Evidence map›Paper›PMID 42843973›Full record

ArticleLife science alliance2026

Autolysosomal enhancer GHF201 attenuates pathophysiology in a mucopolysaccharidosis type I mouse model.

Pradeep Kumar, Emmanuelle Merquiol, Talya Avraham, Reut Sinai-Turyanski, Pankaj K Singh, Roberto A Ribas, Sahar Sweetat, Uri Sprecher, Noam Muchtar, Manjeet Gupta and 15 more

Abstract read
PubMed Full text at PMC Publisher
In one paragraph

Article in Life science alliance, 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

25 authors.

Pradeep KumarDepartment of Neurology, The Agnes Ginges Center for Human Neurogenetics, Hadassah Hebrew University Medical Center, Jerusalem, Israel kumar.pradeep@mail.huji.ac.il.ORCID https://orcid.org/0009-0000-4089-2283
Emmanuelle MerquiolThe Institute of Drug Research, School of Pharmacy, Faculty of Medicine, The Hebrew University of Jerusalem, Jerusalem, Israel.ORCID https://orcid.org/0000-0001-5754-498X
Talya AvrahamThe Institute of Drug Research, School of Pharmacy, Faculty of Medicine, The Hebrew University of Jerusalem, Jerusalem, Israel.
Reut Sinai-TuryanskiThe Institute of Drug Research, School of Pharmacy, Faculty of Medicine, The Hebrew University of Jerusalem, Jerusalem, Israel.
Pankaj K SinghDepartment of Biochemistry and Molecular Biology, College of Medicine, University of Florida, Gainesville, FL, USA.ORCID https://orcid.org/0000-0001-9626-5182
Roberto A RibasDepartment of Biochemistry and Molecular Biology, College of Medicine, University of Florida, Gainesville, FL, USA.ORCID https://orcid.org/0009-0008-9024-1292
Sahar SweetatDepartment of Neurology, The Agnes Ginges Center for Human Neurogenetics, Hadassah Hebrew University Medical Center, Jerusalem, Israel.
Uri SprecherThe Shmunis School of Biomedicine and Cancer Research, The George S. Wise Faculty for Life Sciences, Tel Aviv University, Tel Aviv, Israel.ORCID https://orcid.org/0000-0003-0284-5448
Noam MuchtarThe Shmunis School of Biomedicine and Cancer Research, The George S. Wise Faculty for Life Sciences, Tel Aviv University, Tel Aviv, Israel.ORCID https://orcid.org/0009-0001-4754-5089
Manjeet GuptaDepartment of Neurology, The Agnes Ginges Center for Human Neurogenetics, Hadassah Hebrew University Medical Center, Jerusalem, Israel.
Kumudesh MishraDepartment of Neurology, The Agnes Ginges Center for Human Neurogenetics, Hadassah Hebrew University Medical Center, Jerusalem, Israel.
Shashank Kumar OjhaThe Institute of Drug Research, School of Pharmacy, Faculty of Medicine, The Hebrew University of Jerusalem, Jerusalem, Israel.
Zhanna YekhtinDepartments of Bone Marrow Transplantation and Pulmonology, Hadassah Hebrew University Medical Center, Jerusalem, Israel.
Hava GlicksteinElectron Microscopy Unit, The Hebrew University-Hadassah Medical School, Ein Kerem, Jerusalem, Israel.
Ori ShalevThe Metabolic Profiling Unit, Core Research Facility, Faculty of Medicine, The Hebrew University of Jerusalem, Jerusalem, Israel.
Alexandra EliassafThe Metabolic Profiling Unit, Core Research Facility, Faculty of Medicine, The Hebrew University of Jerusalem, Jerusalem, Israel.
Anna MorshinaThe Metabolic Profiling Unit, Core Research Facility, Faculty of Medicine, The Hebrew University of Jerusalem, Jerusalem, Israel.
Hadas Segev-YekutielFlow Cytometry Unit, The Core Research Facility, Faculty of Medicine, The Hebrew University of Jerusalem, Jerusalem, Israel.
Sharon GartiLyotropic Delivery Systems Ltd., Jerusalem, Israel.
Nissim GartiLyotropic Delivery Systems Ltd., Jerusalem, Israel.
Hannah RosenmannDepartment of Neurology, The Agnes Ginges Center for Human Neurogenetics, Hadassah Hebrew University Medical Center, Jerusalem, Israel.
Miguel WeilThe Shmunis School of Biomedicine and Cancer Research, The George S. Wise Faculty for Life Sciences, Tel Aviv University, Tel Aviv, Israel.ORCID https://orcid.org/0000-0001-5721-653X
Matthew S GentryDepartment of Biochemistry and Molecular Biology, College of Medicine, University of Florida, Gainesville, FL, USA.
Galia BlumThe Institute of Drug Research, School of Pharmacy, Faculty of Medicine, The Hebrew University of Jerusalem, Jerusalem, Israel.
Or KakhlonDepartment of Neurology, The Agnes Ginges Center for Human Neurogenetics, Hadassah Hebrew University Medical Center, Jerusalem, Israel ork@hadassah.org.il.ORCID https://orcid.org/0000-0001-7435-6454

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Mucopolysaccharidosis type I (MPS-I) is a hereditary lysosomal storage disorder caused by deficiency in the lysosomal enzyme α-L-iduronidase (IDUA) leading to pathogenic over-accumulation of glycosaminoglycans (GAGs), major constituent of connective tissue and mediators of cell signaling. We tested the therapeutic efficacy of the autolysosomal activator GHF201 in the

Indexed as

LysosomesMucopolysaccharidosis IAnimalsAutophagyBrainCathepsinsDisease Models, AnimalFemaleFibroblastsGlycosaminoglycansHumansIduronidaseMiceMice, KnockoutCathepsinsGlycosaminoglycansIduronidase

Identifiers

PMID42843973
PMCPMC13646730

What OpenQuestion holds

Textmetadata
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.