Evidence map›Paper›PMID 39763989›Full record

ArticlebioRxiv : the preprint server for biology2024

Haplotype editing with CRISPR/Cas9 as a therapeutic approach for dominant-negative missense mutations in

Poorvi H Dua, Bazilco M J Simon, Chiara B E Marley, Carissa M Feliciano, Hannah L Watry, Dylan Steury, Abin Abraham, Erin N Gilbertson, Grace D Ramey, John A Capra and 2 more

Abstract readPreprint
PubMed Full text at PMC Publisher
In one paragraph

Article in bioRxiv : the preprint server for biology, 2024. 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

5 · Who and what money

Authors and funding

12 authors.

Poorvi H DuaDepartment of Pediatrics, University of California, San Francisco, San Francisco, CA, USA.
Bazilco M J SimonGladstone Institutes, San Francisco, CA, United States.
Chiara B E MarleyDepartment of Pediatrics, University of California, San Francisco, San Francisco, CA, USA.
Carissa M FelicianoDepartment of Pediatrics, University of California, San Francisco, San Francisco, CA, USA.
Hannah L WatryGladstone Institutes, San Francisco, CA, United States.
Dylan SteuryGladstone Institutes, San Francisco, CA, United States.
Abin AbrahamVanderbilt Genetics Institute, Vanderbilt University, Nashville, TN, USA.
Erin N GilbertsonBiomedical Informatics Graduate Program, University of California, San Francisco, San Francisco, CA, USA.ORCID 0000-0002-2426-9966
Grace D RameyBiomedical Informatics Graduate Program, University of California, San Francisco, San Francisco, CA, USA.
John A CapraBakar Computational Health Sciences Institute, University of California, San Francisco, CA, USA.ORCID 0000-0001-9743-1795
Bruce R ConklinGladstone Institutes, San Francisco, CA, United States.
Luke M JudgeDepartment of Pediatrics, University of California, San Francisco, San Francisco, CA, USA.

Funding

MEDICAL SCIENTIST TRAINING PROGRAMT32GM007347 · NIGMS · VANDERBILT UNIVERSITY · PI WILLIAMS, CHRISTOPHER S. · 1985 to 2023
$26.3M
The Evolution of Gene Regulation and Human DiseaseR35GM127087 · NIGMS · VANDERBILT UNIVERSITY · PI John Anthony Capra · 2018 to 2026
$3.2M
JAX-Gladstone, SCGE Disease Models Studies SupplementU01ES032673 · NIEHS · J. DAVID GLADSTONE INSTITUTES · PI CONKLIN, BRUCE R · 2020 to 2022
$2.4M
Allele-specific inactivation for dominant negative NEFL MutationsR01NS119678 · NINDS · UNIVERSITY OF CALIFORNIA, SAN FRANCISCO · PI JUDGE, LUKE M · 2021 to 2025
$1.9M
Investigating clinical risk between autoimmunity and Alzheimer’s Disease in diverse human populationsF31AG090013 · NIA · UNIVERSITY OF CALIFORNIA, SAN FRANCISCO · PI Grace Ramey · 2024 to 2026
$93k
NIA NIH HHS F31 AG090013NIEHS NIH HHS U01 ES032673NIGMS NIH HHS R35 GM127087NIGMS NIH HHS T32 GM007347NINDS NIH HHS R01 NS119678
6 · The paper itself

Abstract

Inactivation of disease alleles by allele-specific editing is a promising approach to treat dominant-negative genetic disorders, provided the causative gene is haplo-sufficient. We previously edited a dominant

Identifiers

PMID39763989
PMCPMC11702708

What OpenQuestion holds

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Registered trials

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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.