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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.

79% funded
1,284 backers18 days remaining

// 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 ↗
Full spending ledger ↗

// Research updates

  1. 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.

  2. 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.

  3. Campaign start

    Protocol pre-registered

    The full protocol, analysis plan, and budget breakdown were registered publicly before a single dollar was spent.

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