ArticlemedRxiv : the preprint server for health sciences2026
Leveraging human genetic variation to therapeutically target hundreds of genes with dominant & dispensable disease alleles.
Grace D Ramey, Quinn T Cowan, Akshita G Saxena, Bria L Macklin, Hannah L Watry, Xiaoyue Mei, Philip Dierks, Luke M Judge, Bruce R Conklin, John A Capra
Abstract readPreprint
In one paragraphArticle in medRxiv : the preprint server for health sciences, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.
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0citing papers in PubMed
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What it found
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
2 · The registryThe trial behind it
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3 · Its place in the literatureWho cites it
0 citing papers in PubMed.
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4 · The recordCorrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
5 · Who and what moneyAuthors and funding
10 authors.
Grace D RameyBiomedical Informatics PhD Program, University of California, San Francisco, San Francisco, CA 94143, USA.ORCID 0000-0001-5321-131X Quinn T CowanGladstone Institute of Data Science and Biotechnology, San Francisco, CA 94158, USA.
Akshita G SaxenaGladstone Institute of Data Science and Biotechnology, San Francisco, CA 94158, USA.
Bria L MacklinGladstone Institute of Data Science and Biotechnology, San Francisco, CA 94158, USA.
Hannah L WatryGladstone Institute of Data Science and Biotechnology, San Francisco, CA 94158, USA.
Xiaoyue MeiBioengineering PhD Program, University of California, San Francisco, San Francisco, CA 94143, USA and University of California, Berkeley, Berkeley, CA 94720.
Philip DierksGladstone Institute of Data Science and Biotechnology, San Francisco, CA 94158, USA.
Luke M JudgeGladstone Institute of Data Science and Biotechnology, San Francisco, CA 94158, USA.
Bruce R ConklinGladstone Institute of Data Science and Biotechnology, San Francisco, CA 94158, USA.
John A CapraBakar Computational Health Sciences Institute, University of California, San Francisco, San Francisco, CA 94143, USA.ORCID 0000-0001-9743-1795 Funding
The Evolution of Gene Regulation and Human DiseaseR35GM127087 · NIGMS · VANDERBILT UNIVERSITY · PI John Anthony Capra · 2018 to 2026
$3.2MAllele-specific inactivation for dominant negative NEFL MutationsR01NS119678 · NINDS · UNIVERSITY OF CALIFORNIA, SAN FRANCISCO · PI JUDGE, LUKE M · 2021 to 2025
$1.9MC9orf72 frontotemporal dementia (FTD) and amyotrophic lateral sclerosis(ALS): using patient cells and CRISPR to reveal therapeutic approachesR01AG072052 · NIA · J. DAVID GLADSTONE INSTITUTES · PI CONKLIN, BRUCE R · 2024 to 2025
$1.4MInvestigating clinical risk between autoimmunity and Alzheimer’s Disease in diverse human populationsF31AG090013 · NIA · UNIVERSITY OF CALIFORNIA, SAN FRANCISCO · PI Grace Ramey · 2024 to 2026
$93kNIA NIH HHS F31 AG090013NIA NIH HHS R01 AG072052NIGMS NIH HHS R35 GM127087NINDS NIH HHS R01 NS119678
6 · The paper itselfAbstract
Here we identify a novel therapeutic opportunity for over 500 genes with putative "dominant & dispensable" (D&D) disease alleles. In these haplo
Identifiers
PMID41929339
PMCPMC13042096
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