Evidence map›Paper›PMID 33854948›Full record

ArticleMolecular genetics and metabolism reports2021

Examination of a blood-brain barrier targeting β-galactosidase-monoclonal antibody fusion protein in a murine model of GM1-gangliosidosis.

Michael J Przybilla, Christine Stewart, Timothy W Carlson, Li Ou, Brenda L Koniar, Rohini Sidhu, Pamela J Kell, Xuntian Jiang, Jeanine R Jarnes, M Gerard O'Sullivan and 1 more

Abstract read
PubMed Full text at PMC Publisher
In one paragraph

Article in Molecular genetics and metabolism reports, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 7 papers.

0numbers the graph read from it
0cells of the map it votes in
7citing 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

7 citing papers in PubMed.

  1. Article
  2. Review
  3. Ganglioside GM1 and the Central Nervous System.International journal of molecular sciences · 2023
    Review
  4. Article
  5. Review
  6. Article
  7. GM1 Gangliosidosis-A Mini-Review.Frontiers in genetics · 2021
    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

11 authors.

Michael J PrzybillaGene Therapy Center, Department of Pediatrics, University of Minnesota, Minneapolis, MN, USA.
Christine StewartResearch Department, Tanabe Research Laboratories U.S.A., Inc, San Diego, CA, USA.
Timothy W CarlsonComparative Pathology Shared Resource, University of Minnesota Masonic Cancer Center, Saint Paul, MN, USA.
Li OuGene Therapy Center, Department of Pediatrics, University of Minnesota, Minneapolis, MN, USA.
Brenda L KoniarCenter for Translational Medicine, Academic Health Center, University of Minnesota, Minneapolis, MN, USA.
Rohini SidhuDepartment of Medicine, Washington University School of Medicine, Saint Louis, MO, USA.
Pamela J KellDepartment of Medicine, Washington University School of Medicine, Saint Louis, MO, USA.
Xuntian JiangDepartment of Medicine, Washington University School of Medicine, Saint Louis, MO, USA.
Jeanine R JarnesDepartment of Experimental and Clinical Pharmacology, College of Pharmacy, University of Minnesota, Minneapolis, MN, USA.
M Gerard O'SullivanComparative Pathology Shared Resource, University of Minnesota Masonic Cancer Center, Saint Paul, MN, USA.
Chester B WhitleyGene Therapy Center, Department of Pediatrics, University of Minnesota, Minneapolis, MN, USA.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

GM1-gangliosidosis is a lysosomal disease resulting from a deficiency in the hydrolase β-galactosidase (β-gal) and subsequent accumulation of gangliosides, primarily in neuronal tissue, leading to progressive neurological deterioration and eventually early death. Lysosomal diseases with neurological involvement have limited non-invasive therapies due to the inability of lysosomal enzymes to cross the blood-brain barrier (BBB). A novel fusion enzyme, labeled mTfR-GLB1, was designed to act as a ferry across the BBB by fusing β-gal to the mouse monoclonal antibody against the mouse transferrin receptor and tested in a murine model of GM1-gangliosidosis (β-gal

Indexed as

Enzyme replacement therapyFusion enzymeGM1-gangliosidosis

Identifiers

PMID33854948
PMCPMC8025141

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.