Evidence map›Paper›PMID 37298170›Full record

ArticleInternational journal of molecular sciences2023

Biochemical Correction of GM2 Ganglioside Accumulation in AB-Variant GM2 Gangliosidosis.

Natalie M Deschenes, Camilyn Cheng, Alex E Ryckman, Brianna M Quinville, Prem Khanal, Melissa Mitchell, Zhilin Chen, Waheed Sangrar, Steven J Gray, Jagdeep S Walia

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

Article in International journal of molecular sciences, 2023. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 5 papers.

0numbers the graph read from it
0cells of the map it votes in
5citing papers in PubMed
1.9field-weighted citation impact, top 13% 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

5 citing papers in PubMed, 6 citations in OpenAlex.

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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 2 institutions in 2 countries.

Natalie M DeschenesCentre for Neuroscience Studies, Queen's University, Kingston, ON K7L 3N6, Canada.ORCID 0000-0003-2306-2661
Camilyn ChengCentre for Neuroscience Studies, Queen's University, Kingston, ON K7L 3N6, Canada.
Alex E RyckmanCentre for Neuroscience Studies, Queen's University, Kingston, ON K7L 3N6, Canada.
Brianna M QuinvilleCentre for Neuroscience Studies, Queen's University, Kingston, ON K7L 3N6, Canada.
Prem KhanalDepartment of Pediatrics, Queen's University, Kingston, ON K7L 2V7, Canada.
Melissa MitchellDepartment of Pediatrics, Queen's University, Kingston, ON K7L 2V7, Canada.
Zhilin ChenDepartment of Biomedical and Molecular Sciences, Queen's University, Kingston, ON K7L 3N6, Canada.
Waheed SangrarCentre for Neuroscience Studies, Queen's University, Kingston, ON K7L 3N6, Canada.
Steven J GrayDepartment of Pediatrics, UT Southwestern Medical Center, Dallas, TX 75390, USA.ORCID 0000-0002-6240-8621
Jagdeep S WaliaCentre for Neuroscience Studies, Queen's University, Kingston, ON K7L 3N6, Canada.
Queen's University · CASouthwestern Medical Center · US

Funding

Cure Tay-Sachs Foundation 1079923GlycoNet ND06
6 · The paper itself

Abstract

GM2 gangliosidosis is a group of genetic disorders that result in the accumulation of GM2 ganglioside (GM2) in brain cells, leading to progressive central nervous system (CNS) atrophy and premature death in patients. AB-variant GM2 gangliosidosis (ABGM2) arises from loss-of-function mutations in the GM2 activator protein (GM2AP), which is essential for the breakdown of GM2 in a key catabolic pathway required for CNS lipid homeostasis. In this study, we show that intrathecal delivery of self-complementary adeno-associated virus serotype-9 (scAAV9) harbouring a functional human

Indexed as

Gangliosidoses, GM2G(M2) GangliosideAnimalsBrainCentral Nervous SystemG(M2) Activator ProteinHumansMiceMutationG(M2) Activator ProteinG(M2) GangliosideAAV9adeno-associated viral vectorsdose-responsegene therapyGM2 activator proteinGM2 gangliosideGM2 gangliosidosisintrathecalneurodegeneration

Identifiers

PMID37298170
PMCPMC10253223
OpenAlexW4378228074

What Socratic 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 Socratic graph.