Rita Perlingeiro, PhD

Our laboratory has a long-term interest in understanding the molecular mechanisms controlling lineage-specific differentiation of pluripotent stem cells (i.e. embryonic and adult reprogrammed stem cells), and applying this information to efficiently generate tissue-specific stem/progenitor cells endowed with in vivo regenerative potential. Our ultimate goal is to develop safe strategies to enable their future therapeutic application.

• Department of Medicine Award for Research Excellence, University of Minnesota (2017)
• Lillehei Professorship in Stem Cell and Regenerative Cardiovascular Medicine (2014)
• National Scientist Development Award from AHA (2005)

Committees:

• Chair, Musculo-Skeletal Gene & Cell Therapy Committee – ASGCT (2020-2023)
• Member of the Musculo-Skeletal Gene & Cell Therapy Committee – ASGCT (2014-2023)
• Skills and Development Coordinator for NHLBI Progenitor Cell Biology Consortium (2013 - 2017)

Editorial Board:

• Skeletal Muscle, Journal of Stem Cell Research and Therapy, and Stem Cell Reviews and Reports

Research Projects

• Transcriptional mechanisms and signaling pathways controlling mesodermal cell fate
• Strategies to enable translational application of iPS cells to treat muscular dystrophies
• Genetic correction of muscular dystrophy patient-specific iPS cells
• Disease modeling of muscular dystrophy using patient-specific iPS cells

NIH-NIAMS (R01AR071439-01)     04/01/17 – 03/31/22
“Targeting Dystroglycanopathies using Pluripotent-derived Myogenic Progenitors”

Duchenne UK 2019-08     09/15/19 – 09/14/21
“Preclinical Studies in Pluripotent Stem Cell-Derived Myogenic Progenitors to Enable a First-in-Human Phase 1 Safety/Dose Escalation Trial for Duchenne Muscular Dystrophy”

DMDRP/DOD MD160035     09/30/17 – 09/29/20
“Scalability and Safety Studies in Clinical Grade Pluripotent-Derived Myogenic Progenitors for Therapeutic Application in DMD”

FSHD Society FSHS-22018-01     12/01/18 – 11/30/20
“Determining the therapeutic potential of pluripotent stem cell-derived myogenic progenitors in the iDUX4pA mouse model”

T32 GM113846     07/01/15- 06/30/20
“NIH Stem Cell Biology Training Program”

Full list of publications at Experts@Minnesota.

1. Incitti T, Magli A, Jenkins A, Lin K, Yamamoto A, Perlingeiro RCR (2020). Pluripotent stem cell-derived skeletal muscle fibers preferentially express myosin heavy-chain isoforms associated with slow and oxidative muscles. Skeletal Muscle. Jun 3;10(1):17. PCMID: PMC7268645.
2. Azzag K, Ortiz-Cordero C, Oliveira NAJ, Magli A, Selvaraj S, Tungtur S, Upchurch W, Iaizzo PA, Lu QL, Perlingeiro RCR (2020). Efficient engraftment of pluripotent stem cell-derived myogenic progenitors in a novel immunodeficient mouse model of limb girdle muscular dystrophy 2I. Skeletal Muscle. Apr 22;10(1):10. PCMID: PMC7175515.
3. Selvaraj S, Mondragon-Gonzalez R, Xu B, Magli A, Kim H, Lainé J, Kiley J, McKee H, Rinaldi F, Aho J, Tabti N, Shen W, Perlingeiro RCR (2019) Screening identifies small molecules that enhance the maturation of human pluripotent stem cell-derived myotubes. eLIFE, 8. pii: e47970. PMCID: PMC6845233.
4. Selvaraj S, Dhoke N, Kiley J, Aierdi AJM, Mondragon-Gonzalez R, Killeen G, Oliveira VKP, Tungtur S, Munain AL, Perlingeiro RCR (2019) Gene correction of Limb Girdle Muscular Dystrophy type 2A patient-specific iPS cells for the development of targeted autologous cell therapy. Molecular Therapy, 27:2147-2157. PMCID: PMC6904833.
5. Xu B, Zhang M, Perlingeiro RCR, Shen W. (2019) Skeletal Muscle Constructs Engineered from Human Embryonic Stem Cell Derived Myogenic Progenitors Exhibit Enhanced Contractile Forces When Differentiated in a Medium Containing EGM-2 Supplements. Advanced Biosystems 2019 Dec;3(12):e1900005. doi: 10.1002/adbi.201900005. Epub 2019 Nov 4. PMID: 32648685.
6. Mondragon-Gonzalez R, Azzag K, Selvaraj S, Yamamoto A, Perlingeiro RCR (2019) Transplantation studies reveal internuclear transfer of toxic RNA in engrafted muscles of myotonic dystrophy 1 mice. eBioMedicine. 2019:47:553-562. PMCID: PMC6796515
7. Magli A, Baik J, Pota P, Ortiz Cordero C, Kwak IY, Garry DJ, Love PE, Dynlacht BD, Perlingeiro RCR (2019) Pax3 cooperates with Ldb1 to direct local chromosome architecture during myogenic lineage specification. Nature Communications, 10:2316. PMCID: PMC6534668.
8. Magli A, Baik J, Kwak IY, Stafford D, Swanson SA, Stewart R, Thomson JA, Garry DJ, Perlingeiro RCR (2019) Time-dependent Pax3-mediated chromatin remodeling and cooperation with Six4 and Tead2 specify the skeletal myogenic lineage in developing mesoderm. PLOS Biology, 17(2): e3000153. PMCID: PMC6534668.
9. Incitti T, Magli A, Darabi R, Arpke RJ, Kyba M, Perlingeiro RCR (2019) Pluripotent stem cell-derived myogenic progenitors remodel their molecular signature upon in vivo engraftment. PNAS, Mar 2019, 116 (10) 4346-4351; DOI: 10.1073/pnas.1808303116.
10. Borges L, Oliveira VKP, Baik J, Bendall SC, Perlingeiro RCR (2018) Serial transplantation reveals a critical role for endoglin in hematopoietic stem cell quiescence. Blood, 133:688-696; doi: https://doi.org/10.1182/blood-2018-09-874677.
11. Mondragon-Gonzalez R, Perlingeiro RCR (2018) Recapitulating muscle disease phenotypes with myotonic dystrophy 1 iPS cells: a tool for disease modeling and drug discovery. Disease Models & Mechanisms. 2018 Jun 13. pii: dmm.034728. doi: 10.1242/dmm.034728. [Epub ahead of print]
12. Magli A, Perlingeiro RCR (2017) Myogenic Progenitor Specification from Pluripotent Stem Cells. Invited Review for Seminars in Cell and Developmental Biology, 2017:72:87-98.
13. Kim, J, Oliveira VKP, Yamamoto A, Perlingeiro RCR (2017) Generation of Skeletal Myogenic Progenitors from Human Pluripotent Stem Cells Using Non-Viral Delivery of Minicircle DNA. Stem Cell Research 23:87-94. PMCID: PMC5582001.
14. Magli A, Incitti T, Kiley J, Swanson SA, Darabi R, Rinaldi F, Selvaraj S, Yamamoto A, Tolar J, Yuan C, Stewart R, Thomson JA, Perlingeiro RCR (2017) PAX7 Targets, CD54, Integrin ?9?1, and SDC2, Allow Isolation of Human ESC/iPSC-Derived Myogenic Progenitors. Cell Reports 19:2867-2877. PMID: 28658631.
15. Kim J, Magli A, Chan SSK, Oliveira VKP, Wu J, Darabi R, Kyba M, Perlingeiro RCR (2017) Expansion and Purification Are Critical for the Therapeutic Application of Pluripotent Stem Cell-Derived Myogenic Progrenitors. Stem Cell Reports 9:12-22. PMID: 28528701.
16. Dourado KC, Baik J, Oliveira VKP, Beltrame M, Yamamoto A, Theuer, CP, Figueiredo CAV, Verneris MR, Perlingeiro RCR (2017) Endoglin: a Novel Target for Therapeutic Intervention in Acute Leukemias Revealed in Xenograft Mouse Models. Blood 129:2526-2536. PMID: 28351936.
17. Baik J, Magli A, Tahara N, Swanson SA, Borges L, Koyano-Nakagawa N, Stewart R, Garry DG, Kawakami Y, Thomson JA, Perlingeiro RCR (2016) Endoglin Integrates BMP and Wnt Signaling to Induce Hematopoiesis through JDP2. Nature Communications 7:13101. PMID: 27713415.
18. Carrió E, Magli A, Muñoz M, Peinado MA, Perlingeiro RCR, Suelves M (2016) Muscle cell identity requires Pax7-mediated lineage-specific DNA demethylation. BMC Biology 14:30. PMID: 27075038.
19. Filareto A, Rinaldi F, Arpke RW, Darabi R, Belanto JJ, Toso EA, Miller AZ, Ervasti JM, McIvor RS, Kyba M, Perlingeiro RCR (2015) Pax3-induced expansion enables the genetic correction of dystrophic satellite cells. Skeletal Muscle 5:36. PMID: 26504514.
20. McCullagh KJA, Perlingeiro RCR (2015) Coaxing stem cells for skeletal muscle repair. Advanced Drug Delivery Reviews 84:198-207. PMID: 25049085.

Full list of publications at Experts@Minnesota