iPSc modeling for novel partial lipodystrophy syndromes: platform for disease modeling and precision therapeutics
NIDDK - National Institute of Diabetes and Digestive and Kidney Diseases
About This Grant
Project Summary / Abstract Partial lipodystrophy (PLD) syndromes are characterized by regional loss of white adipose tissue (WAT), leading to insulin resistance, diabetes mellitus, and dyslipidemia. While genetic research has provided significant insights into PLD, over 50% of patients lack a clear genetic diagnosis. The Rare and Atypical Diabetes Network (RADIANT) has identified novel pathogenic variants in key adipose tissue genes among PLD patients. We have demonstrated that modeling PLD using induced pluripotent stem cells (iPSCs), specifically for Familial Partial Lipodystrophy Type 2 (FPLD2) and the LMNA variant p.R482W, can elucidate adipose tissue pathology. However, controversy remains regarding the optimal approach for modeling PLD. One possibility is to reprogram patient-derived peripheral blood mononuclear cells (PBMCs) to iPSCs, correcting genetic variants via CRISPR/Cas9 before differentiating into adipocytes. Another possibility is to introduce multiple variants into a wild-type iPSC line, allowing for consistency and high-throughput testing. This proposal will test and compare these approaches for 5 patients with PLD harboring potentially pathogenic variants. Successful completion will identify new genetic causes of PLD, elucidate PLD pathophysiology, resolve modeling strategy debates, and pave the way for personalized cellular therapies. Additionally, it will establish a platform for screening therapeutic compounds, laying the groundwork for future studies improving the lives of individuals with PLD. Therefore, this proposal directly advances the NIDDK's priorities in understanding the heterogeneity of rare and atypical forms of diabetes.
Grant Summary
iPSc modeling for novel partial lipodystrophy syndromes: platform for disease modeling and precision therapeutics is a NIDDK - National Institute of Diabetes and Digestive and Kidney Diseases grant providing up to $764K for university, nonprofit, healthcare org. Applications are due 2031-04-30 (open). Check eligibility and apply with FindGrants.
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Eligibility
How to Apply
Up to $764K
2031-04-30
- 1Confirm your organization is eligible for iPSc modeling for novel partial lipodystrophy syndromes: platform for disease modeling and precision therapeutics from NIDDK - National Institute of Diabetes and Digestive and Kidney Diseases, checking organization type, location, and any population or project requirements.
- 2Gather the required documents and information, including your organization details, project plan, and budget figures.
- 3Draft your application narrative and budget addressing the funder's priorities and review criteria. FindGrants can draft each section for you to review and edit.
- 4Review every section against the requirements checklist, then export a submission-ready application pack and submit it to NIDDK - National Institute of Diabetes and Digestive and Kidney Diseases before the deadline.
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iPSc modeling for novel partial lipodystrophy syndromes: platform for disease modeling and precision therapeutics: Frequently Asked Questions
Who is eligible for the iPSc modeling for novel partial lipodystrophy syndromes: platform for disease modeling and precision therapeutics?
iPSc modeling for novel partial lipodystrophy syndromes: platform for disease modeling and precision therapeutics is offered by NIDDK - National Institute of Diabetes and Digestive and Kidney Diseases and is generally open to university, nonprofit, healthcare org. It is open to organizations nationwide unless the funder specifies otherwise. Review the specific eligibility terms before applying, since funders set their own requirements around organization type, location, and the population or project being served.
How much funding does the iPSc modeling for novel partial lipodystrophy syndromes: platform for disease modeling and precision therapeutics provide?
iPSc modeling for novel partial lipodystrophy syndromes: platform for disease modeling and precision therapeutics provides up to $764K per award from NIDDK - National Institute of Diabetes and Digestive and Kidney Diseases. Actual award sizes depend on the scope of your project, available program funds, and the number of applicants, so build a budget that reflects realistic, allowable costs rather than the maximum figure.
When is the iPSc modeling for novel partial lipodystrophy syndromes: platform for disease modeling and precision therapeutics deadline?
Applications for iPSc modeling for novel partial lipodystrophy syndromes: platform for disease modeling and precision therapeutics are due 2031-04-30 (open). Because deadlines can change, verify the date with the funder, NIDDK - National Institute of Diabetes and Digestive and Kidney Diseases, and give yourself enough time to prepare a complete, competitive application before the close date.
How do you apply for the iPSc modeling for novel partial lipodystrophy syndromes: platform for disease modeling and precision therapeutics?
To apply for iPSc modeling for novel partial lipodystrophy syndromes: platform for disease modeling and precision therapeutics, confirm your eligibility, gather the required documents, and prepare a narrative and budget that address the funder's priorities. FindGrants guides you step by step and can draft each section, then exports a submission-ready application pack for this grant from NIDDK - National Institute of Diabetes and Digestive and Kidney Diseases.