Evidence map›Paper›PMID 34485593›Full record

ArticleMolecular therapy. Methods & clinical development2021

Outcomes of progranulin gene therapy in the retina are dependent on time and route of delivery.

Emilia A Zin, Daisy Han, Jennifer Tran, Nikolas Morisson-Welch, Meike Visel, Mervi Kuronen, John G Flannery

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

Article in Molecular therapy. Methods & clinical development, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 3 papers.

0numbers the graph read from it
0cells of the map it votes in
3citing papers in PubMed
0.3field-weighted citation impact, top 39% 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

3 citing papers in PubMed, 4 citations in OpenAlex.

  1. Article
  2. Review
  3. 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

7 authors at 2 institutions in 1 country.

Emilia A ZinVision Science Group, School of Optometry, UC Berkeley, Berkeley, CA 94720, USA.
Daisy HanDepartment of Integrative Biology, UC Berkeley, Berkeley, CA 94720, USA.
Jennifer TranSchool of Optometry, UC Berkeley, Berkeley, CA 94720, USA.
Nikolas Morisson-WelchHelen Wills Neuroscience Institute, UC Berkeley, Berkeley, CA 94720, USA.
Meike ViselHelen Wills Neuroscience Institute, UC Berkeley, Berkeley, CA 94720, USA.
Mervi KuronenHelen Wills Neuroscience Institute, UC Berkeley, Berkeley, CA 94720, USA.
John G FlanneryVision Science Group, School of Optometry, UC Berkeley, Berkeley, CA 94720, USA.
University of California, Berkeley · USAcupuncture & Integrative Medicine College · US

Funding

A Zeiss LSM 710 confocal microscope for the Biological Imaging Facility at the US10RR026866 · NCRR · UNIVERSITY OF CALIFORNIA BERKELEY · PI RUZIN, STEVEN EARL · 2010 to 2010
$419k
NCRR NIH HHS S10 RR026866
6 · The paper itself

Abstract

Neuronal ceroid lipofuscinosis (NCL) is a family of neurodegenerative diseases caused by mutations to genes related to lysosomal function. One variant, CNL11, is caused by mutations to the gene encoding the protein progranulin, which regulates neuronal lysosomal function. Absence of progranulin causes cerebellar atrophy, seizures, dementia, and vision loss. As progranulin gene therapies targeting the brain are developed, it is advantageous to focus on the retina, as its characteristics are beneficial for gene therapy development: the retina is easily visible through direct imaging, can be assessed through quantitative methods

Indexed as

AAVneuronal ceroid lipofuscinosisprogranulinretina

Identifiers

PMID34485593
PMCPMC8390452
OpenAlexW3169973796

What OpenQuestion holds

Textmetadata
LicenceCC BY-NC-ND
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.