signaling of hESC-RPE

Investigators identified the role of retinal pigment epithelium (RPE) basement membrane proteins type IV collagen, laminin, and nidogen-1 in the maturation and functionality of humn ESC-RPE after cryopreservation.
[Scientific Reports]
Viheriälä, T., Sorvari, J., Ihalainen, T. O., Mörö, A., Grönroos, P., Schlie-Wolter, S., Chichkov, B., Skottman, H., Nymark, S., & Ilmarinen, T. (2021). Culture surface protein coatings affect the barrier properties and calcium signalling of hESC-RPE. Scientific Reports, 11(1), 933. https://doi.org/10.1038/s41598-020-79638-8 Cite
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Immune-Tolerance to Human iPS-Derived Neural Progenitors Xenografted Into the Immature Cerebellum Is Overridden by Species-Specific Differences in Differentiation Timing

Scientists found that the slower pace of differentiation of human neural precursors compared to that of rodents restricted induction of immune-tolerance to human antigens expressed before completion of maturation of the immune system.
[Scientific Reports]
Nato, G., Corti, A., Parmigiani, E., Jachetti, E., Lecis, D., Colombo, M. P., Delia, D., Buffo, A., & Magrassi, L. (2021). Immune-tolerance to human iPS-derived neural progenitors xenografted into the immature cerebellum is overridden by species-specific differences in differentiation timing. Scientific Reports, 11(1), 651. https://doi.org/10.1038/s41598-020-79502-9 Cite
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Stem-Cell-Derived Human Microglia Transplanted Into Mouse Brain To Study Human Disease

Scientists detailed an accurate, step-by-step workflow that includes in vitro microglia differentiation from human pluripotent stem cells, transplantation into the mouse brain and quantitative analysis of engraftment.
[Nature Protocols]
Fattorelli, N., Martinez-Muriana, A., Wolfs, L., Geric, I., De Strooper, B., & Mancuso, R. (2021). Stem-cell-derived human microglia transplanted into mouse brain to study human disease. Nature Protocols, 1–22. https://doi.org/10.1038/s41596-020-00447-4 Cite
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Induced Organoids Derived from Patients with Ulcerative Colitis Recapitulate Colitic Reactivity

Researchers took advantage of induced pluripotent stem cells to develop an induced human UC-derived organoid model and compared it with the induced human normal organoid model.
[Nature Communications]
Sarvestani, S. K., Signs, S., Hu, B., Yeu, Y., Feng, H., Ni, Y., Hill, D. R., Fisher, R. C., Ferrandon, S., DeHaan, R. K., Stiene, J., Cruise, M., Hwang, T. H., Shen, X., Spence, J. R., & Huang, E. H. (2021). Induced organoids derived from patients with ulcerative colitis recapitulate colitic reactivity. Nature Communications, 12(1), 262. https://doi.org/10.1038/s41467-020-20351-5 Cite
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Engineering Human Hepato-Biliary-Pancreatic Organoids from Pluripotent Stem Cells

Investigators describe a protocol for the continuous patterning of hepatic, biliary and pancreatic structures from a 3D culture of human pluripotent stem cells.
[Nature Protocols]
Koike, H., Iwasawa, K., Ouchi, R., Maezawa, M., Kimura, M., Kodaka, A., Nishii, S., Thompson, W. L., & Takebe, T. (2021). Engineering human hepato-biliary-pancreatic organoids from pluripotent stem cells. Nature Protocols, 1–18. https://doi.org/10.1038/s41596-020-00441-w Cite
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BlueRock Therapeutics in Collaboration with Memorial Sloan Kettering Cancer Center Receives IND Clearance for DA01 in Parkinson’s Disease

BlueRock Therapeutics, LLC. announced that the FDA has cleared their Investigational New Drug (IND) application to proceed with a Phase I study in patients with advanced Parkinson’s disease (PD). This is the first trial in the United States to study pluripotent stem cell-derived dopaminergic neurons in patients with PD.
[BlueRock Therapeutics, LLC.]
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Chromatin Regulatory Dynamics of Early Human Small Intestinal Development Using a Directed Differentiation Model

Researchers leveraged chromatin run-on sequencing to define the landscape of active promoters, enhancers and gene bodies across distinct stages of directed differentiation of human pluripotent stem cells into small intestinal spheroids with regional specification.
[Nucleic Acids Research]
Hung, Y.-H., Huang, S., Dame, M. K., Yu, Q., Yu, Q. C., Zeng, Y. A., Camp, J. G., Spence, J. R., & Sethupathy, P. (2021). Chromatin regulatory dynamics of early human small intestinal development using a directed differentiation model. Nucleic Acids Research, gkaa1204. https://doi.org/10.1093/nar/gkaa1204 Cite
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