Evidence map›Paper›PMID 29916295›Full record

ArticleAutophagy2018

Trehalose reduces retinal degeneration, neuroinflammation and storage burden caused by a lysosomal hydrolase deficiency.

Parisa Lotfi, Dennis Y Tse, Alberto Di Ronza, Michelle L Seymour, Giuseppe Martano, Jonathan D Cooper, Fred A Pereira, Maria Passafaro, Samuel M Wu, Marco Sardiello

Open access · bronzeAbstract read
In one paragraph

Article in Autophagy, 2018. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 59 papers, 2 of them syntheses that pooled it.

0numbers the graph read from it
0cells of the map it votes in
59citing papers in PubMed, 2 pooled it
10.6field-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.

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

59 citing papers in PubMed, 2 syntheses or guidelines pooled it, 105 citations in OpenAlex.

  1. Pooled it
  2. Guideline
  3. Article
  4. Trehalose's untapped mechanisms in alzheimer's: gut-brain-autophagy signalling beyond the usual targets.Daru : journal of Faculty of Pharmacy, Tehran University of Medical Sciences · 2026
    Review
  5. Article
  6. Review
  7. Article
  8. Article
  9. Article
  10. Review
  11. Review
  12. Article
  13. Article
  14. Review
  15. Article
  16. Lysosomes in retinal health and disease.Trends in neurosciences · 2023
    Review
  17. Review
  18. Review
  19. Bicalutamide and Trehalose Ameliorate Spinal and Bulbar Muscular Atrophy Pathology in Mice.Neurotherapeutics : the journal of the American Society for Experimental NeuroTherapeutics · 2023
    Article
  20. 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 at 6 institutions in 4 countries.

Parisa Lotfia Department of Molecular and Human Genetics , Baylor College of Medicine, Jan and Dan Duncan Neurological Research Institute at Texas Children's Hospital , Houston , TX , USA.
Dennis Y Tseb Department of Ophthalmology , Cullen Eye Institute, Baylor College of Medicine , Houston , TX , USA.
Alberto Di Ronzaa Department of Molecular and Human Genetics , Baylor College of Medicine, Jan and Dan Duncan Neurological Research Institute at Texas Children's Hospital , Houston , TX , USA.
Michelle L Seymourd Huffington Center on Aging, Department of Molecular and Cellular Biology , Baylor College of Medicine , Houston , TX , USA.
Giuseppe Martanof CNR Institute of Neuroscience , Milan , Italy.
Jonathan D Cooperg Department of Basic and Clinical Neuroscience , Maurice Wohl Clinical Neuroscience Institute, Institute of Psychiatry, Psychology and Neuroscience , Kings College London , London , UK.
Fred A Pereirad Huffington Center on Aging, Department of Molecular and Cellular Biology , Baylor College of Medicine , Houston , TX , USA.
Maria Passafarof CNR Institute of Neuroscience , Milan , Italy.
Samuel M Wub Department of Ophthalmology , Cullen Eye Institute, Baylor College of Medicine , Houston , TX , USA.
Marco Sardielloa Department of Molecular and Human Genetics , Baylor College of Medicine, Jan and Dan Duncan Neurological Research Institute at Texas Children's Hospital , Houston , TX , USA.
Baylor College of Medicine · USNeuroscience Institute · ITTexas Children's Hospital · USHong Kong Polytechnic University · HKKing's College London · GBNeurological Research Institute · US

Funding

VISION RESEARCH CENTERP30EY002520 · NEI · BAYLOR COLLEGE OF MEDICINE · PI Samuel M Wu · 1985 to 2026
$14.8M
MEDICAL GENETICS RESEARCH FELLOWSHIP PROGRAMT32GM007526 · NIGMS · BAYLOR COLLEGE OF MEDICINE · PI Brendan Lee · 1985 to 2026
$11.4M
Steps towards a paternal gene activation therapy for Angelman syndromeU54HD083092 · NICHD · BAYLOR COLLEGE OF MEDICINE · PI NELSON, DAVID LOREN, NEUL, JEFFREY L · 2014 to 2019
$7.8M
Mutidisciplinary Training;Brain Disorders &DevelopmentT32NS043124 · NINDS · BAYLOR COLLEGE OF MEDICINE · PI SWANN, JOHN WILLIAM · 2002 to 2021
$4.6M
ROD AND CONE SIGNALING PATHWAYS IN MAMMALIAN RETINAR01EY019908 · NEI · BAYLOR COLLEGE OF MEDICINE · PI WU, SAMUEL M · 2010 to 2020
$3.8M
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
Tecan EVO GenePaint robotS10OD016167 · OD · BAYLOR COLLEGE OF MEDICINE · PI LJUNGBERG, CECILIA · 2013 to 2013
$215k
NEI NIH HHS P30 EY002520NEI NIH HHS R01 EY019908NICHD NIH HHS U54 HD083092NIGMS NIH HHS T32 GM007526NIH HHS S10 OD016167NINDS NIH HHS R01 NS079618NINDS NIH HHS R56 NS079618NINDS NIH HHS T32 NS043124
6 · The paper itself

Abstract

The accumulation of undegraded molecular material leads to progressive neurodegeneration in a number of lysosomal storage disorders (LSDs) that are caused by functional deficiencies of lysosomal hydrolases. To determine whether inducing macroautophagy/autophagy via small-molecule therapy would be effective for neuropathic LSDs due to enzyme deficiency, we treated a mouse model of mucopolysaccharidosis IIIB (MPS IIIB), a storage disorder caused by deficiency of the enzyme NAGLU (alpha-N-acetylglucosaminidase [Sanfilippo disease IIIB]), with the autophagy-inducing compound trehalose. Treated naglu ABBREVIATIONS: ANOVA: analysis of variance; Atg7: autophagy related 7; AV: autophagic vacuoles; CD68: cd68 antigen; ERG: electroretinogram; ERT: enzyme replacement therapy; GAPDH: glyceraldehyde-3-phosphate dehydrogenase; GFAP: glial fibrillary acidic protein; GNAT2: guanine nucleotide binding protein, alpha transducing 2; HSCT: hematopoietic stem cell transplantation; INL: inner nuclear layer; LC3: microtubule-associated protein 1 light chain 3 alpha; MPS: mucopolysaccharidoses; NAGLU: alpha-N-acetylglucosaminidase (Sanfilippo disease IIIB); ONL: outer nuclear layer; PBS: phosphate-buffered saline; PRKCA/PKCα: protein kinase C, alpha; S1BF: somatosensory cortex; SQSTM1: sequestosome 1; TEM: transmission electron microscopy; TFEB: transcription factor EB; VMP/VPL: ventral posterior nuclei of the thalamus.

Indexed as

Disease ProgressionAcetylglucosaminidaseAnimalsAstrocytesAutophagyBasic Helix-Loop-Helix Leucine Zipper Transcription FactorsBrainCell NucleusGene Regulatory NetworksInflammationLysosomesMice, Inbred C57BLMice, KnockoutMucopolysaccharidosis IIIRetinal Bipolar CellsRetinal DegenerationAcetylglucosaminidasealpha-N-acetyl-D-glucosaminidaseBasic Helix-Loop-Helix Leucine Zipper Transcription FactorsTcfeb protein, mouseTrehaloseAutophagic vacuolesautophagyERGlysosomal storage diseasesMPS IIIBmucopolysaccharidosis type IIIBretinaSanfilippo syndromeTFEBtrehalose

Identifiers

PMID29916295
PMCPMC6103706
OpenAlexW2808836146

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.