Evidence map›Paper›PMID 42771312›Full record

ArticleMethods in molecular biology (Clifton, N.J.)2027

Adeno-Associated Viral Vector (AAV)-Mediated In Vivo CRISPR-Cas9 Delivery.

Andrea Llanos-Ardaiz, Nerea Zabaleta, Laura Torella, Gloria Gonzalez-Aseguinolaza

Abstract read
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In one paragraph

Article in Methods in molecular biology (Clifton, N.J.), 2027. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.

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

0 citing papers in PubMed.

No citing paper in PubMed yet.

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

4 authors.

Andrea Llanos-ArdaizDNA & RNA Medicine Division, CIMA, University of Navarra, Pamplona, Navarra, Spain. andreallanos@unav.es.
Nerea ZabaletaDNA & RNA Medicine Division, CIMA, University of Navarra, Pamplona, Navarra, Spain.
Laura TorellaDNA & RNA Medicine Division, CIMA, University of Navarra, Pamplona, Navarra, Spain.
Gloria Gonzalez-AseguinolazaDNA & RNA Medicine Division, CIMA, University of Navarra, Pamplona, Navarra, Spain. ggasegui@unav.es.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

CRISPR-based gene editing is a growing therapeutic strategy for modifying or silencing disease-causing genes. This chapter focuses on the in vivo delivery of CRISPR systems using recombinant adeno-associated viral (rAAV) vectors. Specifically, we detail a methodology using a rAAV vector carrying Staphylococcus aureus Cas9 (SaCas9) to target the murine Hao1 gene in liver parenchymal cells as a curative treatment for primary hyperoxaluria type 1 (PH1). We provide comprehensive protocols for plasmid design and cloning, rAAV production, animal administration, and evaluation of editing efficacy.

Indexed as

CRISPR-Cas SystemsDependovirusGene EditingGenetic VectorsGene Transfer TechniquesAnimalsHumansLiverMicePlasmidsStaphylococcus aureusCRISPR-SaCas9Gene editingIn vivoLiverMouserAAV

Identifiers

PMID42771312

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.