Evidence map›Paper›PMID 32443895›Full record

ArticleCells2020

Extracellular Vesicles as Drug Carriers for Enzyme Replacement Therapy to Treat CLN2 Batten Disease: Optimization of Drug Administration Routes.

Matthew J Haney, Yuling Zhao, Yeon S Jin, Elena V Batrakova

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

Article in Cells, 2020. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 23 papers.

0numbers the graph read from it
0cells of the map it votes in
23citing papers in PubMed
2.1field-weighted citation impact, top 12% 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

23 citing papers in PubMed, 37 citations in OpenAlex.

  1. Review
  2. Review
  3. Review
  4. Review
  5. Extracellular Vesicles as a Potential Therapy for Stroke.International journal of molecular sciences · 2025
    Review
  6. Article
  7. Review
  8. Review
  9. Review
  10. Review
  11. Article
  12. Article
  13. Review
  14. Article
  15. Review
  16. Article
  17. A kaleidoscopic view of extracellular vesicles in lysosomal storage disorders.Extracellular vesicles and circulating nucleic acids · 2022
    Review
  18. Article
  19. Article
  20. 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

4 authors at 1 institution in 1 country.

Matthew J HaneyCenter for Nanotechnology in Drug Delivery, University of North Carolina at Chapel Hill, Chapel Hill, NC 27599, USA.
Yuling ZhaoCenter for Nanotechnology in Drug Delivery, University of North Carolina at Chapel Hill, Chapel Hill, NC 27599, USA.
Yeon S JinEshelman School of Pharmacy, University of North Carolina at Chapel Hill, Chapel Hill, NC 27599, USA.
Elena V BatrakovaCenter for Nanotechnology in Drug Delivery, University of North Carolina at Chapel Hill, Chapel Hill, NC 27599, USA.
University of North Carolina at Chapel Hill · US

Funding

Cell-based Platform for Gene Delivery to the BrainR01NS102412 · NINDS · UNIV OF NORTH CAROLINA CHAPEL HILL · PI BATRAKOVA, ELENA · 2018 to 2022
$1.7M
Extracellular Vesicles for CNS Delivery of Therapeutic Enzymes to Treat Lysosomal Storage DisordersR01NS112019 · NINDS · UNIV OF NORTH CAROLINA CHAPEL HILL · PI BATRAKOVA, ELENA · 2019 to 2023
$1.5M
NINDS NIH HHS R01 NS102412NINDS NIH HHS R01 NS112019
6 · The paper itself

Abstract

CLN2 Batten disease (BD) is one of a broad class of lysosomal storage disorders that is characterized by the deficiency of lysosomal enzyme, TPP1, resulting in a build-up of toxic intracellular storage material in all organs and subsequent damage. A major challenge for BD therapeutics is delivery of enzymatically active TPP1 to the brain to attenuate progressive loss of neurological functions. To accomplish this daunting task, we propose the harnessing of naturally occurring nanoparticles, extracellular vesicles (EVs). Herein, we incorporated TPP1 into EVs released by immune cells, macrophages, and examined biodistribution and therapeutic efficacy of EV-TPP1 in BD mouse model, using various routes of administration. Administration through intrathecal and intranasal routes resulted in high TPP1 accumulation in the brain, decreased neurodegeneration and neuroinflammation, and reduced aggregation of lysosomal storage material in BD mouse model, CLN2 knock-out mice. Parenteral intravenous and intraperitoneal administrations led to TPP1 delivery to peripheral organs: liver, kidney, spleen, and lungs. A combination of intrathecal and intraperitoneal EV-TPP1 injections significantly prolonged lifespan in BD mice. Overall, the optimization of treatment strategies is crucial for successful applications of EVs-based therapeutics for BD.

Indexed as

Enzyme Replacement TherapyAminopeptidasesAnimalsBrainCells, CulturedDipeptidyl-Peptidases and Tripeptidyl-PeptidasesDrug Administration RoutesDrug CarriersExtracellular VesiclesHumansLuminescent MeasurementsMice, Inbred C57BLMice, KnockoutNeuronal Ceroid-LipofuscinosesNeuronsSerine ProteasesAminopeptidasesDipeptidyl-Peptidases and Tripeptidyl-PeptidasesDrug CarriersSerine ProteasesTPP1 protein, humanTpp1 protein, mouseTripeptidyl-Peptidase 1Batten diseasebrain bioavailabilitydrug deliveryextracellular vesiclesneuroinflammation

Identifiers

PMID32443895
PMCPMC7290714
OpenAlexW3026429179

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.