Evidence map›Paper›PMID 39687731›Full record

ArticleMolecular therapy. Methods & clinical development2024

Intrathecal or intravenous AAV9-IDUA/RGX-111 at minimal effective dose prevents cardiac, skeletal and neurologic manifestations of murine MPS I.

Lalitha R Belur, Avery K Huber, Hillary Mantone, Mason Robertson, Miles C Smith, Andrea D Karlen, Kelley F Kitto, Li Ou, Chester B Whitley, Elizabeth Braunlin and 7 more

Abstract read
PubMed Full text at PMC Publisher
In one paragraph

Article in Molecular therapy. Methods & clinical development, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 4 papers.

0numbers the graph read from it
0cells of the map it votes in
4citing papers in PubMed
–field-weighted citation impact
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

4 citing papers in PubMed.

  1. Article
  2. Advances in Therapies for Mucopolysaccharidoses.Current issues in molecular biology · 2026
    Review
  3. Review
  4. Advancing AAV technology-From promise to product.Molecular therapy. Advances · 2026
    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

17 authors.

Lalitha R BelurCenter for Genome Engineering, Department of Genetics, Cell Biology and Development, University of Minnesota, Minneapolis, MN, USA.
Avery K HuberCenter for Genome Engineering, Department of Genetics, Cell Biology and Development, University of Minnesota, Minneapolis, MN, USA.
Hillary MantoneCenter for Genome Engineering, Department of Genetics, Cell Biology and Development, University of Minnesota, Minneapolis, MN, USA.
Mason RobertsonCenter for Genome Engineering, Department of Genetics, Cell Biology and Development, University of Minnesota, Minneapolis, MN, USA.
Miles C SmithCenter for Genome Engineering, Department of Genetics, Cell Biology and Development, University of Minnesota, Minneapolis, MN, USA.
Andrea D KarlenCenter for Genome Engineering, Department of Genetics, Cell Biology and Development, University of Minnesota, Minneapolis, MN, USA.
Kelley F KittoDepartment of Pharmaceutics, University of Minnesota, Minneapolis MN, USA.
Li OuDepartment of Pediatrics, University of Minnesota, Minneapolis, MN, USA.
Chester B WhitleyDepartment of Pediatrics, University of Minnesota, Minneapolis, MN, USA.
Elizabeth BraunlinDepartment of Pediatrics, University of Minnesota, Minneapolis, MN, USA.
Justin FurcichDepartment of Pediatrics, University of Minnesota, Minneapolis, MN, USA.
Troy C LundDepartment of Pediatrics, University of Minnesota, Minneapolis, MN, USA.
Davis SeeligComparative Pathology Shared Resource, University of Minnesota, Minneapolis, MN, USA.
Carolyn A FairbanksDepartment of Pharmaceutics, University of Minnesota, Minneapolis MN, USA.
Nicholas BussREGENXBIO, Inc., Rockville, MD, USA.
Kwi Hye KimREGENXBIO, Inc., Rockville, MD, USA.
R Scott McIvorCenter for Genome Engineering, Department of Genetics, Cell Biology and Development, University of Minnesota, Minneapolis, MN, USA.

Funding

NINDS Center Core at the University of MinnesotaP30NS062158 · NINDS · UNIVERSITY OF MINNESOTA · PI EBNER, TIMOTHY · 2011 to 2015
$3.5M
NINDS NIH HHS P30 NS062158
6 · The paper itself

Abstract

Mucopolysaccharidosis type I (MPS I) is a rare metabolic disorder caused by deficiency of α-L-iduronidase (IDUA), resulting in glycosaminoglycan (GAG) accumulation and multisystemic disease. Current treatments include hematopoietic stem cell transplantation and enzyme replacement therapy, but these do not address all manifestations of the disease. We infused MPS I mice with an adeno-associated virus 9 (AAV9)-IDUA vector (RGX-111) at doses from 10

Indexed as

AAV9cardiacdose responsegene therapyintrathecalintravenousMPS Ineurocognitiveskeletal

Identifiers

PMID39687731
PMCPMC11646787

What OpenQuestion holds

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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.