Evidence map›Paper›PMID 38633814›Full record

ArticlemedRxiv : the preprint server for health sciences2025

Antisense oligonucleotide depletion of

Sai Zhang, Tobias Moll, Jasper Rubin-Sigler, Sharon Tu, Shuya Li, Enming Yuan, Menghui Liu, Marriane King, Elham Alhathli, Connie Treanor and 24 more

Open access · greenAbstract readPreprint
PubMed Full text at PMC Publisher
In one paragraph

Article in medRxiv : the preprint server for health sciences, 2025. 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, 4 citations in OpenAlex.

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

34 authors at 13 institutions in 6 countries.

Sai ZhangDepartment of Epidemiology, University of Florida, Gainesville, FL, USA.
Tobias MollSheffield Institute for Translational Neuroscience (SITraN), University of Sheffield, Sheffield, UK.
Jasper Rubin-SiglerDepartment of Stem Cell Biology and Regenerative Medicine, Eli and Edythe Broad Center for Regenerative Medicine and Stem Cell Research, University of Southern California, Los Angeles, CA, USA.
Sharon TuDepartment of Stem Cell Biology and Regenerative Medicine, Eli and Edythe Broad Center for Regenerative Medicine and Stem Cell Research, University of Southern California, Los Angeles, CA, USA.
Shuya LiSchool of Engineering, Research Center for Industries of the Future, Westlake University, Hangzhou, Zhejiang, China.
Enming YuanInstitute for Interdisciplinary Information Sciences, Tsinghua University, Beijing, China.
Menghui LiuDepartment of Epidemiology, University of Florida, Gainesville, FL, USA.
Marriane KingSheffield Institute for Translational Neuroscience (SITraN), University of Sheffield, Sheffield, UK.
Elham AlhathliSheffield Institute for Translational Neuroscience (SITraN), University of Sheffield, Sheffield, UK.
Connie TreanorSheffield Institute for Translational Neuroscience (SITraN), University of Sheffield, Sheffield, UK.
Alison TwelvetreesSheffield Institute for Translational Neuroscience (SITraN), University of Sheffield, Sheffield, UK.
Marcel WeinreichSheffield Institute for Translational Neuroscience (SITraN), University of Sheffield, Sheffield, UK.
Sam BonsallSheffield Institute for Translational Neuroscience (SITraN), University of Sheffield, Sheffield, UK.
Calum HarveySheffield Institute for Translational Neuroscience (SITraN), University of Sheffield, Sheffield, UK.
Sarah GornallSheffield Institute for Translational Neuroscience (SITraN), University of Sheffield, Sheffield, UK.
Allan ShawSheffield Institute for Translational Neuroscience (SITraN), University of Sheffield, Sheffield, UK.
Johanna GanssaugeLiving Systems Institute, University of Exeter, Exeter, UK.
Akshay BhingeLiving Systems Institute, University of Exeter, Exeter, UK.
Cleide Dos Santos SouzaSheffield Institute for Translational Neuroscience (SITraN), University of Sheffield, Sheffield, UK.
Laura FerraiuoloSheffield Institute for Translational Neuroscience (SITraN), University of Sheffield, Sheffield, UK.
Eran HornsteinDepartments of Molecular Genetics and Molecular Neuroscience, Weizmann Institute of Science, Rehovot, Israel.
Ophir ShalemDepartment of Genetics, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA.
Anna AvilaSheffield Institute for Translational Neuroscience (SITraN), University of Sheffield, Sheffield, UK.
Tatyana ShelkovnikovaSheffield Institute for Translational Neuroscience (SITraN), University of Sheffield, Sheffield, UK.
Charlotte H van DijkDepartment of Neurology, Brain Center Rudolf Magnus, University Medical Center Utrecht, Utrecht, The Netherlands.
Ilia S TimpanaroDepartment of Neurology, Brain Center Rudolf Magnus, University Medical Center Utrecht, Utrecht, The Netherlands.
Kevin P KennaDepartment of Neurology, Brain Center Rudolf Magnus, University Medical Center Utrecht, Utrecht, The Netherlands.
Jianyang ZengSchool of Engineering, Research Center for Industries of the Future, Westlake University, Hangzhou, Zhejiang, China.
Philip van DammeNeurology Department, University of Leuven (KU Leuven), Belgium.
Philip S TsaoVA Palo Alto Healthcare System, Palo Alto, CA, USA.
Pamela J ShawSheffield Institute for Translational Neuroscience (SITraN), University of Sheffield, Sheffield, UK.
Justin K IchidaDepartment of Stem Cell Biology and Regenerative Medicine, Eli and Edythe Broad Center for Regenerative Medicine and Stem Cell Research, University of Southern California, Los Angeles, CA, USA.
Johnathan Cooper-KnockSheffield Institute for Translational Neuroscience (SITraN), University of Sheffield, Sheffield, UK.
Michael P SnyderDepartment of Genetics, Center for Genomics and Personalized Medicine, Stanford University School of Medicine, Stanford, CA, USA.
University of Sheffield · GBUniversity Medical Center Utrecht · NLUniversity of Southern California · USWestlake University · CNUniversity of Exeter · GBUniversity of Florida · USVA Palo Alto Health Care System · USKU Leuven · BEStanford Medicine · USTsinghua University · CNUniversity of Pennsylvania · USWeizmann Institute of Science · ILZero to Three · US

Funding

Special EquipmentP50HG007735 · NHGRI · STANFORD UNIVERSITY · PI CHANG, HOWARD Y · 2014 to 2018
$15.2M
Validating Secretory Autophagy as a Therapeutic Strategy for Diverse Forms of ALS and FTDR01NS097850 · NINDS · UNIVERSITY OF SOUTHERN CALIFORNIA · PI ICHIDA, JUSTIN KAWIKA · 2016 to 2024
$5.5M
Leveraging Natural Phenotypic Variations of Heterogenous ALS Populations-in-a-Dish to Enable Scalable Drug DiscoveryR01NS131409 · NINDS · UNIVERSITY OF SOUTHERN CALIFORNIA · PI GOODARZI, HANI, ICHIDA, JUSTIN KAWIKA · 2022 to 2025
$4.9M
NHGRI NIH HHS P50 HG007735NINDS NIH HHS R01 NS097850NINDS NIH HHS R01 NS131409Wellcome Trust
6 · The paper itself

Abstract

Amyotrophic lateral sclerosis (ALS) is a heritable and incurable disease defined by the degeneration of motor neurons (MNs), yet the genetics of ALS remain partially understood. Using a genomic deep learning-powered whole-genome analysis of 6,715 ALS patients, we identify four rare noncoding variants associated with patient survival, including chr7:76,009,472:C>T which is linked to a 70.6% reduction in survival. Genetic editing of this variant into iPSC-derived MNs increases

Identifiers

PMID38633814
PMCPMC11023684
OpenAlexW4393370941

What OpenQuestion holds

Textmetadata
LicenceCC BY-NC-ND
Read underepoch 390

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.