Evidence map›Paper›PMID 37633404›Full record

ArticleNanomedicine : nanotechnology, biology, and medicine2023

Apolipoprotein-mimetic nanodiscs reduce lipid accumulation and improve liver function in acid sphingomyelinase deficiency.

Troy A Halseth, Adele B Correia, Mark L Schultz, Maria V Fawaz, Esmée Q Kuiper, Preethi Kumaran, Kristen Hong Dorsey, Edward H Schuchman, Andrew P Lieberman, Anna Schwendeman

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

Article in Nanomedicine : nanotechnology, biology, and medicine, 2023. 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
0.2field-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

0 citing papers in PubMed, 1 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

10 authors at 4 institutions in 1 country.

Troy A HalsethDepartment of Medicinal Chemistry, College of Pharmacy, University of Michigan, 428 Church St, Ann Arbor, MI 48109, USA.
Adele B CorreiaDepartment of Pathology, University of Michigan Medical School, 3510 MSRB1, 1150 W. Medical Center Dr., Ann Arbor, MI, 48109, USA.
Mark L SchultzStead Family Department of Pediatrics, University of Iowa Carver College of Medicine, 2182 ML, Iowa City, IA 52242, USA.
Maria V FawazDepartment of Medicinal Chemistry, College of Pharmacy, University of Michigan, 428 Church St, Ann Arbor, MI 48109, USA.
Esmée Q KuiperDepartment of Pathology, University of Michigan Medical School, 3510 MSRB1, 1150 W. Medical Center Dr., Ann Arbor, MI, 48109, USA.
Preethi KumaranDepartment of Pharmaceutical Sciences, College of Pharmacy, University of Michigan, 428 Church St, Ann Arbor, MI 48109, USA.
Kristen Hong DorseyDepartment of Pharmaceutical Sciences, College of Pharmacy, University of Michigan, 428 Church St, Ann Arbor, MI 48109, USA.
Edward H SchuchmanDepartment of Genetics and Genomic Sciences, The Icahn School of Medicine at Mount Sinai, New York, NY 10029, USA.
Andrew P LiebermanDepartment of Pathology, University of Michigan Medical School, 3510 MSRB1, 1150 W. Medical Center Dr., Ann Arbor, MI, 48109, USA.
Anna SchwendemanDepartment of Pharmaceutical Sciences, College of Pharmacy, University of Michigan, 428 Church St, Ann Arbor, MI 48109, USA; Biointerfaces Institute, NCRC, 2800 Plymouth Rd, Ann Arbor, MI 48109, USA. Electronic address: annaschw@umich.edu.
University of Michigan–Ann Arbor · USBioSurfaces (United States) · USIcahn School of Medicine at Mount Sinai · USUniversity of Iowa · US

Funding

Therapeutic Targets for Niemann-Pick Type C NeurodegenerationR01NS122746 · NINDS · UNIVERSITY OF MICHIGAN AT ANN ARBOR · PI LIEBERMAN, ANDREW P · 2021 to 2025
$3.0M
Training Program in Translational Cardiovascular Research and EntrepreneurshipT32HL125242 · NHLBI · UNIVERSITY OF MICHIGAN AT ANN ARBOR · PI MICHELE, DANIEL E · 2015 to 2024
$1.9M
Niemann-Pick C liver-specific proteostasis and pathologyK01DK124450 · NIDDK · UNIVERSITY OF MICHIGAN AT ANN ARBOR · PI SCHULTZ, MARK LOUIS · 2021 to 2024
$493k
NHLBI NIH HHS T32 HL125242NIDDK NIH HHS K01 DK124450NINDS NIH HHS R01 NS122746
6 · The paper itself

Abstract

Acid sphingomyelinase deficiency (ASMD) is a severe lipid storage disorder caused by the diminished activity of the acid sphingomyelinase enzyme. ASMD is characterized by the accumulation of sphingomyelin in late endosomes and lysosomes leading to progressive neurological dysfunction and hepatosplenomegaly. Our objective was to investigate the utility of synthetic apolipoprotein A-I (ApoA-I) mimetics designed to act as lipid scavengers for the treatment of ASMD. We determined the lead peptide, 22A, could reduce sphingomyelin accumulation in ASMD patient skin fibroblasts in a dose dependent manner. Intraperitoneal administration of 22A formulated as a synthetic high-density lipoprotein (sHDL) nanodisc mobilized sphingomyelin from peripheral tissues into circulation and improved liver function in a mouse model of ASMD. Together, our data demonstrates that apolipoprotein mimetics could serve as a novel therapeutic strategy for modulating the pathology observed in ASMD.

Indexed as

Niemann-Pick Disease, Type AAnimalsHumansLiverMicePeptidesSphingomyelinsPeptidesSphingomyelinsAcid sphingomyelinase deficiencyApolipoprotein mimetics

Identifiers

PMID37633404
PMCPMC10530155
OpenAlexW4386122611

What OpenQuestion holds

Textmetadata
LicenceTDM
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.