Evidence map›Paper›PMID 34869463›Full record

ReviewFrontiers in medicine2021

Krabbe Disease: Prospects of Finding a Cure Using AAV Gene Therapy.

Gibran Nasir, Rajiv Chopra, Fiona Elwood, Seemin S Ahmed

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

Review in Frontiers in medicine, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 9 papers, 1 of them a synthesis that pooled it.

0numbers the graph read from it
0cells of the map it votes in
9citing papers in PubMed, 1 pooled it
1.1field-weighted citation impact, top 23% 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

9 citing papers in PubMed, 1 synthesis or guideline pooled it, 12 citations in OpenAlex.

  1. Pooled it
  2. Review
  3. Article
  4. Article
  5. Article
  6. Review
  7. Neurodegenerative Disorder Risk in Krabbe Disease Carriers.International journal of molecular sciences · 2022
    Article
  8. Review
  9. 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

4 authors.

Gibran NasirDepartment of Neuroscience, Novartis Institutes for BioMedical Research (NIBR), Cambridge, MA, United States.
Rajiv ChopraAllianThera Biopharma, Boston, MA, United States.
Fiona ElwoodDepartment of Neuroscience, Novartis Institutes for BioMedical Research (NIBR), Cambridge, MA, United States.
Seemin S AhmedDepartment of Neuroscience, Novartis Institutes for BioMedical Research (NIBR), Cambridge, MA, United States.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Krabbe Disease (KD) is an autosomal metabolic disorder that affects both the central and peripheral nervous systems. It is caused by a functional deficiency of the lysosomal enzyme, galactocerebrosidase (GALC), resulting in an accumulation of the toxic metabolite, psychosine. Psychosine accumulation affects many different cellular pathways, leading to severe demyelination. Although there is currently no effective therapy for Krabbe disease, recent gene therapy-based approaches in animal models have indicated a promising outlook for clinical treatment. This review highlights recent findings in the pathogenesis of Krabbe disease, and evaluates AAV-based gene therapy as a promising strategy for treating this devastating pediatric disease.

Indexed as

adeno-associated virusgalactocerebrosidasegene therapyKrabbe disease (globoid cell leukodystrophy)leukodystrophiespsychosine

Identifiers

PMID34869463
PMCPMC8633897
OpenAlexW3213872789

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.