Evidence map›Paper›PMID 37958544›Full record

ArticleInternational journal of molecular sciences2023

AAV-SPL 2.0, a Modified Adeno-Associated Virus Gene Therapy Agent for the Treatment of Sphingosine Phosphate Lyase Insufficiency Syndrome.

Ranjha Khan, Babak Oskouian, Joanna Y Lee, Jeffrey B Hodgin, Yingbao Yang, Gizachew Tassew, Julie D Saba

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 6 papers.

0numbers the graph read from it
0cells of the map it votes in
6citing papers in PubMed
2.3field-weighted citation impact, top 11% 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

6 citing papers in PubMed, 7 citations in OpenAlex.

  1. New therapeutic hope for rare podocytopathies.Pediatric nephrology (Berlin, Germany) · 2026
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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

7 authors at 2 institutions in 1 country.

Ranjha KhanDepartment of Pediatrics, Division of Hematology/Oncology, University of California, San Francisco, CA 94143, USA.
Babak OskouianDepartment of Pediatrics, Division of Hematology/Oncology, University of California, San Francisco, CA 94143, USA.
Joanna Y LeeDepartment of Pediatrics, Division of Hematology/Oncology, University of California, San Francisco, CA 94143, USA.
Jeffrey B HodginDepartment of Pathology, University of Michigan School of Medicine, Ann Arbor, MI 48109, USA.
Yingbao YangDepartment of Pathology, University of Michigan School of Medicine, Ann Arbor, MI 48109, USA.
Gizachew TassewDepartment of Pediatrics, Division of Hematology/Oncology, University of California, San Francisco, CA 94143, USA.
Julie D SabaDepartment of Pediatrics, Division of Hematology/Oncology, University of California, San Francisco, CA 94143, USA.ORCID 0000-0002-4894-919X
University of California, San Francisco · USUniversity of Michigan · US

Funding

California Institute of Regenerative Medicine DISC2-13072Swim Across America Foundation N/A
6 · The paper itself

Abstract

Sphingosine-1-phosphate lyase insufficiency syndrome (SPLIS) is an inborn error of metabolism caused by inactivating mutations in

Indexed as

Genetic TherapyParvovirinaeSphingosineAldehyde-LyasesAnimalsDependovirusHumansLysophospholipidsMiceMice, KnockoutPhosphatesAldehyde-LyasesLysophospholipidsPhosphatesSphingosinesphingosine 1-phosphate lyase (aldolase)AAV-SPL 2.0adeno-associated virusgene therapyinborn-error of metabolismSgpl1sphingolipidsphingosine phosphate lyase insufficiency syndrome

Identifiers

PMID37958544
PMCPMC10648410
OpenAlexW4387940066

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.