Evidence map›Paper›PMID 35694211›Full record

ArticleMolecular therapy. Nucleic acids2022

Intermittent lipid nanoparticle mRNA administration prevents cortical dysmyelination associated with arginase deficiency.

Suhail Khoja, Xiao-Bo Liu, Brian Truong, Matthew Nitzahn, Jenna Lambert, Adam Eliav, Eram Nasser, Emma Randolph, Kristine E Burke, Rebecca White and 5 more

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

Article in Molecular therapy. Nucleic acids, 2022. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 12 papers.

0numbers the graph read from it
0cells of the map it votes in
12citing papers in PubMed
3.0field-weighted citation impact, top 8% 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

12 citing papers in PubMed, 23 citations in OpenAlex.

  1. Article
  2. RNA-Based Therapies for Inherited Metabolic Disorders.Journal of inherited metabolic disease · 2026
    Review
  3. Review
  4. Review
  5. Review
  6. Review
  7. Article
  8. Exploring RNA therapeutics for urea cycle disorders.Journal of inherited metabolic disease · 2024
    Review
  9. Review
  10. Article
  11. Article
  12. The role and control of arginine levels in arginase 1 deficiency.Journal of inherited metabolic disease · 2023
    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

15 authors at 3 institutions in 1 country.

Suhail KhojaDepartment of Surgery, David Geffen School of Medicine at UCLA, Los Angeles, CA 90095, USA.
Xiao-Bo LiuDepartment of Surgery, David Geffen School of Medicine at UCLA, Los Angeles, CA 90095, USA.
Brian TruongDepartment of Surgery, David Geffen School of Medicine at UCLA, Los Angeles, CA 90095, USA.
Matthew NitzahnMolecular Biology Institute, David Geffen School of Medicine at UCLA, Los Angeles, CA 90095, USA.
Jenna LambertDepartment of Surgery, David Geffen School of Medicine at UCLA, Los Angeles, CA 90095, USA.
Adam EliavDepartment of Surgery, David Geffen School of Medicine at UCLA, Los Angeles, CA 90095, USA.
Eram NasserDepartment of Surgery, David Geffen School of Medicine at UCLA, Los Angeles, CA 90095, USA.
Emma RandolphDepartment of Surgery, David Geffen School of Medicine at UCLA, Los Angeles, CA 90095, USA.
Kristine E BurkeModerna Inc., 200 Technology Square, Cambridge, MA 02139, USA.
Rebecca WhiteModerna Inc., 200 Technology Square, Cambridge, MA 02139, USA.
Xuling ZhuModerna Inc., 200 Technology Square, Cambridge, MA 02139, USA.
Paolo G V MartiniModerna Inc., 200 Technology Square, Cambridge, MA 02139, USA.
Itzhak NissimDivision of Metabolism and Human Genetics, The Children Hospital of Philadelphia and The Department of Biochemistry and Biophysics, Perlman School of Medicine, Philadelphia, PA 19104, USA.
Stephen D CederbaumDepartment of Psychiatry, David Geffen School of Medicine at UCLA, Los Angeles, CA 90095, USA.
Gerald S LipshutzDepartment of Surgery, David Geffen School of Medicine at UCLA, Los Angeles, CA 90095, USA.
University of California, Los Angeles · USModerna Therapeutics (United States) · USChildren's Hospital of Philadelphia · US

Funding

UCLA IDDRC: Translational CoreP50HD103557 · NICHD · UNIVERSITY OF CALIFORNIA LOS ANGELES · PI SUSAN Y BOOKHEIMER · 2020 to 2026
$9.6M
Understanding the Mechanism and Preventing the Unique Neuropathology of Arginase DeficiencyR01NS110596 · NINDS · UNIVERSITY OF CALIFORNIA LOS ANGELES · PI LIPSHUTZ, GERALD S · 2019 to 2023
$1.7M
Cell and Gene Replacement Strategies for Arginase DeficiencyR01NS100979 · NINDS · UNIVERSITY OF CALIFORNIA LOS ANGELES · PI LIPSHUTZ, GERALD S · 2017 to 2021
$1.7M
HIGH-END CRYOEM INSTRUMENT FOR UCLA ELECTRON IMAGING CTR FOR NANOMACHINES: AIDSS10RR023057 · NCRR · UNIVERSITY OF CALIFORNIA LOS ANGELES · PI ZHOU, Z HONG · 2006 to 2006
$1.6M
Elucidation of the Role of the Arginase Isoforms in Normal Neuronal Development and FunctionR03NS114623 · NINDS · UNIVERSITY OF CALIFORNIA LOS ANGELES · PI LIPSHUTZ, GERALD S · 2020 to 2020
$156k
Investigation of Neuroanatomical Alterations in Arginase Deficiency After Neonatal Gene TherapyR03NS101682 · NINDS · UNIVERSITY OF CALIFORNIA LOS ANGELES · PI LIPSHUTZ, GERALD S · 2017 to 2018
$154k
NCRR NIH HHS S10 RR023057NINDS NIH HHS R01 NS100979NINDS NIH HHS R01 NS110596NINDS NIH HHS R03 NS101682NINDS NIH HHS R03 NS114623
6 · The paper itself

Abstract

Arginase deficiency is associated with prominent neuromotor features, including spastic diplegia, clonus, and hyperreflexia; intellectual disability and progressive neurological decline are other signs. In a constitutive murine model, we recently described leukodystrophy as a significant component of the central nervous system features of arginase deficiency. In the present studies, we sought to examine if the administration of a lipid nanoparticle carrying human

Indexed as

arginase deficiencydysmyelinationlipid nanoparticlemessenger RNAMT: Oligonucleotides: Therapies and Applicationsurea cycle

Identifiers

PMID35694211
PMCPMC9156989
OpenAlexW4224948686

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.