iSCORE-PD cell collection

The research record

This is the project's file for this landmark, as written and checked. Sources are listed at the end; anything not yet confirmed is marked "Not yet verified".

Who

Oriol Busquets, Hanqin Li, Khaja Mohieddin Syed, Pilar Alvarez Jerez and Jesse Dunnack (joint first authors), supervised by Dirk Hockemeyer (UC Berkeley) and Frank Soldner (Albert Einstein College of Medicine), with Cornelis Blauwendraat and Donald Rio. The work is part of the Aligning Science Across Parkinson's (ASAP) research network; the paper lists US National Institute of Neurological Disorders and Stroke and National Cancer Institute grants, and reports no competing interests. Lines are distributed through the WiCell Research Institute with support from ASAP and the Michael J. Fox Foundation; academic requests need a material transfer agreement. Preprint on bioRxiv, Feb 2024 (DOI 10.1101/2024.02.12.579917); Nat Commun 2026.

What happened

The team edited a well-characterised female human embryonic stem cell line (WIBR3). The published version describes 65 lines with disease-causing or high-risk variants in 11 genes: SNCA, PRKN, PINK1, DJ1/PARK7, LRRK2, ATP13A2, FBXO7, DNAJC6, SYNJ1, VPS13C and GBA1. The preprint also listed specific variants such as SNCA A53T and A30P and LRRK2 G2019S. Every line had whole-genome sequencing. Genetic variation between lines was minimal compared with differences between patients' own cells, and most of it arose from culture rather than editing. Using several independent clones per mutation controls for that drift. The paper also highlights prime editing (a more precise editing method) over standard CRISPR/Cas9, and says the resource is publicly available.

Why it moved us toward a cure

It extends the single-lab isogenic design of landmark 272 into a shared resource, in the spirit of the open-science programmes of landmark 78. Many labs can now test the same mutation in the same background, which should make cell-model results easier to compare and drug screens more reliable.

Outcome

Advanced as a tool: the collection is published and distributed. No therapy has come from it yet, and it measures nothing about patients.

Sources

• Busquets et al., Nat Commun 2026, DOI 10.1038/s41467-026-74355-8, PMID 42310027 (PMC13434110)
• Busquets et al., bioRxiv preprint 2024, DOI 10.1101/2024.02.12.579917
• Full text read via Europe PMC (PMC13434110, open access). No correction or retraction listed.
• Not yet verified: the Nature Communications article number and page details; how many labs have used the lines.