Evidence map›Paper›PMID 40994008›Full record

ArticleMolecular therapy : the journal of the American Society of Gene Therapy2026

Nonclinical evaluation of renizgamglogene autogedtemcel for SCD and TDT.

Eugenio Marco, Patricia Sousa, Tusneem Janoudi, Edouard de Dreuzy, Jack M Heath, Ramya Viswanathan, John A Zuris, Gregory M Gotta, Georgia Giannoukos, Scott Hansen and 5 more

Abstract read
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In one paragraph

Article in Molecular therapy : the journal of the American Society of Gene Therapy, 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
1 · What the graph read from it

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 registry

The 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 literature

Who cites it

0 citing papers in PubMed.

No citing paper in PubMed yet.

4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

15 authors.

Eugenio MarcoEditas Medicine, Cambridge, MA 02141, USA. Electronic address: eugenio.marco@editasmed.com.
Patricia SousaEditas Medicine, Cambridge, MA 02141, USA.
Tusneem JanoudiEditas Medicine, Cambridge, MA 02141, USA.
Edouard de DreuzyEditas Medicine, Cambridge, MA 02141, USA.
Jack M HeathEditas Medicine, Cambridge, MA 02141, USA.
Ramya ViswanathanEditas Medicine, Cambridge, MA 02141, USA.
John A ZurisEditas Medicine, Cambridge, MA 02141, USA.
Gregory M GottaEditas Medicine, Cambridge, MA 02141, USA.
Georgia GiannoukosEditas Medicine, Cambridge, MA 02141, USA.
Scott HansenDepartment of Biomedical Engineering, University of Minnesota, Minneapolis, MN 55455, USA.
David K WoodDepartment of Biomedical Engineering, University of Minnesota, Minneapolis, MN 55455, USA.
Mark C WaltersSchool of Medicine, University of California, San Francisco Benioff Children's Hospital, Oakland, CA 94609, USA.
John F TisdaleCellular and Molecular Therapeutics Branch, National Heart, Lung, and Blood Institute, National Institutes of Health, Bethesda, MD 20892, USA.
Christopher J WilsonEditas Medicine, Cambridge, MA 02141, USA.
Kai-Hsin ChangEditas Medicine, Cambridge, MA 02141, USA.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Sickle cell disease and transfusion-dependent β-thalassemia can be treated by fetal hemoglobin upregulation. Disruption of the distal BCL11A binding site at the HBG1/2 promoters to induce fetal hemoglobin using either SpCas9 or AsCas12a mimics multiple hereditary persistence of fetal hemoglobin mutations. AsCas12a showed higher editing efficiency, higher specificity, and increased fetal hemoglobin induction potential compared with SpCas9. AsCas12a-edited healthy donor CD34

Indexed as

Anemia, Sickle Cellbeta-ThalassemiaGene EditingAnimalsCarrier ProteinsCRISPR-Cas SystemsErythroid CellsFetal HemoglobinHumansMicePromoter Regions, GeneticRepressor ProteinsBCL11A protein, humanCarrier ProteinsFetal HemoglobinRepressor ProteinsAsCas12aautologous cell therapyCRISPRgenome editingHbFsickle cell diseasetransfusion-dependent thalassemia

Identifiers

PMID40994008
PMCPMC12925820

What OpenQuestion holds

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LicenceCC BY-NC-ND
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Registered trials

None linked

Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.