Evidence map›Paper›PMID 33320673›Full record

ArticleMolecular pharmaceutics2021

Enzyme Replacement Therapy for Mucopolysaccharidosis IIID using Recombinant Human α-

Feng Wang, Derek R Moen, Chelsee Sauni, Shih-Hsin Kan, Shan Li, Steven Q Le, Brett Lomenick, Xiaoyi Zhang, Sean Ekins, Srikanth Singamsetty and 3 more

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

Article in Molecular pharmaceutics, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 10 papers.

0numbers the graph read from it
0cells of the map it votes in
10citing papers in PubMed
0.8field-weighted citation impact, top 26% 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

10 citing papers in PubMed, 17 citations in OpenAlex.

  1. Article
  2. Article
  3. Article
  4. Article
  5. Article
  6. Review
  7. Article
  8. Mucopolysaccharidoses and the blood-brain barrier.Fluids and barriers of the CNS · 2022
    Review
  9. The p97 Inhibitor UPCDC-30245 Blocks Endo-Lysosomal Degradation.Pharmaceuticals (Basel, Switzerland) · 2022
    Article
  10. 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

13 authors at 5 institutions in 1 country.

Feng WangDivision of Medical Genetics, Department of Pediatrics, Harbor-UCLA Medical Center and The Lundquist Institute, Torrance, California 90502, United States.
Derek R MoenPhoenix Nest Inc., Brooklyn, New York 11232, United States.
Chelsee SauniPhoenix Nest Inc., Brooklyn, New York 11232, United States.
Shih-Hsin KanDivision of Medical Genetics, Department of Pediatrics, Harbor-UCLA Medical Center and The Lundquist Institute, Torrance, California 90502, United States.
Shan LiDivision of Medical Genetics, Department of Pediatrics, Harbor-UCLA Medical Center and The Lundquist Institute, Torrance, California 90502, United States.
Steven Q LeDivision of Medical Genetics, Department of Pediatrics, Harbor-UCLA Medical Center and The Lundquist Institute, Torrance, California 90502, United States.
Brett LomenickProteome Exploration Laboratory, Beckman Institute, California Institute of Technology, Pasadena, California 91125, United States.
Xiaoyi ZhangDivision of Medical Genetics, Department of Pediatrics, Harbor-UCLA Medical Center and The Lundquist Institute, Torrance, California 90502, United States.
Sean EkinsPhoenix Nest Inc., Brooklyn, New York 11232, United States.ORCID 0000-0002-5691-5790
Srikanth SingamsettyPhoenix Nest Inc., Brooklyn, New York 11232, United States.
Jill WoodPhoenix Nest Inc., Brooklyn, New York 11232, United States.
Patricia I DicksonDivision of Medical Genetics, Department of Pediatrics, Harbor-UCLA Medical Center and The Lundquist Institute, Torrance, California 90502, United States.
Tsui-Fen ChouDivision of Medical Genetics, Department of Pediatrics, Harbor-UCLA Medical Center and The Lundquist Institute, Torrance, California 90502, United States.ORCID 0000-0003-2410-2186
Phoenix Nest (United States) · USCalifornia Institute of Technology · USWashington University in St. Louis · USChildren's Hospital of Orange County · USUCLA Medical Center · US

Funding

UCLA Medical Genetics Training ProgramT32GM008243 · NIGMS · UNIVERSITY OF CALIFORNIA LOS ANGELES · PI Julian Martinez-Agosto, STANLEY F. NELSON · 1987 to 2026
$6.8M
Development and Validation of Therapy for Mucopolysaccharidosis IIIU44NS089061 · NINDS · PHOENIX NEST, INC. · PI CHOU, TSUI-FEN, DICKSON, PATRICIA I · 2018 to 2022
$5.6M
Phenotypic Effects of Brain-Directed Enzyme Therapy for Sanfilippo B SyndromeR01NS088766 · NINDS · WASHINGTON UNIVERSITY · PI DICKSON, PATRICIA I · 2015 to 2019
$1.7M
Development and validation of therapy for mucopolysaccharidosis IIIR42NS089061 · NINDS · PHOENIX NEST, INC. · PI EKINS, SEAN · 2016 to 2017
$1.5M
Development and in vitro validation of therapy for mucopolysaccharidosis IIIR41NS089061 · NINDS · PHOENIX NEST, INC. · PI EKINS, SEAN · 2014 to 2015
$225k
NIGMS NIH HHS T32 GM008243NINDS NIH HHS R01 NS088766NINDS NIH HHS R41 NS089061NINDS NIH HHS R42 NS089061NINDS NIH HHS U44 NS089061
6 · The paper itself

Abstract

There is currently no cure or effective treatment available for mucopolysaccharidosis type IIID (MPS IIID, Sanfilippo syndrome type D), a lysosomal storage disorder (LSD) caused by the deficiency of α-

Indexed as

AnimalsAnimals, NewbornBrainCHO CellsCricetulusDisease Models, AnimalEnzyme Replacement TherapyGlycosaminoglycansHeparan SulfateHumansLiverLysosomal Storage DiseasesLysosomesMiceMucopolysaccharidosis IIINeuronsGlycosaminoglycansHeparan SulfateN-acetylglucosamine-6-sulfataseReceptor, IGF Type 2Recombinant ProteinsSulfatasesCHO cellsDHFRenzyme replacement therapyGNSMPS IIIDrecombinant protein production

Identifiers

PMID33320673
PMCPMC8362844
OpenAlexW3111786821

What OpenQuestion holds

Textmetadata
LicenceTDM
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.