Evidence map›Paper›PMID 40083646›Full record

ArticleMolecular therapy. Nucleic acids2025

Use of an oversized AAV8 vector for CPS1 deficiency results in long-term survival and ammonia control.

Taryn Diep, Wesley Zhou, Rachel E Reyes, Matthew Nitzahn, Isabel L Day, Georgios Makris, Lindsay Lueptow, Irina Zhuravka, Stuti Bakshi, Jon Gangoiti and 5 more

Abstract read
PubMed Full text at PMC Publisher
In one paragraph

Article in Molecular therapy. Nucleic acids, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 2 papers.

0numbers the graph read from it
0cells of the map it votes in
2citing 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

2 citing papers in PubMed.

  1. Article
  2. An oversized AAV8 vector to deliver CPS1.Molecular therapy. Nucleic acids · 2025
    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

15 authors.

Taryn DiepDepartment of Surgery, David Geffen School of Medicine at UCLA, Los Angeles, CA, USA.
Wesley ZhouDepartment of Surgery, David Geffen School of Medicine at UCLA, Los Angeles, CA, USA.
Rachel E ReyesDepartment of Surgery, David Geffen School of Medicine at UCLA, Los Angeles, CA, USA.
Matthew NitzahnMolecular Biology Institute, David Geffen School of Medicine at UCLA, Los Angeles, CA, USA.
Isabel L DayDepartment 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, Zurich, Switzerland.
Lindsay LueptowDepartment of Psychology, David Geffen School of Medicine at UCLA, Los Angeles, CA, USA.
Irina ZhuravkaDepartment of Psychology, David Geffen School of Medicine at UCLA, Los Angeles, CA, USA.
Stuti BakshiDepartment of Surgery, David Geffen School of Medicine at UCLA, Los Angeles, CA, USA.
Jon GangoitiDepartment of Pediatrics, Division of Biochemical Genetics, University of California, San Diego, San Diego, CA, USA.
Hyacinth PadaonDepartment of Pediatrics, Division of Biochemical Genetics, University of California, San Diego, San Diego, CA, USA.
Yunfeng LiDepartment of Pathology and Laboratory Medicine, David Geffen School of Medicine at UCLA, Los Angeles, CA, USA.
Bruce A BarshopDepartment of Pediatrics, Division of Biochemical Genetics, University of California, San Diego, San Diego, CA, USA.
Johannes HaberleDivision of Metabolism and Children's Research Center, University Children's Hospital Zurich, Zurich, Switzerland.
Gerald S LipshutzDepartment of Surgery, David Geffen School of Medicine at UCLA, Los Angeles, CA, USA.

Funding

UCLA IDDRC: Translational CoreP50HD103557 · NICHD · UNIVERSITY OF CALIFORNIA LOS ANGELES · PI Peyman Golshani, SUMA JACOB · 2020 to 2026
$9.6M
Gene Therapy Clinical Candidate Development for Carbamoyl Phosphate Synthetase DeficiencyR61NS121348 · NINDS · UNIVERSITY OF CALIFORNIA LOS ANGELES · PI LIPSHUTZ, GERALD S · 2022 to 2023
$818k
NICHD NIH HHS P50 HD103557NINDS NIH HHS R61 NS121348
6 · The paper itself

Abstract

Carbamoyl phosphate synthetase 1 (CPS1) deficiency, a urea-cycle disorder, results in hyperammonemia initiating a sequence of adverse events that can lead to coma and death if not treated rapidly. There is a high unmet need for an effective therapeutic for this disorder, especially in early neonatal patients where mortality is excessive. However, development of an adeno-associated virus (AAV)-based approach is hampered by large cDNA size and high protein requirement. We developed an oversized AAV vector as a gene therapy to treat

Indexed as

carbamoyl phosphate synthetase deficiencygene therapyhyperammonemiaMT: Delivery Strategiesoversized AAVurea-cycle disorderureagenesis

Identifiers

PMID40083646
PMCPMC11905892

What Socratic holds

Textmetadata
LicenceCC BY
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 Socratic graph.