Lineage tracing of stem cell-derived dopamine grafts in a Parkinson's model reveals shared origin of all graft-derived cells
(2024) In Science Advances 10(42). p.1-14- Abstract
Stem cell therapies for Parkinson's disease are at an exciting time of development, and several clinical trials have recently been initiated. Human pluripotent stem cells are differentiated into transplantable dopamine (DA) progenitors which are proliferative at the time of grafting and undergo terminal differentiation and maturation in vivo. While the progenitors are homogeneous at the time of transplantation, they give rise to heterogeneous grafts composed not only of therapeutic DA neurons but also of other mature cell types. The mechanisms for graft diversification are unclear. We used single-nucleus RNA-seq and ATAC-seq to profile DA progenitors before transplantation combined with molecular barcode-based tracing to determine... (More)
Stem cell therapies for Parkinson's disease are at an exciting time of development, and several clinical trials have recently been initiated. Human pluripotent stem cells are differentiated into transplantable dopamine (DA) progenitors which are proliferative at the time of grafting and undergo terminal differentiation and maturation in vivo. While the progenitors are homogeneous at the time of transplantation, they give rise to heterogeneous grafts composed not only of therapeutic DA neurons but also of other mature cell types. The mechanisms for graft diversification are unclear. We used single-nucleus RNA-seq and ATAC-seq to profile DA progenitors before transplantation combined with molecular barcode-based tracing to determine origin and shared lineages of the mature cell types in the grafts. Our data demonstrate that astrocytes, vascular leptomeningeal cells, and DA neurons are the main component of the DAergic grafts, originating from a common progenitor that is tripotent at the time of transplantation.
(Less)
- author
- organization
-
- MultiPark: Multidisciplinary research focused on Parkinson's disease
- StemTherapy: National Initiative on Stem Cells for Regenerative Therapy
- Developmental and Regenerative Neurobiology (research group)
- Wallenberg Neuroscience Centre, Lund
- WCMM-Wallenberg Centre for Molecular Medicine
- Division of Molecular Hematology (DMH)
- LTH Profile Area: Engineering Health
- Molecular Neuromodulation (research group)
- Developmental Immunology (research group)
- Human Neural Developmental Biology (research group)
- publishing date
- 2024-10-18
- type
- Contribution to journal
- publication status
- published
- subject
- keywords
- Parkinson Disease/metabolism, Cell Lineage/genetics, Animals, Dopaminergic Neurons/metabolism, Humans, Cell Differentiation, Stem Cell Transplantation/methods, Mice, Dopamine/metabolism, Disease Models, Animal, Astrocytes/metabolism
- in
- Science Advances
- volume
- 10
- issue
- 42
- article number
- eadn3057
- pages
- 1 - 14
- publisher
- American Association for the Advancement of Science (AAAS)
- external identifiers
-
- scopus:85206844329
- pmid:39423273
- ISSN
- 2375-2548
- DOI
- 10.1126/sciadv.adn3057
- language
- English
- LU publication?
- yes
- id
- 51986f93-4869-461a-87a8-025165367951
- date added to LUP
- 2024-12-09 11:04:00
- date last changed
- 2025-07-23 11:01:40
@article{51986f93-4869-461a-87a8-025165367951, abstract = {{<p>Stem cell therapies for Parkinson's disease are at an exciting time of development, and several clinical trials have recently been initiated. Human pluripotent stem cells are differentiated into transplantable dopamine (DA) progenitors which are proliferative at the time of grafting and undergo terminal differentiation and maturation in vivo. While the progenitors are homogeneous at the time of transplantation, they give rise to heterogeneous grafts composed not only of therapeutic DA neurons but also of other mature cell types. The mechanisms for graft diversification are unclear. We used single-nucleus RNA-seq and ATAC-seq to profile DA progenitors before transplantation combined with molecular barcode-based tracing to determine origin and shared lineages of the mature cell types in the grafts. Our data demonstrate that astrocytes, vascular leptomeningeal cells, and DA neurons are the main component of the DAergic grafts, originating from a common progenitor that is tripotent at the time of transplantation.</p>}}, author = {{Storm, Petter and Zhang, Yu and Nilsson, Fredrik and Fiorenzano, Alessandro and Krausse, Niklas and Åkerblom, Malin and Davidsson, Marcus and Yuan, Joan and Kirkeby, Agnete and Björklund, Tomas and Parmar, Malin}}, issn = {{2375-2548}}, keywords = {{Parkinson Disease/metabolism; Cell Lineage/genetics; Animals; Dopaminergic Neurons/metabolism; Humans; Cell Differentiation; Stem Cell Transplantation/methods; Mice; Dopamine/metabolism; Disease Models, Animal; Astrocytes/metabolism}}, language = {{eng}}, month = {{10}}, number = {{42}}, pages = {{1--14}}, publisher = {{American Association for the Advancement of Science (AAAS)}}, series = {{Science Advances}}, title = {{Lineage tracing of stem cell-derived dopamine grafts in a Parkinson's model reveals shared origin of all graft-derived cells}}, url = {{http://dx.doi.org/10.1126/sciadv.adn3057}}, doi = {{10.1126/sciadv.adn3057}}, volume = {{10}}, year = {{2024}}, }