EconPapers    
Economics at your fingertips  
 

A tissue-bioengineering strategy for modeling rare human kidney diseases in vivo

J. O. R. Hernandez, X. Wang, M. Vazquez-Segoviano, M. Lopez-Marfil, M. F. Sobral-Reyes, A. Moran-Horowich, M. Sundberg, D. O. Lopez-Cantu, C. K. Probst, G. U. Ruiz-Esparza, K. Giannikou, R. Abdi, E. P. Henske, D. J. Kwiatkowski, M. Sahin and D. R. Lemos ()
Additional contact information
J. O. R. Hernandez: Brigham and Women’s Hospital
X. Wang: Brigham and Women’s Hospital
M. Vazquez-Segoviano: Brigham and Women’s Hospital
M. Lopez-Marfil: Brigham and Women’s Hospital
M. F. Sobral-Reyes: Brigham and Women’s Hospital
A. Moran-Horowich: Brigham and Women’s Hospital
M. Sundberg: Boston Children’s Hospital
D. O. Lopez-Cantu: Brigham and Women’s Hospital
C. K. Probst: Brigham and Women’s Hospital
G. U. Ruiz-Esparza: Brigham and Women’s Hospital
K. Giannikou: Harvard Medical School
R. Abdi: Harvard Medical School
E. P. Henske: Harvard Medical School
D. J. Kwiatkowski: Harvard Medical School
M. Sahin: Boston Children’s Hospital
D. R. Lemos: Brigham and Women’s Hospital

Nature Communications, 2021, vol. 12, issue 1, 1-16

Abstract: Abstract The lack of animal models for some human diseases precludes our understanding of disease mechanisms and our ability to test prospective therapies in vivo. Generation of kidney organoids from Tuberous Sclerosis Complex (TSC) patient-derived-hiPSCs allows us to recapitulate a rare kidney tumor called angiomyolipoma (AML). Organoids derived from TSC2−/− hiPSCs but not from isogenic TSC2+/− or TSC2+/+ hiPSCs share a common transcriptional signature and a myomelanocytic cell phenotype with kidney AMLs, and develop epithelial cysts, replicating two major TSC-associated kidney lesions driven by genetic mechanisms that cannot be consistently recapitulated with transgenic mice. Transplantation of multiple TSC2−/− renal organoids into the kidneys of immunodeficient rats allows us to model AML in vivo for the study of tumor mechanisms, and to test the efficacy of rapamycin-loaded nanoparticles as an approach to rapidly ablate AMLs. Collectively, our experimental approaches represent an innovative and scalable tissue-bioengineering strategy for modeling rare kidney disease in vivo.

Date: 2021
References: View references in EconPapers View complete reference list from CitEc
Citations:

Downloads: (external link)
https://www.nature.com/articles/s41467-021-26596-y Abstract (text/html)

Related works:
This item may be available elsewhere in EconPapers: Search for items with the same title.

Export reference: BibTeX RIS (EndNote, ProCite, RefMan) HTML/Text

Persistent link: https://EconPapers.repec.org/RePEc:nat:natcom:v:12:y:2021:i:1:d:10.1038_s41467-021-26596-y

Ordering information: This journal article can be ordered from
https://www.nature.com/ncomms/

DOI: 10.1038/s41467-021-26596-y

Access Statistics for this article

Nature Communications is currently edited by Nathalie Le Bot, Enda Bergin and Fiona Gillespie

More articles in Nature Communications from Nature
Bibliographic data for series maintained by Sonal Shukla () and Springer Nature Abstracting and Indexing ().

 
Page updated 2025-03-19
Handle: RePEc:nat:natcom:v:12:y:2021:i:1:d:10.1038_s41467-021-26596-y