Evidence map›Paper›PMID 40126917›Full record

ArticleCell transplantation

Transplantation of Wild-Type Hematopoietic Stem and Progenitor Cells Improves Disease Phenotypes in a Mucopolysaccharidosis IIIC Mouse Model.

Rafael A Badell-Grau, Kasra Pakravesh, Kevin Eric Thai, Frankie Son, Rola Chen, Joseph Rainaldi, Kalvin Duong, Pauline Losay, Anusha Sivakumar, Veenita Khare and 5 more

Abstract read
PubMed Full text at PMC Publisher
In one paragraph

Article in Cell transplantation. 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.

Rafael A Badell-GrauDepartment of Pediatrics, University of California, San Diego, La Jolla, CA, USA.ORCID 0000-0001-7640-822X
Kasra PakraveshDepartment of Pediatrics, University of California, San Diego, La Jolla, CA, USA.
Kevin Eric ThaiDepartment of Pediatrics, University of California, San Diego, La Jolla, CA, USA.
Frankie SonDepartment of Pediatrics, University of California, San Diego, La Jolla, CA, USA.
Rola ChenDepartment of Pediatrics, University of California, San Diego, La Jolla, CA, USA.
Joseph RainaldiDepartment of Pediatrics, University of California, San Diego, La Jolla, CA, USA.ORCID 0000-0003-4123-0008
Kalvin DuongDepartment of Pediatrics, University of California, San Diego, La Jolla, CA, USA.
Pauline LosayDepartment of Pediatrics, University of California, San Diego, La Jolla, CA, USA.
Anusha SivakumarDepartment of Pediatrics, University of California, San Diego, La Jolla, CA, USA.
Veenita KhareDepartment of Pediatrics, University of California, San Diego, La Jolla, CA, USA.
Alexis N CorlDepartment of Pediatrics, University of California, San Diego, La Jolla, CA, USA.
Rushil PithiaDepartment of Pediatrics, University of California, San Diego, La Jolla, CA, USA.
Christine TranDepartment of Pediatrics, University of California, San Diego, La Jolla, CA, USA.
Jefferey D EskoDepartment of Cellular & Molecular Medicine, University of California, San Diego, La Jolla, CA, USA.
Stephanie CherquiDivision of Genetics, Department of Pediatrics, University of California, San Diego, La Jolla, CA, USA.ORCID 0000-0003-1240-5219

Funding

Microgial contribution and therapeutic potential in Friedreich's ataxiaR01NS135162 · NINDS · UNIVERSITY OF CALIFORNIA, SAN DIEGO · PI Stephanie Cherqui, Nicole Gabriele Coufal · 2024 to 2026
$1.8M
Hematopoietic Stem Cell Gene Therapy for Friedreich's ataxiaR01NS108965 · NINDS · UNIVERSITY OF CALIFORNIA, SAN DIEGO · PI CHERQUI, STEPHANIE · 2018 to 2022
$1.7M
NINDS NIH HHS R01 NS108965NINDS NIH HHS R01 NS135162
6 · The paper itself

Abstract

Mucopolysaccharidosis type IIIC (MPS IIIC) is a severe neurodegenerative lysosomal storage disease caused by the loss-of-function of the lysosomal transmembrane protein acetyl-CoA: heparan-α-glucosamine

Indexed as

Hematopoietic Stem CellsHematopoietic Stem Cell TransplantationMucopolysaccharidosis IIIAcetyltransferasesAnimalsCell DifferentiationDisease Models, AnimalGlycosaminoglycansMiceMice, Inbred C57BLMice, KnockoutPhenotypeAcetyltransferasesGlycosaminoglycansglycosaminoglycans (GAGs)hematopoietic stem and progenitor cells (HSPCs)heparan sulfate (HS)lysosomal storage disorder (LSD)macrophagesmicrogliaSanfilippo syndrometransmembrane lysosomal protein

Identifiers

PMID40126917
PMCPMC11938874

What OpenQuestion holds

Textmetadata
LicenceCC BY-NC
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.