Evidence map›Paper›PMID 27299292›Full record

ReviewAnnals of the New York Academy of Sciences2016

Transcription factor EB: from master coordinator of lysosomal pathways to candidate therapeutic target in degenerative storage diseases.

Marco Sardiello

Registry-linked trialOpen access · greenAbstract readReview
In one paragraph

Review in Annals of the New York Academy of Sciences, 2016. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT04808297 (A Study in Subjects With Neuronal Ceroid Lipofuscinoses Taking Trehalose), which is not on this map. Cited by 77 papers.

0numbers the graph read from it
0cells of the map it votes in
77citing papers in PubMed
11.2field-weighted citation impact, top 1% 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.

NCT04808297 completednot on this mapstarted 2020, after this paper: background citation

A Study in Subjects With Neuronal Ceroid Lipofuscinoses Taking Trehalose

TypeobservationalSponsorIRCCS Fondazione Stella MarisRan2020 to 2021Enrolled8ConditionsNeuronal Ceroid-Lipofuscinoses
3 · Its place in the literature

Who cites it

77 citing papers in PubMed, 130 citations in OpenAlex.

  1. Article
  2. Review
  3. Article
  4. Article
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  6. Article
  7. Cardiac dysfunction and altered gene expression in acid ceramidase-deficient mice.American journal of physiology. Heart and circulatory physiology · 2025
    Article
  8. Review
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17 more citing papers are in PubMed but not listed here.

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

1 author at 1 institution in 1 country.

Marco SardielloDepartment of Molecular and Human Genetics, Baylor College of Medicine, and Jan and Dan Duncan Neurological Research Institute at Texas Children's Hospital, Houston, Texas.
Neurological Research Institute · US

Funding

Modulation of lysosomal function for the treatment of neuronal ceroid lipofuscinoR01NS079618 · NINDS · WASHINGTON UNIVERSITY · PI SARDIELLO, MARCO · 2012 to 2021
$3.1M
Modulation of lysosomal function for the treatment of Batten diseaseR56NS079618 · NINDS · BAYLOR COLLEGE OF MEDICINE · PI SARDIELLO, MARCO · 2017 to 2017
$396k
NINDS NIH HHS R01 NS079618NINDS NIH HHS R56 NS079618
6 · The paper itself

Abstract

The lysosome is the main catabolic hub of the cell. Owing to its role in fundamental processes such as autophagy, plasma membrane repair, mTOR signaling, and maintenance of cellular homeostasis, the lysosome has a profound influence on cellular metabolism and human health. Indeed, inefficient or impaired lysosomal function has been implicated in the pathogenesis of a number of degenerative diseases affecting various organs and tissues, most notably the brain, liver, and muscle. The discovery of the coordinated lysosomal expression and regulation (CLEAR) genetic program and its master controller, transcription factor EB (TFEB), has provided an unprecedented tool to study and manipulate lysosomal function. Most lysosome-based processes-including macromolecule degradation, autophagy, lysosomal exocytosis, and proteostasis-are under the transcriptional control of TFEB. Interestingly, impaired TFEB signaling has been suggested to be a contributing factor in the pathogenesis of several degenerative storage diseases. Preclinical studies based on TFEB exogenous expression to reinstate TFEB activity or promote CLEAR network-based lysosomal enhancement have highlighted TFEB as a candidate therapeutic target for the treatment of various degenerative storage diseases.

Indexed as

Molecular Targeted TherapyAnimalsBasic Helix-Loop-Helix Leucine Zipper Transcription FactorsHumansLysosomal Storage DiseasesLysosomesModels, BiologicalSignal TransductionBasic Helix-Loop-Helix Leucine Zipper Transcription Factorsautophagydegenerative storage diseasesdegradative pathwayslysosome enhancementproteinopathiesTFEBtranscription factor EB

Identifiers

PMID27299292
PMCPMC5032832
OpenAlexW2436851548

What OpenQuestion holds

Textmetadata
LicenceTDM
Read underepoch 390

Registered trials

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.