Evidence map›Paper›PMID 36419468›Full record

ArticleMolecular therapy. Methods & clinical development2022

Brain transplantation of genetically corrected Sanfilippo type B neural stem cells induces partial cross-correction of the disease.

Yewande Pearse, Don Clarke, Shih-Hsin Kan, Steven Q Le, Valentina Sanghez, Anna Luzzi, Ivy Pham, Lina R Nih, Jonathan D Cooper, Patricia I Dickson and 1 more

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

Article in Molecular therapy. Methods & clinical development, 2022. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 5 papers.

0numbers the graph read from it
0cells of the map it votes in
5citing papers in PubMed
0.6field-weighted citation impact, top 33% 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

5 citing papers in PubMed, 5 citations in OpenAlex.

  1. Article
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  5. 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

11 authors at 3 institutions in 1 country.

Yewande PearseDepartment of Pediatrics, the Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center, Torrance, CA 90502, USA.
Don ClarkeDepartment of Pediatrics, the Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center, Torrance, CA 90502, USA.
Shih-Hsin KanDepartment of Pediatrics, the Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center, Torrance, CA 90502, USA.
Steven Q LeDepartment of Pediatrics, the Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center, Torrance, CA 90502, USA.
Valentina SanghezDepartment of Pediatrics, the Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center, Torrance, CA 90502, USA.
Anna LuzziDepartment of Pediatrics, the Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center, Torrance, CA 90502, USA.
Ivy PhamDepartment of Neurology, the Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center, Torrance, CA 90502, USA.
Lina R NihDepartment of Pediatrics, the Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center, Torrance, CA 90502, USA.
Jonathan D CooperDepartment of Pediatrics, Washington University, Saint Louis, MO 63110, USA.
Patricia I DicksonDepartment of Pediatrics, Washington University, Saint Louis, MO 63110, USA.
Michelina IacovinoDepartment of Pediatrics, the Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center, Torrance, CA 90502, USA.
UCLA Medical Center · USUniversity of California, Los Angeles · USWashington University in St. Louis · US

Funding

WUIDDRC Supplement-Supporting the health and well-being of children with intellectual and developmental disability during COVID-19 pandemicP50HD103525 · NICHD · WASHINGTON UNIVERSITY · PI JOSEPH D DOUGHERTY, Natasha Marrus · 2020 to 2026
$15.5M
UCLA Medical Genetics Training ProgramT32GM008243 · NIGMS · UNIVERSITY OF CALIFORNIA LOS ANGELES · PI Julian Martinez-Agosto, STANLEY F. NELSON · 1987 to 2026
$6.8M
Phenotypic Effects of Brain-Directed Enzyme Therapy for Sanfilippo B SyndromeR01NS088766 · NINDS · WASHINGTON UNIVERSITY · PI DICKSON, PATRICIA I · 2015 to 2019
$1.7M
Modified neural stem cells for glycosylation-independent treatment of MPS IIIBR33NS096044 · NINDS · LUNDQUIST INSTITUTE FOR BIOMEDICAL INNOVATION AT HARBOR-UCLA MEDICAL CENTER · PI IACOVINO, MICHELINA · 2019 to 2019
$576k
NICHD NIH HHS P50 HD103525NIGMS NIH HHS T32 GM008243NINDS NIH HHS R01 NS088766NINDS NIH HHS R33 NS096044
6 · The paper itself

Abstract

Sanfilippo syndrome type B (mucopolysaccharidosis type IIIB) is a recessive genetic disorder that severely affects the brain due to a deficiency in the enzyme α-

Indexed as

cell therapyLSDMPSneural progenitor cellsSanfilippo type B

Identifiers

PMID36419468
PMCPMC9672419
OpenAlexW4307721544

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.