Evidence map›Paper›PMID 34823997›Full record

ArticleMolecular genetics and metabolism2021

Deficiency of the sedoheptulose kinase (Shpk) does not alter the ability of hematopoietic stem cells to rescue cystinosis in the mouse model.

Spencer Goodman, Meisha Khan, Jay Sharma, Zijie Li, Jose Cano, Carlos Castellanos, Monica V Estrada, Ilya Gertsman, Stephanie Cherqui

Open access · hybridAbstract read
PubMed Full text at PMC Publisher
In one paragraph

Article in Molecular genetics and metabolism, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 4 papers.

0numbers the graph read from it
0cells of the map it votes in
4citing papers in PubMed
0.3field-weighted citation impact, top 41% of its field
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

4 citing papers in PubMed, 5 citations in OpenAlex.

  1. IMPC impact on preclinical mouse models.Mammalian genome : official journal of the International Mammalian Genome Society · 2025
    Article
  2. Article
  3. Review
  4. Review
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

9 authors at 1 institution in 1 country.

Spencer GoodmanDepartment of Pediatrics, Division of Genetics, University of California, San Diego, La Jolla, CA, USA.
Meisha KhanDepartment of Pediatrics, Division of Genetics, University of California, San Diego, La Jolla, CA, USA.
Jay SharmaDepartment of Pediatrics, Division of Genetics, University of California, San Diego, La Jolla, CA, USA.
Zijie LiDepartment of Pediatrics, Division of Genetics, University of California, San Diego, La Jolla, CA, USA.
Jose CanoDepartment of Pediatrics, Division of Genetics, University of California, San Diego, La Jolla, CA, USA.
Carlos CastellanosDepartment of Pediatrics, Division of Genetics, University of California, San Diego, La Jolla, CA, USA.
Monica V EstradaTissue Technology Shared Resource, Biorepository and Tissue Technology, Moores Cancer Center, University of California, San Diego, La Jolla, CA, USA.
Ilya GertsmanClarus Analytical, LLC, San Diego, CA, USA.
Stephanie CherquiDepartment of Pediatrics, Division of Genetics, University of California, San Diego, La Jolla, CA, USA. Electronic address: scherqui@ucsd.edu.
University of California, San Diego · US

Funding

U of Calif, San Diego Neuroscience Microscopy ImagingP30NS047101 · NINDS · UNIVERSITY OF CALIFORNIA, SAN DIEGO · PI GLEESON, JOSEPH G, ZHENG, BINHAI · 2003 to 2022
$9.0M
Lentiviral-transduced hematopoictic stem cell transplantation for cystinosisR01DK090058 · NIDDK · UNIVERSITY OF CALIFORNIA, SAN DIEGO · PI CHERQUI, STEPHANIE · 2011 to 2019
$2.7M
Hematopoietic Stem Cell Gene Therapy for Friedreich's ataxiaR01NS108965 · NINDS · UNIVERSITY OF CALIFORNIA, SAN DIEGO · PI CHERQUI, STEPHANIE · 2018 to 2022
$1.7M
NIDDK NIH HHS R01 DK090058NINDS NIH HHS P30 NS047101NINDS NIH HHS R01 NS108965
6 · The paper itself

Abstract

Cystinosis is an autosomal recessive lysosomal storage disorder caused by mutations in the CTNS gene encoding the lysosomal cystine transporter, cystinosin, and leading to multi-organ degeneration including kidney failure. A clinical trial for cystinosis is ongoing to test the safety and efficacy of transplantation of autologous hematopoietic stem and progenitor cells (HSPCs) ex vivo gene-modified to introduce functional CTNS cDNA. Preclinical studies in Ctns

Indexed as

Amino Acid Transport Systems, NeutralAnimalsCell DifferentiationCystinosisDisease Models, AnimalGenetic TherapyHematopoietic Stem CellsHematopoietic Stem Cell TransplantationMetabolomicsMiceMice, Inbred C57BLPentose Phosphate PathwayPhosphotransferases (Alcohol Group Acceptor)Amino Acid Transport Systems, Neutralcystinosin protein, mousePhosphotransferases (Alcohol Group Acceptor)Shpk protein, mouseCystinosisHematopoietic stem and progenitor cellsMacrophagesPentose phosphate pathwaySedopheptulose kinase

Identifiers

PMID34823997
PMCPMC8935660
OpenAlexW3212394422

What Socratic 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 Socratic graph.