Evidence map›Paper›PMID 39408666›Full record

ArticleInternational journal of molecular sciences2024

A Novel Murine Model Enabling rAAV8-PC Gene Therapy for Severe Protein C Deficiency.

Sarina Levy-Mendelovich, Einat Avishai, Benjamin J Samelson-Jones, Rima Dardik, Tami Brutman-Barazani, Yael Nisgav, Tami Livnat, Gili Kenet

Abstract read
PubMed Full text at PMC Publisher
In one paragraph

Article in International journal of molecular sciences, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 3 papers.

0numbers the graph read from it
0cells of the map it votes in
3citing 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

3 citing papers in PubMed.

  1. Review
  2. Review
  3. Current clinical applications of AAV-mediated gene therapy.Molecular therapy : the journal of the American Society of Gene Therapy · 2025
    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

8 authors.

Sarina Levy-MendelovichNational Hemophilia Center, Thrombosis & Hemostasis Institute, Sheba Medical Center, Ramat Gan 52621, Israel.ORCID 0000-0003-3971-0336
Einat AvishaiNational Hemophilia Center, Thrombosis & Hemostasis Institute, Sheba Medical Center, Ramat Gan 52621, Israel.ORCID 0009-0002-6920-790X
Benjamin J Samelson-JonesDepartment of Pediatrics, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA 19104, USA.ORCID 0000-0001-6772-4140
Rima DardikNational Hemophilia Center, Thrombosis & Hemostasis Institute, Sheba Medical Center, Ramat Gan 52621, Israel.
Tami Brutman-BarazaniNational Hemophilia Center, Thrombosis & Hemostasis Institute, Sheba Medical Center, Ramat Gan 52621, Israel.ORCID 0009-0009-8651-2501
Yael NisgavNational Hemophilia Center, Thrombosis & Hemostasis Institute, Sheba Medical Center, Ramat Gan 52621, Israel.
Tami LivnatNational Hemophilia Center, Thrombosis & Hemostasis Institute, Sheba Medical Center, Ramat Gan 52621, Israel.ORCID 0000-0002-1279-2557
Gili KenetNational Hemophilia Center, Thrombosis & Hemostasis Institute, Sheba Medical Center, Ramat Gan 52621, Israel.ORCID 0000-0003-3494-9823

Funding

Translating Mechanistic Insights into Intrinsic Xase FunctionP01HL139420 · NHLBI · CHILDREN'S HOSP OF PHILADELPHIA · PI Sriram Krishnaswamy · 2018 to 2026
$22.4M
BSF 2021191NHLBI NIH HHS P01 HL139420
6 · The paper itself

Abstract

Severe protein C deficiency (SPCD) is a rare inherited thrombotic disease associated with high morbidity and mortality. In the current study, we established a viable murine model of SPCD, enabling preclinical gene therapy studies. By creating SPCD mice with severe hemophilia A (PROC

Indexed as

DependovirusDisease Models, AnimalGenetic TherapyGenetic VectorsProtein CProtein C DeficiencyAnimalsHemophilia ALiverMaleMiceMice, KnockoutProtein Cadeno-associated virusgene therapyprotein C deficiencythrombosis

Identifiers

PMID39408666
PMCPMC11477312

What OpenQuestion holds

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Registered trials

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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.