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.
- Current Lab Projects
- Selected Publications
- Lab Members
- Lab Life
"Creating New Muscle by Using Stem Cells" (Duchenne UK)
"UMN Researchers Discover How Three-Dimensional Organization of the Genome Regulates Cell Differentiation" (UMN Med School)
"UMN Medical School Study Provides New Insight Into the Use of Cell Replacement Therapies to Treat Muscular Dystrophies" (UMN Med School)
"Feature Researcher: Rita Perlingeiro" (Stem Cell Institute)
"U Of M Makes Muscular Dystrophy Breakthrough" (WCCO video)
"U researchers use stem cells to regenerate muscles in diseased mice" (MN Public Radio)
"U of M researchers develop new muscular dystrophy treatment approach using human stem cells" (Science NewsLine)
"U of M researchers utilize genetically corrected stem cells to spark muscle regeneration"
Current Lab 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 stem cell self-renewal and long-term engraftment
Full list of publications at Experts@Minnesota.
- Dhoke N, Kim H, Selvaraj S, Oliveira NAJ, Azzag K, Tungtur S, Ortiz-Cordero C, Kiley J, Lu QL, Bang A, & Perlingeiro RCR, (2021), “A universal gene correction approach for FKRP-associated dystroglycanopathies to enable autologous cell therapy”. Cell Reports. (in press).
- Ortiz-Cordero C, Magli A, Dhoke N, Kuebler T, Selvaraj S, Oliveira NA, Zhou H, Sham YY, Bang AG, & Perlingeiro RCR, (2021), “NAD+ enhances ribitol and ribose rescue of α-dystroglycan functional glycosylation in human FKRP-mutant myotubes”. Elife, 2021 10:e65443. PMCID: PMC7924940
- Ortiz-Cordero C, Azzag K, & Perlingeiro RCR, (2021), “Fukutin-Related Protein: from Pathology to Treatments”. Trends in Cell Biology, 31:197-210. PMID: 33272829 (cover article).
- Kim H, Selvaraj S, Kiley J, Azzag K, Garay BI, & Perlingeiro RCR, (2021), “Genomic safe harbor expression of PAX7 for the generation of engraftable myogenic progenitors”. Stem Cell Reports, 16:10-19.PMCID: PMC7815936
- BaikJ, Felices M, Yingst A, Theuer, CP, Verneris MR, Miller JS, & Perlingeiro RCR,(2020), “Therapeutic effect of TRC105 and decitabine combination in AML xenografts”. Heliyon, 6(10):e05242. PMCID: PMC7566100.
- Incitti T, Magli A, Jenkins J, Lin K, Yamamoto A and Perlingeiro RCR, (2020), “Pluripotent stem cell-derived skeletal muscle fibers preferentially express oxidative myosin heavy-chain isoforms: new implications for Duchenne Muscular Dystrophy”. Skeletal Muscle, 10(1):17. PMCID: PMC7268645
- Azzag K, Ortiz-Cordero C, Oliveira NAJ, Magli A, Selvaraj S, Tungtur S, Upchurch W, Iaizzo PA, Lu QL and 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, 10(1):10. PMCID: PMC7175515.
- 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.
- 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.
- Selvaraj S, Kyba M & Perlingeiro RCR, (2019) “Pluripotent Stem Cell-Based Therapeutics for Muscular Dystrophies” Trends in Molecular Medicine. 25:803-816. PMCID: PMC6721995. (cover article)
- 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. 47:553-562. PMCID: PMC6796515.
- Magli A, Baik J, Pota P, Ortiz Cordero C, Kwak IY, Garry DJ, Love PE, Dynlacht BD and Perlingeiro RCR, (2019) “Pax3 cooperates with Ldb1 to direct local chromosome architecture during myogenic lineage specification”. Nature Communications, 10:2316. PMCID: PMC6534668.
Full list of publications at Experts@Minnesota
Alessandro Magli, Ph.D.
Position: Assistant Professor of Medicine
Joined the Lab: April 2009
Education: 2008 PhD - Molecular & Regenerative Medicine - University of Modena and Reggio Emilia (Italy)
Research Interests: Molecular Mechanisms behind Cell Differentiation, specifically Various Transcription Factor Contribution to Cell Fate Determination
- 2015 - Recipient of the Regenerative Medicine Minnesota Educational Grant
- 2010 - FASEB Summer Research Conferences - Skeletal Muscle Satellite and Stem Cells. July 18 – 23 - Carefree, AZ, USA. Magli A. (oral presentation). "Molecular analysis of Pax3-dependent gene regulation in the embryonic skeletal myogenic program."
- 2010 - Visscher symposium – June 3rd, Minneapolis (MN) USA Magli A (oral presentation). "Insights on mesoderm formation: a new role for Pax3."
Karim Azzag, Ph.D.
Position: Post-Doctoral Fellow
Joined the lab: January 2018
Education: 2017 PhD, cellular biology, Montpellier II University
Research Interests: Molecular Mechanisms behind Cell Differentiation and iPSC-based cell therapy for muscular dystrophies
June Baik, Ph.D.
Position: Post-Doctoral Fellow
Joined the Lab: November 2007
Education: 2015 Ph.D. – Molecular, Cellular, Developmental Biology and Genetics – University of Minnesota
Research Interests: The role of endoglin in leukemia
- 2021 Recipient, T32 Training Program in Cardiac Innovation, NIH Heart, Lung, and Blood Institute (NHLBI), United States
- 2017-2019 Recipient, Hartwell Foundation Fellowship, Hartwell Foundation, United states
- 2017 Recipient, Hematology Research Training Program (2T32HL007062), NIH Heart, Lung, and Blood Institute (NHLBI), United States
- 2014-2016 Recipient, PCBC Skills and Development Trainee Award (UMN Hub), and PCBC Jump Start Grant, NIH Heart, Lung, and Blood Institute (NHLBI), United States
- 2014-2015 Recipient, Doctoral Dissertation Fellowship, University of Minnesota, United States
- 2013-2015 Recipient, MN Muscle Training Grant (5T32AR007612-12), National Institutes of Health (NIH), United State
Neha Dhoke, Ph.D.
Position: Post-Doctoral Associate
Joined the Lab: December 2017
Education: PhD 2017 Stem Cell Biology and Regenerative Medicine, CSIR-Indian Institute of Chemical Technology
Research Interests: Targeting dystroglycanopathies using pluripotent stem cell-derived myogeneic progenitors
Accomplishments: Abstract for oral presentation accepted for 2020 annual meeting of the American Society for Gene and Cell Therapy
Aline Miyoko Sakaguchi Yamashita, Ph.D.
Position: Post-Doctoral Associate
Joined the Lab: February 2021
Education: 2019 PhD Biological Chemistry, Federal University of Rio de Janeiro
Research Interests: Molecular mechanisms during maturation of Pluripotent Stem Cell derived myogenic progenitors
Position: Lab Manager, Researcher 2
Joined the Lab: April 2013
Education: 2004 BS – Biology – University of Minnesota
Research Interests: Cardiology and Muscular Dystrophy, previous research included vascular biology and sickle cell anemia
Position: Researcher 1
Joined the lab: March 2018
Education: M.S., Biology and Computer Science, Bowling Green State University
Research Interests: In vivo studies of Muscular Dystrophies
Position: M.D.-Ph.D. Student (MSTP)
Joined the Lab: July 2018
Education: University of California, Berkeley, B.A. Molecular and Cell Biology-Neurobiology
Research Interests: My primary scientific interest is to understand the mechanisms driving cellular degeneration with an emphasis on early detection, rescue, and prevention. One of my goals is to use patient-derived and CRISPR-Cas9-corrected iPSCs in the context of striated muscle differentiation (diseased and normal stage), and the neuromuscular junction (NMJ) as a platform for drug screening, and to unravel biological processes that might not be easily accessible in vivo. My ultimate goal is to develop an in vitro model of Duchenne and Becker muscular dystrophies using hiPSCs: skeletal muscle, motor neurons, and cardiomyocytes.
Ruth L. Kirschstein National Research Service Award F30 Fellowship, NHLBI, March 5, 2020
Sping and Ying Ngoh Lin Award, March 7th, 2020
Carolina Ortiz, M.S.
Position: PhD Candidate in Integrative Biology and Physiology
Joined the Lab: October 2013
Education: B.Sc. Biology, Universidad de Costa Rica, San José, Costa Rica 2012
M.S. in Stem Cell Biology, University of Minnesota, 2015
Candidate to Professional Master's degree in Project Management, Universidad Latina, San José, Costa Rica
Research Interests: In vitro modeling of muscular dystrophies
- 2021 Dr. Marvin and Hadassah Bacaner Research Award, University of Minnesota, Minneapolis, MN
Recognizes excellence and creative basic medical science research in graduating Ph.D. students.
- 2021 Beatrice Z. Milne and Theodore Brandenburg Award, University of Minnesota, Minneapolis, MN
Recognizes exceptional thesis research by graduate students in the basic biomedical sciences.
- 2019 Award for Best Poster, Dept. of Medicine Hebbel Research Day, University of Minnesota, MN, United States
- 2019 Frontiers in Myogenesis Meeting Travel Award, Society for Muscle Biology, Rockville, MD
- 2015-2019 PINN fellowship, Ministry of Science and Technology, Costa Rica
- 2017 I.J. Fox Scholarship Travel Award, School of Medicine, University of Minnesota, Minneapolis, MN
- 2016 Allan Hemingway Scholarship, Integrated Biology and Physiology, University of Minnesota, MN, United States
- 2014 2nd Annual Regional Regeneration Symposium, November 2014, University of Minnesota, MN, United States
Oral presentation: "In vitro modeling of Duchenne Muscular Dystrophy using patient-specific iPS cell-derived skeletal muscle derivatives."
Position: Graduate Student
Joined the Lab: March 2018
Education: BS Biology, University of Wisconsin-Madison
Graduate student in Molecular, Cellular, Developmental Biology and Genetics, University of Minnesota, Expected graduation-2022
Research Interests: In vitro modeling of Duchene and Becker muscular dystrophies using skeletal muscles from patient-derived iPSCs.
Poster Presentation Award in 7th Annual Regional Regeneration Symposium, Fall 2019.
Joined the lab: January 2020
Education: Expected Graduation Date 2022: B.S., Neuroscience and History, University of Minnesota
Research Interests: Pluripotent Stem Cell Derived myogenic progenitors.
Joined the lab: April 2021
Education: Expected Graduation Date 2023: B.S., Neuroscience, University of Minnesota
Research Interests: Neuroscience, Duchenne muscular dystrophy.