Closed. Age-related Macular Degeneration (AMD) Integrative Biology Initiative: Discovery of AMD Pathobiology using Patient-Derived Induced Pluripotent Stem Cell (iPSC)-derived Retinal Pigment Epithelium (RPE) (U01 Clinical Trial Not Allowed)
This funding opportunity aims to support collaborative studies of a unique resource of patient-derived induced pluripotent stem cell (iPSC) lines generated by the NEI age-related macular degeneration (AMD) Integrative Biology Initiative. The goal of this FOA is to determine if these iPSC-derived cell lines can be used to discover the underlying pathophysiology of AMD. Collaborative effort is highly encouraged with investigators bringing in the needed areas of expertise for successful projects.
Newly Launched AMD Integrative Biology Initiative - A Community Resource of Patient iPSC lines
In a bold effort to build on the AREDS2 investments, the NEI is working to:
- correlate clinical AMD disease phenotypes in a cellular model with patient genotype and imaging information in the newly created AMD Integrative Biology Initiative,
- make this Initiative available to the research community with induced pluripotent stem cells (iPSCs) derived from AREDS2 participants with specific genetic risk factors for AMD, and
- cover a cohort of 73 AREDS2 patients (chosen based on known AMD risk alleles) in this undertaking that is done in partnership with the New York Stem Cell Foundation Research Institute (NYSCF).
There are now 60 iPSC lines now available from NYSCF and the accompanying genomic and phenotypic data will be available to the research community later this year. You can view this data NEI EyePSC page and it will be updated as new information is posted.
Isogenic control lines in which the risk alleles have been corrected will be available next year. These iPSC lines are intended for research use and requestors will be required to submit a description of their research when requesting the lines.
How do I get these iPSC lines? Contact NYSCF for the NEI Age-related Macular Degeneration Panel and include a brief statement of work on how you will use the lines.
Flow data for first 11 iPSC lines with high prevalence risk alleles
The first 11 iPSC lines have been validated in Dr. Kapil Bharti’s lab for differentiation to RPE. Flow cytometry data using MITF and TYRP1 as markers are available upon request. Isogenic control lines in which the risk alleles have been corrected will be available later this year for the first 11 iPSC lines with high-prevalence risk alleles.
For acknowledgment of this data, please note Ruchi Sharma, Devika Bose, Genqing Liang, Roba Dejene, and Kapil Bharti (all NEI) as originators of the data.
RPE Protocol Resources
- Science Translational Medicine Research Article: Clinical-grade stem cell–derived retinal pigment epithelium patch rescues retinal degeneration in rodents and pigs
- Supplementary Materials for Clinical-grade stem cell–derived retinal pigment epithelium patch rescues retinal degeneration in rodents and pigs