ReviewInternational journal of molecular sciences2025
An AAV-Based Therapy Approach for Neurological Phenotypes of X-Linked Adrenoleukodystrophy.
Ekaterina Gornostal, Almaqdad Alsalloum, Egor Degtyarev, Ekaterina Kuznetsova, Aygun Levashova, Daria Mishina, Natalia Mingaleva, Ali Mazloum, Viktor Bogdanov, Julia Krupinova and 4 more
Abstract readReview
In one paragraphReview in International journal of molecular sciences, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.
0numbers the graph read from it
0cells of the map it votes in
0citing papers in PubMed
–field-weighted citation impact
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 registryThe 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 literatureWho cites it
0 citing papers in PubMed.
No citing paper in PubMed yet.
4 · The recordCorrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
5 · Who and what moneyAuthors and funding
14 authors.
Ekaterina GornostalFederal State Budgetary Scientific Institution "Federal Research Center for Innovator and Emerging Biomedical and Pharmaceutical Technologies", 125315 Moscow, Russia.ORCID 0000-0002-3708-5891 Almaqdad AlsalloumFederal State Budgetary Scientific Institution "Federal Research Center for Innovator and Emerging Biomedical and Pharmaceutical Technologies", 125315 Moscow, Russia.ORCID 0000-0002-9698-0634 Egor DegtyarevFederal State Budgetary Scientific Institution "Federal Research Center for Innovator and Emerging Biomedical and Pharmaceutical Technologies", 125315 Moscow, Russia.
Ekaterina KuznetsovaFederal State Budgetary Scientific Institution "Federal Research Center for Innovator and Emerging Biomedical and Pharmaceutical Technologies", 125315 Moscow, Russia.
Aygun LevashovaFederal State Budgetary Scientific Institution "Federal Research Center for Innovator and Emerging Biomedical and Pharmaceutical Technologies", 125315 Moscow, Russia.
Daria MishinaFederal State Budgetary Scientific Institution "Federal Research Center for Innovator and Emerging Biomedical and Pharmaceutical Technologies", 125315 Moscow, Russia.
Natalia MingalevaFederal State Budgetary Scientific Institution "Federal Research Center for Innovator and Emerging Biomedical and Pharmaceutical Technologies", 125315 Moscow, Russia.
Ali MazloumFederal State Budgetary Scientific Institution "Federal Research Center for Innovator and Emerging Biomedical and Pharmaceutical Technologies", 125315 Moscow, Russia.ORCID 0000-0002-5982-8393 Viktor BogdanovFederal State Budgetary Scientific Institution "Federal Research Center for Innovator and Emerging Biomedical and Pharmaceutical Technologies", 125315 Moscow, Russia.ORCID 0000-0001-6377-9056 Julia KrupinovaFederal State Budgetary Scientific Institution "Federal Research Center for Innovator and Emerging Biomedical and Pharmaceutical Technologies", 125315 Moscow, Russia.
Sergey MikhalkovMoscow Center for Advanced Studies, Kulakova Str. 20, 123592 Moscow, Russia.
Irina RybkinaDepartment of Endocrinology, Morozov Children's Clinical Hospital, 119049 Moscow, Russia.
Olga MityaevaFederal State Budgetary Scientific Institution "Federal Research Center for Innovator and Emerging Biomedical and Pharmaceutical Technologies", 125315 Moscow, Russia.ORCID 0000-0001-9252-1601 Pavel VolchkovFederal State Budgetary Scientific Institution "Federal Research Center for Innovator and Emerging Biomedical and Pharmaceutical Technologies", 125315 Moscow, Russia.
Funding
Russian Science Foundation 23 64-00002
6 · The paper itselfAbstract
X-linked adrenoleukodystrophy (X-ALD) is a monogenic progressive neurodegenerative disorder, being simultaneously a systemic metabolic disease and demonstrating severe neurological manifestations with effects to the brain and spinal cord. The objective of the current review is to provide a detailed approach to adeno-associated virus (AAV)-based gene therapy for neurological manifestations of X-ALD. The development of a successful AAV-mediated gene therapy hinges on its ability to deliver
Indexed as
AdrenoleukodystrophyDependovirusGenetic TherapyGenetic VectorsAnimalsATP Binding Cassette Transporter, Subfamily D, Member 1HumansPhenotypeABCD1 protein, humanATP Binding Cassette Transporter, Subfamily D, Member 1AAVdemyelinating disordersgene therapyX-ALDX-linked adrenoleukodystrophy
Identifiers
PMID41373793
PMCPMC12691817
What OpenQuestion holds
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LicenceCC BY
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