Evidence map›Paper›PMID 32637448›Full record

ArticleMolecular therapy. Methods & clinical development2020

Cerebral Organoids: A Human Model for AAV Capsid Selection and Therapeutic Transgene Efficacy in the Brain.

Josse A Depla, Marina Sogorb-Gonzalez, Lance A Mulder, Vivi M Heine, Pavlina Konstantinova, Sander J van Deventer, Katja C Wolthers, Dasja Pajkrt, Adithya Sridhar, Melvin M Evers

Open access · goldAbstract read
In one paragraph

Article in Molecular therapy. Methods & clinical development, 2020. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 27 papers, 2 of them syntheses that pooled it.

0numbers the graph read from it
0cells of the map it votes in
27citing papers in PubMed, 2 pooled it
1.8field-weighted citation impact, top 14% 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

27 citing papers in PubMed, 2 syntheses or guidelines pooled it, 49 citations in OpenAlex.

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  12. In vivo selection in non-human primates identifies AAV capsids for on-target CSF delivery to spinal cord.Molecular therapy : the journal of the American Society of Gene Therapy · 2024
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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 4 institutions in 1 country.

Josse A DeplaDepartment of Research & Development, uniQure Biopharma B.V., Amsterdam, the Netherlands.
Marina Sogorb-GonzalezDepartment of Research & Development, uniQure Biopharma B.V., Amsterdam, the Netherlands.
Lance A MulderDepartment of Medical Microbiology, Laboratory of Clinical Virology, Amsterdam UMC, Amsterdam, the Netherlands.
Vivi M HeineDepartment of Complex Trait Genetics, Center for Neurogenomics and Cognitive Research, Amsterdam Neuroscience, Vrije Universiteit, Amsterdam, the Netherlands.
Pavlina KonstantinovaDepartment of Research & Development, uniQure Biopharma B.V., Amsterdam, the Netherlands.
Sander J van DeventerDepartment of Research & Development, uniQure Biopharma B.V., Amsterdam, the Netherlands.
Katja C WolthersDepartment of Medical Microbiology, Laboratory of Clinical Virology, Amsterdam UMC, Amsterdam, the Netherlands.
Dasja PajkrtDepartment of Pediatric Infectious Diseases, Emma Children's Hospital, Amsterdam UMC, Amsterdam, the Netherlands.
Adithya SridharDepartment of Medical Microbiology, Laboratory of Clinical Virology, Amsterdam UMC, Amsterdam, the Netherlands.
Melvin M EversDepartment of Research & Development, uniQure Biopharma B.V., Amsterdam, the Netherlands.
Amsterdam University Medical Centers · NLUniQure (Netherlands) · NLAmsterdam Neuroscience · NLLeiden University Medical Center · NL

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

The development of gene therapies for central nervous system disorders is challenging because it is difficult to translate preclinical data from current

Indexed as

AAVadeno-associated viruscentral nervous systemCerebral organoidsCNSgene therapyinduced pluripotent stem cellsiPSCmicro RNAmiRNAorganoidsSCA3spinocerebellar ataxia 3Transduction

Identifiers

PMID32637448
PMCPMC7327852
OpenAlexW3033277640

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.