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.

• Lillehei Professorship in Stem Cell and Regenerative Cardiovascular Medicine (2014)
• National Scientist Development Award from AHA (2005)
• Postdoctoral Fellowship from FAPESP, Government of Sao Paulo, Brazil (1999)
• FAPESP Summer Research Award (1996)
• PhD Fellowship from FAPESP, Government of Sao Paulo, Brazil
• PhD Fellowship from CAPES, Government of Brazil (declined) (1993)
• Master’s Fellowship from FAPESP, Government of Sao Paulo, Brazil (1991)

Committees:

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

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 disease- and patient-specific iPS cells
• Dissecting the mechanisms associated with hematopoietic stem cell self-renewal in homeostasis and leukemia

1. Targeting Dystroglycanopathies using Pluripotent-derived Myogenic Progenitors, R01 AR071439-01, National Institutes of Health - NIAMS, Principal Investigator, 4/01/2017 – 03/31/22
2. Skeletal Muscle Regeneration from Differentiating Pluripotent Stem Cells, R01 AR055299, National Institutes of Health – NIAMS, Principal Investigator, 07/01/07 - 08/31/19
3. Gene editing of Calpain 3 in LGMD2A iPS cells, C3 Coallition Grant, Principal Investigator, 08/01/17 – 07/31/18
4. Scalability and Safety Studies in Clinical Grade Pluripotent-Derived Myogenic Progenitors for Therapeutic Application in DMD, MD160035, DMDRP/DOD, Principal Investigator, 09/01/17-08/31/20

• 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.
• 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.
• 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.
• 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.
• 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 Communications7:13101. PMID: 27713415.
• 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.
• 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.
• McCullagh KJA & Perlingeiro RCR, (2015) "Coaxing stem cells for skeletal muscle repair." Advanced Drug Delivery Reviews 84:198-207. PMID: 25049085.
• Skoglund G, Laine J, Darabi R, Fournier E, Perlingeiro RCR, Tabti N, (2014) Physiological and ultrastructural features of human induced pluripotent and embryonic stem cell-derived skeletal myocytes in vitro. Proc Natl Acad Sci USA 111:8275-8280.
• Magli A, Schnettler E, Swanson SA, Borges L, Hoffman K, Stewart R, Thomson JA, Perlingeiro RCR, (2014) Pax3 and Tbx5 specify whether PDGFR?+ cells assume skeletal or cardiac muscle fate in differentiating ES cells. Stem Cells 32:2072-2083. PMID: 24677751.
• Filareto A, Parker S, Darabi R, Borges L, Iacovino M, Schaaf T, Mayerhofer T, Chamberlain JS, Ervasti JM, McIvor RS, Kyba M, Perlingeiro RCR, (2013) An Ex Vivo Gene Therapy Approach to Treat Muscular Dystrophy Using Inducible Pluripotent Stem Cells. Nat Communication 4:1549. PMID: 23462992.
• Magli A, Schnettler E, Rinaldi F, Brember P, Perlingeiro RCR, (2013) Functional Dissection of Pax3 in Paraxial Mesoderm Development and Myogenesis. Stem Cells 31(1):59-60. PMID: 23081715.
• Borges L, Iacovino M, Mayerhofer T, Koyano-Nakagawa N, Baik J, Garry DJ, Kyba M, Letarte M, Perlingeiro RCR, (2012) A Critical Role for Endoglin in the Emergence of Blood During Embryonic Development. Blood 119(23):5417-5428. PMID: 22535663.
• Darabi R, Arpke RW, Irion S, Dimos JT, Grskovic M, Kyba M, Perlingeiro RCR, (2012) Human ES- and iPS-Derived Myogenic Progenitors Restore DYSTROPHIN and Improve Contractility upon Transplantation in Dystrophic Mice. Cell Stem Cell 10(5):610-9. PMID:22560081.
• Darabi R, Santos FNC, Filareto A, Pan W, Koene R, Rudnicki M, Kyba M, Perlingeiro RCR, (2011) Assessment of the Myogenic Stem Cell Compartment Following Transplantation of Pax3/Pax7-Induced Embryonic Stem Cell-Derived Progenitor. Stem Cells 29:777-790. PMID: 21374762.
• Perlingeiro RCR, (2007) Endoglin is required for hemangioblast and hematopoietic development. Development 134:3041-3048. PMID: 17634194.