Evidence map›Paper›PMID 38795068›Full record

ArticleNucleic acids research2024

AlPaCas: allele-specific CRISPR gene editing through a protospacer-adjacent-motif (PAM) approach.

Serena Rosignoli, Elisa Lustrino, Alessio Conci, Alessandra Fabrizi, Serena Rinaldo, Maria Carmela Latella, Elena Enzo, Gianni Prosseda, Laura De Rosa, Michele De Luca and 1 more

Erratum issuedAbstract read
In one paragraph

Article in Nucleic acids research, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. An erratum has been issued. Cited by 5 papers.

0numbers the graph read from it
0cells of the map it votes in
5citing 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

5 citing papers in PubMed.

  1. Review
  2. Review
  3. Article
  4. TOP-SECRETS enables Cas9 nucleases to discriminate SNVs outside of PAMs.bioRxiv : the preprint server for biology · 2025
    Article
  5. Article
4 · The record

Corrections and comments

5 · Who and what money

Authors and funding

11 authors.

Serena RosignoliDepartment of Biochemical Sciences "A. Rossi Fanelli", Sapienza University of Rome, Rome 00185, Italy.ORCID 0000-0002-0164-8998
Elisa LustrinoDepartment of Biochemical Sciences "A. Rossi Fanelli", Sapienza University of Rome, Rome 00185, Italy.
Alessio ConciCentre for Regenerative Medicine "Stefano Ferrari", Department of Life Sciences, University of Modena and Reggio Emilia, 41125 Modena, Italy.
Alessandra FabriziCentre for Regenerative Medicine "Stefano Ferrari", Department of Life Sciences, University of Modena and Reggio Emilia, 41125 Modena, Italy.
Serena RinaldoDepartment of Biochemical Sciences "A. Rossi Fanelli", Sapienza University of Rome, Rome 00185, Italy.ORCID 0000-0003-0682-023X
Maria Carmela LatellaHolostem s.r.l, Via Gottardi 100, 41125 Modena, Italy.
Elena EnzoCentre for Regenerative Medicine "Stefano Ferrari", Department of Life Sciences, University of Modena and Reggio Emilia, 41125 Modena, Italy.
Gianni ProssedaDepartment of Biology and Biotechnology Charles Darwin, Sapienza University of Rome, Rome 00185, Italy.ORCID 0000-0001-5235-0086
Laura De RosaCentre for Regenerative Medicine "Stefano Ferrari", Department of Life Sciences, University of Modena and Reggio Emilia, 41125 Modena, Italy.
Michele De LucaCentre for Regenerative Medicine "Stefano Ferrari", Department of Life Sciences, University of Modena and Reggio Emilia, 41125 Modena, Italy.
Alessandro PaiardiniDepartment of Biochemical Sciences "A. Rossi Fanelli", Sapienza University of Rome, Rome 00185, Italy.ORCID 0000-0001-9078-7545

Funding

Associazione Italiana Ricerca sul Cancro 20447European Research Council 101019289Italy Ministry of University and Research PRIN 2022N3JXLASapienza University of Rome RM1221815D52AB32
6 · The paper itself

Abstract

Gene therapy of dominantly inherited genetic diseases requires either the selective disruption of the mutant allele or the editing of the specific mutation. The CRISPR-Cas system holds great potential for the genetic correction of single nucleotide variants (SNVs), including dominant mutations. However, distinguishing between single-nucleotide variations in a pathogenic genomic context remains challenging. The presence of a PAM in the disease-causing allele can guide its precise targeting, preserving the functionality of the wild-type allele. The AlPaCas (Aligning Patients to Cas) webserver is an automated pipeline for sequence-based identification and structural analysis of SNV-derived PAMs that satisfy this demand. When provided with a gene/SNV input, AlPaCas can: (i) identify SNV-derived PAMs; (ii) provide a list of available Cas enzymes recognizing the SNV (s); (iii) propose mutational Cas-engineering to enhance the selectivity towards the SNV-derived PAM. With its ability to identify allele-specific genetic variants that can be targeted using already available or engineered Cas enzymes, AlPaCas is at the forefront of advancements in genome editing. AlPaCas is open to all users without a login requirement and is freely available at https://schubert.bio.uniroma1.it/alpacas.

Indexed as

AllelesCRISPR-Cas SystemsGene EditingCamelids, New WorldHumansInternetMutationNucleotide MotifsPolymorphism, Single NucleotideSoftware

Identifiers

PMID38795068
PMCPMC11223865

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.