Evidence map›Paper›PMID 34970092›Full record

ArticleThe Yale journal of biology and medicine2021

CRISPR-Mediated Genomic Addition to CPS1 Deficient iPSCs is Insufficient to Restore Nitrogen Homeostasis.

Matthew Nitzahn, Brian Truong, Suhail Khoja, Agustin Vega-Crespo, Colleen Le, Adam Eliav, Georgios Makris, April D Pyle, Johannes Häberle, Gerald S Lipshutz

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

Article in The Yale journal of biology and medicine, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 8 papers.

0numbers the graph read from it
0cells of the map it votes in
8citing papers in PubMed
0.4field-weighted citation impact, top 39% 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

8 citing papers in PubMed, 7 citations in OpenAlex.

  1. Review
  2. The Role of Urea Cycle Functional Studies in Preclinical Research.Journal of inherited metabolic disease · 2026
    Review
  3. Review
  4. Article
  5. Exploring RNA therapeutics for urea cycle disorders.Journal of inherited metabolic disease · 2024
    Review
  6. Review
  7. Review
  8. Modelling urea cycle disorders using iPSCs.NPJ Regenerative medicine · 2022
    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

10 authors at 2 institutions in 2 countries.

Matthew NitzahnMolecular Biology Institute, David Geffen School of Medicine at UCLA, Los Angeles, CA, USA.
Brian TruongDepartment of Surgery, David Geffen School of Medicine at UCLA, Los Angeles, CA, USA.
Suhail KhojaDepartment of Surgery, David Geffen School of Medicine at UCLA, Los Angeles, CA, USA.
Agustin Vega-CrespoDepartment of Molecular and Medical Pharmacology, David Geffen School of Medicine at UCLA, Los Angeles, CA, USA.
Colleen LeDepartment of Surgery, David Geffen School of Medicine at UCLA, Los Angeles, CA, USA.
Adam EliavDepartment of Surgery, David Geffen School of Medicine at UCLA, Los Angeles, CA, USA.
Georgios MakrisDivision of Metabolism and Children's Research Center, University Children's Hospital Zurich, Switzerland.
April D PyleDepartment of Microbiology, Immunology, and Molecular Genetics, David Geffen School of Medicine at UCLA, Los Angeles, CA, USA.
Johannes HäberleDivision of Metabolism and Children's Research Center, University Children's Hospital Zurich, Switzerland.
Gerald S LipshutzMolecular Biology Institute, David Geffen School of Medicine at UCLA, Los Angeles, CA, USA.
University of California, Los Angeles · USUniversity Children's Hospital Zurich · CH

Funding

UCLA IDDRC: Translational CoreP50HD103557 · NICHD · UNIVERSITY OF CALIFORNIA LOS ANGELES · PI SUSAN Y BOOKHEIMER · 2020 to 2026
$9.6M
Reprogramming and Directed Differentiation of Skeletal Muscle Cells from hPSCs.R01AR064327 · NIAMS · UNIVERSITY OF CALIFORNIA LOS ANGELES · PI April D Pyle · 2013 to 2026
$6.4M
Cell and Gene Replacement Strategies for Arginase DeficiencyR01NS100979 · NINDS · UNIVERSITY OF CALIFORNIA LOS ANGELES · PI LIPSHUTZ, GERALD S · 2017 to 2021
$1.7M
Development of Molecular Therapy for Carbamoyl Phosphate Synthetase DeficiencyR21NS091654 · NINDS · UNIVERSITY OF CALIFORNIA LOS ANGELES · PI LIPSHUTZ, GERALD S · 2015 to 2016
$424k
NIAMS NIH HHS R01 AR064327NICHD NIH HHS P50 HD103557NINDS NIH HHS R01 NS100979NINDS NIH HHS R21 NS091654
6 · The paper itself

Abstract

CPS1 deficiency is an inborn error of metabolism caused by loss-of-function mutations in the

Indexed as

Induced Pluripotent Stem CellsCarbamoyl-Phosphate Synthase (Ammonia)Clustered Regularly Interspaced Short Palindromic RepeatsGenomicsHomeostasisHumansNitrogenCarbamoyl-Phosphate Synthase (Ammonia)NitrogenCarbamoyl phosphate synthetase deficiencyCRISPR/Cas9gene therapyhyperammonemiaiPSC

Identifiers

PMID34970092
PMCPMC8686786
OpenAlexW4206867062

What OpenQuestion holds

Textmetadata
LicenceCC BY-NC
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.