Evidence map›Paper›PMID 36010656›Full record

ArticleCells2022

Preclinical Enzyme Replacement Therapy with a Recombinant β-Galactosidase-Lectin Fusion for CNS Delivery and Treatment of GM1-Gangliosidosis.

Jason Andrew Weesner, Ida Annunziata, Tianhong Yang, Walter Acosta, Elida Gomero, Huimin Hu, Diantha van de Vlekkert, Jorge Ayala, Xiaohui Qiu, Leigh Ellen Fremuth and 3 more

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

Article in Cells, 2022. 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
1.4field-weighted citation impact, top 18% 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, 13 citations in OpenAlex.

  1. Review
  2. Review
  3. Article
  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

13 authors at 2 institutions in 1 country.

Jason Andrew WeesnerDepartment of Genetics, St. Jude Children's Research Hospital, Memphis, TN 38105, USA.ORCID 0000-0002-0630-1715
Ida AnnunziataDepartment of Genetics, St. Jude Children's Research Hospital, Memphis, TN 38105, USA.
Tianhong YangBioStrategies, LC, P.O. Box 2428, State University, Jonesboro, AR 72467, USA.
Walter AcostaBioStrategies, LC, P.O. Box 2428, State University, Jonesboro, AR 72467, USA.ORCID 0000-0002-5525-306X
Elida GomeroDepartment of Genetics, St. Jude Children's Research Hospital, Memphis, TN 38105, USA.
Huimin HuDepartment of Genetics, St. Jude Children's Research Hospital, Memphis, TN 38105, USA.ORCID 0000-0003-2107-5389
Diantha van de VlekkertDepartment of Genetics, St. Jude Children's Research Hospital, Memphis, TN 38105, USA.ORCID 0000-0001-7498-1292
Jorge AyalaBioStrategies, LC, P.O. Box 2428, State University, Jonesboro, AR 72467, USA.
Xiaohui QiuDepartment of Genetics, St. Jude Children's Research Hospital, Memphis, TN 38105, USA.
Leigh Ellen FremuthDepartment of Genetics, St. Jude Children's Research Hospital, Memphis, TN 38105, USA.
David N RadinBioStrategies, LC, P.O. Box 2428, State University, Jonesboro, AR 72467, USA.
Carole L CramerBioStrategies, LC, P.O. Box 2428, State University, Jonesboro, AR 72467, USA.
Alessandra d'AzzoDepartment of Genetics, St. Jude Children's Research Hospital, Memphis, TN 38105, USA.ORCID 0000-0003-1747-7502
St. Jude Children's Research Hospital · USBioStrategies (United States) · US

Funding

Enzyme replacement therapy for GM1 gangliosidosis lysosomal rare diseaseR44NS084565 · NINDS · BIOSTRATEGIES, LC · PI ACOSTA, WALTER, CRAMER, CAROLE L. · 2017 to 2018
$2.4M
NINDS NIH HHS R44 NS084565
6 · The paper itself

Abstract

GM1-gangliosidosis is a catastrophic, neurodegenerative lysosomal storage disease caused by a deficiency of lysosomal β-galactosidase (β-Gal). The primary substrate of the enzyme is GM1-ganglioside (GM1), a sialylated glycosphingolipid abundant in nervous tissue. Patients with GM1-gangliosidosis present with massive and progressive accumulation of GM1 in the central nervous system (CNS), which leads to mental and motor decline, progressive neurodegeneration, and early death. No therapy is currently available for this lysosomal storage disease. Here, we describe a proof-of-concept preclinical study toward the development of enzyme replacement therapy (ERT) for GM1-gangliosidosis using a recombinant murine β-Gal fused to the plant lectin subunit B of ricin (mβ-Gal:RTB). We show that long-term, bi-weekly systemic injection of mβ-Gal:RTB in the

Indexed as

Gangliosidosis, GM1G(M1) GangliosideAnimalsbeta-GalactosidaseCentral Nervous SystemEnzyme Replacement TherapyLectinsMiceTissue Distributionbeta-GalactosidaseG(M1) GangliosideLectinsCNSERTGM1lysosomal storage diseasemβ-Gal:RTB

Identifiers

PMID36010656
PMCPMC9406850
OpenAlexW4292546382

What OpenQuestion holds

Textmetadata
LicenceCC BY
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.