In one paragraphArticle in bioRxiv : the preprint server for biology, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.
0numbers the graph read from it
0cells of the map it votes in
0citing papers in PubMed
–field-weighted citation impact
What it found
Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.
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
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
3 · Its place in the literatureWho cites it
0 citing papers in PubMed.
No citing paper in PubMed yet.
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
32 authors.
Romina MaroneDepartment of Biomedicine, Basel University Hospital and University of Basel, Hebelstrasse 20, CH-4031 Basel, Switzerland.ORCID 0000-0003-1474-1689 Rosalba LeporeDepartment of Biomedicine, Basel University Hospital and University of Basel, Hebelstrasse 20, CH-4031 Basel, Switzerland.ORCID 0000-0002-9481-2557 Jessica ZuinDepartment of Biomedicine, Basel University Hospital and University of Basel, Hebelstrasse 20, CH-4031 Basel, Switzerland.ORCID 0000-0003-3253-1984 Thomas BurgoldDepartment of Biomedicine, Basel University Hospital and University of Basel, Hebelstrasse 20, CH-4031 Basel, Switzerland.ORCID 0009-0006-8324-8934 Ewelina BartoszekDepartment of Biomedicine, Basel University Hospital and University of Basel, Hebelstrasse 20, CH-4031 Basel, Switzerland.
Diego CalabreseDepartment of Biomedicine, Basel University Hospital and University of Basel, Hebelstrasse 20, CH-4031 Basel, Switzerland.
Mylène ToranelliDepartment of Biomedicine, Basel University Hospital and University of Basel, Hebelstrasse 20, CH-4031 Basel, Switzerland.
Julia WittwerInstitute for Transfusion Medicine & Gene Therapy, Medical Center - University of Freiburg, 79106 Freiburg, Germany.ORCID 0009-0009-3023-3044 Manuel RhielInstitute for Transfusion Medicine & Gene Therapy, Medical Center - University of Freiburg, 79106 Freiburg, Germany.ORCID 0000-0003-0741-2780 Geoffroy AndrieuxInstitute of Medical Bioinformatics and Systems Medicine, Medical Center - University of Freiburg, Faculty of Medicine, University of Freiburg, 79110 Freiburg, Germany.ORCID 0000-0002-5389-9481 Chang LiDivision of Medical Genetics, Department of Medicine, University of Washington, Seattle, WA.
Alvin HsuMerkin Institute of Transformative Technologies in Healthcare, Broad Institute of Harvard and MIT; Cambridge, MA, 02142, USA.ORCID 0000-0003-4034-2788 Eva-Maria GrossjohannCimeio Therapeutics AG, Aeschenvorstadt 36, CH-4051 Basel, Switzerland.
Saniye YumluCimeio Therapeutics AG, Aeschenvorstadt 36, CH-4051 Basel, Switzerland.
David R LiuMerkin Institute of Transformative Technologies in Healthcare, Broad Institute of Harvard and MIT; Cambridge, MA, 02142, USA.
André LieberDivision of Medical Genetics, Department of Medicine, University of Washington, Seattle, WA.ORCID 0000-0001-6742-6266 Toni CathomenInstitute for Transfusion Medicine & Gene Therapy, Medical Center - University of Freiburg, 79106 Freiburg, Germany.ORCID 0000-0002-7757-4630 Tatjana I CornuInstitute for Transfusion Medicine & Gene Therapy, Medical Center - University of Freiburg, 79106 Freiburg, Germany.ORCID 0000-0002-7206-7541 Lukas T JekerDepartment of Biomedicine, Basel University Hospital and University of Basel, Hebelstrasse 20, CH-4031 Basel, Switzerland.ORCID 0000-0002-3359-8796 Funding
Center for Genomic Editing and Recording: Development and Application of Next-Generation Genome and Epigenome Editing Methods to Advance the Study and Treatment of Human DiseaseRM1HG009490 · NHGRI · WHITEHEAD INSTITUTE FOR BIOMEDICAL RES · PI Brittany S. Adamson, Martin Joseph Ankrah Aryee · 2017 to 2026
$22.7MIntegrating Chemistry and Evolution to Illuminate Biology and Enable Novel TherapeuticsR35GM118062 · NIGMS · HARVARD UNIVERSITY · PI LIU, DAVID R · 2016 to 2025
$6.4MNHGRI NIH HHS RM1 HG009490NIGMS NIH HHS R35 GM118062
6 · The paper itselfAbstract
Clinical evidence demonstrates that ex vivo gene therapy and genome engineering of hematopoietic stem and progenitor cells (HSPCs) could represent one-time cures. However, while genome editing itself has become increasingly efficient and precise, the toxic conditioning required for hematopoietic stem cell transplantation remains a major barrier to broad clinical implementation of these otherwise curative therapies. In particular, the use of busulfan for myeloablative conditioning constitutes a major safety concern. While preclinical studies established CD117 as a promising target for antigen-specific therapy, clinical translation faced setbacks balancing efficacy and safety. To overcome current limitations, we generated a new CD117-blocking monoclonal antibody (CIM058) and demonstrate its potency to block wild-type HSPCs. To enable long-term blockade of host HSPCs even after transplantation, we used prime editing to engineer CIM058-resistant human CD34
Indexed as
CD117c-kitCRISPR/Cas9epitope engineeringgenome engineeringHematopoietic stem cell transplantation (HSCT)molecular shieldmonoclonal antibody SR-1non-genotoxic conditioningprime editingtargeted immunotherapyβ-thalassemia
Identifiers
PMID42465494
PMCPMC13371095
What Socratic holds
Textmetadata
LicenceCC BY-ND
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