Prime-editing correction of pathogenic ABCA4 variants in retinal organoids
Dr. Lena Okafor · UC San Diego
Deploying a split-intein prime editor to revert Stargardt-causing mutations in patient-derived photoreceptor organoids, with single-cell readout of correction efficiency.
// Current status
- Stage
- Data collection
- Phase
- Phase II of III
- Started
- Jan 2025
- Next milestone
- Interim dataset released to backers
// Team selected by funders
- Dr. Lena OkaforPrincipal investigator
PhD · UC San Diego · 40+ peer-reviewed publications
Named on the original campaign
View credentials ↗ - Jonas SilvaPostdoctoral researcher
PhD · UC San Diego
Voted in by 1,204 funders
View credentials ↗ - Yuki HaddadResearch engineer
MSc · instrumentation & automation
Voted in by 968 funders
View credentials ↗ - Theo IyerData scientist
PhD · statistics and reproducibility
Voted in by 741 funders
View credentials ↗
// Where the money goes
- Personnel & stipends42% of funds
Salaries for the funder-selected team, published quarterly
View receipts ↗ - Reagents & consumables23% of funds
Itemised supplier invoices for every order
View receipts ↗ - Instrument time19% of funds
Core-facility booking logs with hourly rates
View receipts ↗ - Open-access publishing9% of funds
Article processing charges so results stay free to read
View receipts ↗ - Platform & audit fee7% of funds
Independent quarterly audit of all spending
View receipts ↗
// Research updates
This month
Data collection underway
Deploying a split-intein prime editor to revert Stargardt-causing mutations in patient-derived photoreceptor organoids, with single-cell readout of correction efficiency. The team is currently working through the data collection stage and posting weekly notebook entries for backers.
Last month
Funder-selected team confirmed
Backers reviewed shortlisted candidates and voted the postdoc, research engineer, and data scientist onto the project. Credentials are linked above.
Campaign start
Protocol pre-registered
The full protocol, analysis plan, and budget breakdown were registered publicly before a single dollar was spent.