Evidence map›Paper›PMID 38934011›Full record

ArticleMolecular therapy. Methods & clinical development2024

A blood-brain barrier-penetrant AAV gene therapy improves neurological function in symptomatic mucolipidosis IV mice.

Madison L Sangster, Martha M Bishop, Yizheng Yao, Jessica F Feitor, Sanjid Shahriar, Maxwell E Miller, Anil K Chekuri, Bogdan Budnik, Fengfeng Bei, Yulia Grishchuk

Abstract read
PubMed Full text at PMC Publisher
In one paragraph

Article in Molecular therapy. Methods & clinical development, 2024. 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
–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

6 citing papers in PubMed.

  1. Article
  2. Article
  3. Article
  4. Exploring human plasma proteomic variations in mucolipidosis type IV.Molecular therapy. Methods & clinical development · 2025
    Article
  5. Review
  6. Toward a translational gene therapy for mucolipidosis IV.Molecular therapy. Methods & clinical development · 2024
    Article
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.

Madison L SangsterCenter for Genomic Medicine and Department of Neurology, Massachusetts General Hospital Research Institute and Harvard Medical School, 185 Cambridge St, Boston, MA 02114, USA.
Martha M BishopCenter for Genomic Medicine and Department of Neurology, Massachusetts General Hospital Research Institute and Harvard Medical School, 185 Cambridge St, Boston, MA 02114, USA.
Yizheng YaoDepartment of Neurosurgery, Brigham and Women's Hospital, Harvard Medical School, 75 Francis St, Boston, MA 02115, USA.
Jessica F FeitorWyss Institute for Biologically Inspired Engineering, Harvard University, 201 Brookline Avenue, Boston, MA 02215, USA.
Sanjid ShahriarWyss Institute for Biologically Inspired Engineering, Harvard University, 201 Brookline Avenue, Boston, MA 02215, USA.
Maxwell E MillerGrousbeck Gene Therapy Center, Schepens Eye Research Institute, Massachusetts Eye and Ear Infirmary, Harvard Medical School, 20 Staniford St, Boston, MA 02114, USA.
Anil K ChekuriGrousbeck Gene Therapy Center, Schepens Eye Research Institute, Massachusetts Eye and Ear Infirmary, Harvard Medical School, 20 Staniford St, Boston, MA 02114, USA.
Bogdan BudnikWyss Institute for Biologically Inspired Engineering, Harvard University, 201 Brookline Avenue, Boston, MA 02215, USA.
Fengfeng BeiDepartment of Neurosurgery, Brigham and Women's Hospital, Harvard Medical School, 75 Francis St, Boston, MA 02115, USA.
Yulia GrishchukCenter for Genomic Medicine and Department of Neurology, Massachusetts General Hospital Research Institute and Harvard Medical School, 185 Cambridge St, Boston, MA 02114, USA.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Mucolipidosis IV (MLIV) is a rare, autosomal recessive, lysosomal disease characterized by intellectual disability, motor deficits, and progressive vision loss. Using adeno-associated vector 9 (AAV9) and AAV-PHP.B as delivery vectors, we previously demonstrated the feasibility of modifying disease course in a mouse model of MLIV by the human

Indexed as

AAVadeno-associated vectorgene transferlysosomal disordermouse modelpediatric neurological diseaseTRPML1

Identifiers

PMID38934011
PMCPMC11201152

What OpenQuestion holds

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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.