Perlingeiro Lab

Rita Perlingeiro

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.

Rita Perlingeiro faculty profile

Perlingeiro lab members standing outside lab building.
Media

"FDA Clears IND Application for Myogenica’s MyoPAXon in Muscular Dystrophy" (NeurologyLive)
"FDA Clears Investigational New Drug application for muscular dystrophy treatment from University of Minnesota startup Myogenica" (PR Newswire)
"NIH Awards $2.5m R01 Grant to Perlingeiro" (UMN Med School)
"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"

Webinars, Podcasts, and Other Streaming Talks

Feb. 2023: Pluripotent Stem Cell Research and FSHD, FSHD Society, Zoom presentation
May 23, 2022: LGMD Global Advocacy Summit, Zoom presentation
April 2021: Muscular Dystrophy Research Day, Minneapolis, MN, Zoom presentation
Feb 21, 2021: “iPSC-Derived Myogenic Progenitors” (Culturing knowledge in stem cell research; Ep 141 Featuring Dr. Rita Perlingeiro)
Dec 21, 2020: Webinar on Muscle Regeneration, French FSHD Society, Zoom presentation

Current Lab Projects
  • Dissecting the molecular mechanisms controlling in vitro and in vivo maturation of PSC-derivatives
  • Understanding how the muscle environment influences the engraftment of myogenic progenitors
  • Preclinical studies of PSC-derived myogenic progenitors to enable clinical translation
  • Disease modeling of DMD and BMD using patient-specific iPSC-derived skeletal and cardiac muscle cells
  • Understanding the impact of HLA mismatch on muscle engraftment
Selected Publications

Full list of publications: go to Experts@Minnesota or PubMed.

  1. Azzag K & Perlingeiro RCR, (2026) "Progress on cell therapy for skeletal muscle disorders”. Advanced Drug Delivery Reviews, 233:115859. PMID: 41871765. Go to article
  2. Yamashita AMS, Crist SB, Garay BI, Kim H, Azzag K, Bosnakovski D, Abrahante JE, Abreu P, Ahlquist A, & Perlingeiro RCR, (2026) “Necessity of Notch3 signaling in myofiber maturation in a pluripotent stem cell transplant model”. Skeletal Muscle, 16(1):1-15. PMCID: PMC12771941. Go to article
  3. Azzag K, Magli A, Kiley J, Sumstad D, Kadidlo D, Crist SB, Norris B, Ahlquist A, Stone LLH, Everett J, Faustich JS, Seelig D, Rangarajan P, Kim H, Flory C, Bushman F, Ramachandran S, Kang PB, Schumacher RJ, Wagner JE, Kyba M, Graham ME, McKenna, DH & Perlingeiro RCR, (2025) “Preclinical quality, safety, and efficacy of cGMP MyoPAXon, an iPSC-derived myogenic progenitor product for the treatment of muscular dystrophies”. Molecular Therapy, 33(10):5099-5117, 2025. PMCID: PMC12338975. Go to article
  4. Kalita B, Martinez-Cebrian G, McEvoy J, Allensworth M, Knight M, Magli A, Perlingeiro RCR, Dyer MA, Stewart E, & Dynlacht BD, (2025) “PAX translocations remodel mitochondrial metabolism through altered leucine usage in rhabdomyosarcoma”. Cell, 188:1-21. Go to article
  5. Yamashita AMS, Garay BI, Kim H, Bosnakovski D, Abrahante JE, Azzag K, Abreu P, Ahlquist A, Perlingeiro RCR. Effect of Notch1 signaling on muscle engraftment and maturation from pluripotent stem cells. Stem Cell Reports. 2025 Feb 11;20(2):102396. doi: 10.1016/j.stemcr.2024.102396. Epub 2025 Jan 30. PMID: 39889709. Go to article6
  6. Dhoke N, Kim H, Azzag K, Crist SB, Kiley J, & Perlingeiro RCR, (2024) A novel CRISPR-Cas9 strategy to target DYSTROPHIN mutations downstream of exon 44 in patient-specific DMD iPS cells. 2024 Jun 4;13(11):972. Cells. Go to article
  7. Crist SB, Azzag K, Kiley J, Coleman I, Nelson PS, Magli A, & Perlingeiro RCR, (2024) The adult environment promotes the transcriptional maturation of human iPSC-derived muscle grafts. NPG Regenerative Medicine. 2024 Apr 4;9(1):16. Go to article
  8. Azzag K, Gransee HM, Magli A, Yamashita AMS, Tungtur S, Ahlquist A, Zhan W-Z, Onyebu C, Greising SM, Mantilla CB, & Perlingeiro RCR, (2024) Enhanced diaphragm muscle function upon satellite cell transplantation in dystrophic mice. International Journal of Molecular Sciences. 2024 Feb 21;25(5):2503. Go to article
  9. Abreu P, Garay BI, Nemkov T, Yamashita AMS, & Perlingeiro RCR, (2024) Metabolic changes during in vivo maturation of PSC-derived skeletal myogenic progenitors. Cells. 2023 Dec 29;13(1):76. Go to article
  10. Baik J, Ortiz-Cordero C, Magli A, Azzag K, Crist SB, Yamashita A, Kiley J, Selvaraj S, Mondragon-Gonzalez R. Perrin E, Maufort JP, Janecek JL, Lee RM, Stone LH, Rangarajan P, Ramachandran S, Graham ML, & Perlingeiro RCR. (2023). Establishment of skeletal myogenic progenitors from non-human primate induced pluripotent stem cells. Cells, 12(8), 1147; Go to article.
  11. Singh BN, Yucel D, Garay BI, Tolkacheva EG, Kyba M, Perlingeiro RCR, van Berlo J, & Ogle BM, (2023) Proliferation and Maturation: Janus and the art of engineered cardiac tissue. Circulation Research, 132(4):519-540. PMCID: PMC9943541Go to article.
  12. McKenna DH & Perlingeiro RCR, (2023) Development of allogeneic iPS cell-based therapy: from bench to bedside. EMBO Molecular Medicine, 15(2):e15315. PMCID: PMC9906386Go to article.
  13. Azzag K, Bosnakovski D, Tungtur S, Salama P, Kyba M, & Perlingeiro RCR, (2022) Transplantation of pluripotent stem cell-derived myogenic progenitors counteracts disease phenotypes in a mouse model of FSHD. NPG Regenerative Medicine,  7(1):43. PMCID: PMC9440030 Go to article.
  14. Garay BI, Givens S, Stanis N, Magli A, Yücel D, Abrahante JE, Goloviznina NA, Soliman HAN, Dhoke NR, Baik J, Kyba M, van Berlo JH, Ogle B, & Perlingeiro RCR, (2022) Inhibition of mitogen-activated protein kinase pathway enhances maturation of human iPSC-derived cardiomyocytes. Stem Cell Reports. 17(9):2005-2022. PMCID:PMC9481895. Go to article
  15. Kim H, & Perlingeiro RCR, (2022) Generation of human myogenic progenitors from pluripotent stem cells for in vivo regeneration. Cellular and Molecular Life Sciences. 8;79(8):406. doi: 10.1007/s00018-022-04434-8. PMCID: PMC9270264.
  16. Ortiz-Cordero C, Bincoletto, C, Dhoke N, Selvaraj S, Magli A, Zhou H, Kim D-H, Bang AG, & Perlingeiro RCR, (2021), Defective autophagy and increased apoptosis contribute toward the pathogenesis of FKRP-associated muscular dystrophies. Stem Cell Reports. 16(11):2752-2767. PMCID: PMC8581053 Go to article.
  17. 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. 36(2):109360. PMCID: PMC8327854.Go to article
  18. 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: PMC8581053 Go to article
  19. Ortiz-Cordero C, Azzag K, & Perlingeiro RCR, (2021), “Fukutin-Related Protein: from Pathology to Treatments”. Trends in Cell Biology, 31:197-210. PMID: 33272829 Go to article (cover article).
  20. 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 Go to article
  21. Baik J, 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: PMC7566100Go to article.
  22. 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 Go to article
  23. 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 Go to article.
  24. 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 Go to article.
  25. 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 Go to article.
  26. 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) Go to article.
  27. 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. Go to article
  28. 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 Go to article.

Full list of publications go to: Experts@Minnesota or PubMed.

Lab Members

Jump to Lab Alumni >

Jim

Jim Kiley
Position: Lab Manager, Researcher
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
Contact: [email protected]

Karim

Karim Azzag, Ph.D.     
Position: Researcher
Joined the lab: January 2018
Education: 2011 PhD, cellular biology, Montpellier II University     
Research Interests: Molecular Mechanisms behind Cell Differentiation and iPSC-based cell therapy for muscular dystrophies
Accomplishments:
2022 - Abstract selected for oral presentation for the 2022 Stem Cell Institute Spring Conference: State of the Art Approaches for Cell, Tissue & Organ Transplantation.
2022 - Abstract selected for oral presentation for the 2022 29th annual FSHD society international research congress.     
2022 - Abstract selected for oral presentation for the 2022 FASEB conference: The Skeletal Muscle Stem Cells and Regeneration Conference.
Contact: [email protected]

Aline

Aline Miyoko Sakaguchi Yamashita, Ph.D.
Position: Researcher
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
Accomplishments:
2023 - Best Poster (postdoc category), Greg Marzolf, Jr. Symposium, University of Minnesota, April 1, 2023
2025 - Kathleen Moriarity Trainee Travel Award, Greg Marzolf Jr. Foundation, April 17,2025
Contact: [email protected]

Sarah Crist

Sarah Crist, Ph.D.
Position: Post-Doctoral Fellow
Joined the Lab: November 2021
Education:
2021 Ph.D. – Molecular and Cellular Biology – University of Washington/Fred Hutchinson CRC     
2013 B.S. – Biology – Haverford College
Research Interests: Exploring the active role of the skeletal muscle microenvironment on iPax3 myogenic progenitor maturation, with particular interest in both endogenous extracellular matrix constituents and novel deposition by the transplanted myogenic progenitors.
Accomplishments:
2022 - Harold M. Weintraub Graduate Student Award
2022 - Recipient. T32 Training Program in Cardiac Innovation, NHLBI
2022 - University of Washington Graduate School’s 2022 Distinguished Dissertation Award (category: Biological Sciences)
Contact: [email protected]

Aaron

Aaron Ahlquist
Position: PhD Student
Joined the lab: October 2021
Education: 2017 BS, Biology, University of Minnesota
Research Interests: Muscle regeneration and the effect of the environment
Accomplishments: 
2026 - Best Graduate Student Poster, Marzolf Symposium, April 28
Contact: [email protected]

Josh Lihnakevic

Joshua Lihnakevic
Position: Researcher 1
Joined the Lab: July 2024
Education: 2021 BS – Biology – Concordia University, St. Paul, MN
Contact: [email protected]

Dr. Minatel

Elaine Minatel, Ph.D.
Position: Visiting Professor
Joined the lab: August 2025
Education: 2003 PhD - Cell and Structural Biology - University of Campinas (UNICAMP) Brazil
Research Interests: To develop strategies to enhance the maturation of iPSC-derived myotubes
Accomplishments:
2023- Frontiers in Myogenesis Conference. Poster: LED therapy reduces oxidative stress in dystrophin-deficient muscle cells targeting mitochondrial signaling pathways.
2022- Academic Recognition Award “Zeferino Vaz,” UNICAMP.
Contact:[email protected]

Dr. dos Reis Santos

Audrei dos Reis Santos, Ph.D.
Position:Visiting Post-Doctoral Fellow
Joined the Lab: September 2025
Education:
2024 Ph.D. - Morphofunctional Biology - University of Sao Paulo, USP - Brazil
2013 - MS - Biodynamic Studies of Physical Education and Sport - University of Sao Paulo, USP - Brazil
2008 - B.S. - Physical Activity Sciences - University of Sao Paulo, USP - Brazil
Research Interests: The morphofunctional and molecular adaptations induced by physical training in skeletal muscle during aging and in dystrophic diseases.
Accomplishments:
2024 - Second place for best doctoral work, IV Symposium of the Graduate Program in Systems Biology, ICB-USP.
2022 - Honorable mention, II Congress of Nutrition and Health.
2009 - Best student of the undergraduate course in Physical Activity Sciences, University of São Paulo.
Contact:[email protected]

Sutra Iyer

Sutra Iyer
Position: Undergraduate
Joined the lab:February, 2025
Education: Expected Gradation Date May, 2028: B.S., Biochemistry & Psychology, University of Minnesota
Research Interests: Investigating iPSC-based therapy and the pathogenesis of muscular dystrophies; supporting translational and regenerative biology research
Contact:  [email protected]

Ethan Murphy

Ethan Murphy
Position: Undergraduate
Joined the lab:February, 2026
Education: Expected Graduation Date May, 2027: B.S., Neuroscience & Genetics, Cell Biology, and Development, University of Minnesota
Research Interests: Applying bioinformatics to study the molecular mechanisms of aging-related diseases, particularly in muscle and regenerative biology, with the goal of supporting translational research.
Contact:  [email protected]

Henry

Henry Xiong
Position: Undergraduate
Joined the lab: February, 2026
Education: Expected Graduation Date May 2027 - B.S., Biology, University of Minnesota
Research Interests: iPSC-based cell therapy in regenerative medicine
Contact:  [email protected]

Kendall

Kendall Ziegler
Position: Undergraduate
Joined the lab: September 9, 2022
Education: Expected Graduation Date May 2026: B.S., Microbiology, University of Minnesota
Research Interests: Application of genome editing with CRISPR/Cas9     
Contact: [email protected]

Lab Life

Position Available: NIH-funded PostDoctoral Researcher

Skeletal muscle from iPSCs

NIH-funded postdoctoral researcher position is available in the laboratory of Dr. Rita Perlingeiro at the Lillehei Heart Institute, University of Minnesota, to study the mechanisms regulating the engraftment and maturation of pluripotent stem cell-derived myogenic progenitors. Preferred applicants will have published experience in bioinformatics, muscle biology, and pluripotent stem cells. Candidates should send CV and three letters of reference to Dr. Rita Perlingeiro at [email protected].