Evidence map›Paper›PMID 34159305›Full record

ReviewJHEP reports : innovation in hepatology2021

Novel vectors and approaches for gene therapy in liver diseases.

Sheila Maestro, Nicholas D Weber, Nerea Zabaleta, Rafael Aldabe, Gloria Gonzalez-Aseguinolaza

Open access · goldAbstract readReview
PubMed Full text at PMC Publisher
In one paragraph

Review in JHEP reports : innovation in hepatology, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 82 papers, 1 of them a synthesis that pooled it.

0numbers the graph read from it
0cells of the map it votes in
82citing papers in PubMed, 1 pooled it
18.9field-weighted citation impact, top 1% 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

82 citing papers in PubMed, 1 synthesis or guideline pooled it, 129 citations in OpenAlex.

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  19. Identification of a robust promoter in mouse and human hepatocytes by in vivo biopanning of a barcoded AAV library.Molecular therapy : the journal of the American Society of Gene Therapy · 2025
    Article
  20. iPSC-hepatocyte organoids as a novel platform to predict AAV gene therapy efficacy.Molecular therapy. Methods & clinical development · 2025
    Article

22 more citing papers are in PubMed but not listed here.

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

5 authors at 2 institutions in 2 countries.

Sheila MaestroGene Therapy Area, Foundation for Applied Medical Research, University of Navarra, IdisNA, Pamplona, Spain.
Nicholas D WeberVivet Therapeutics, Pamplona, Spain.
Nerea ZabaletaGrousbeck Gene Therapy Center, Schepens Eye Research Institute, Mass Eye and Ear, Boston, MA, USA.
Rafael AldabeGene Therapy Area, Foundation for Applied Medical Research, University of Navarra, IdisNA, Pamplona, Spain.
Gloria Gonzalez-AseguinolazaGene Therapy Area, Foundation for Applied Medical Research, University of Navarra, IdisNA, Pamplona, Spain.
Universidad de Navarra · ESSmith-Kettlewell Eye Research Institute · US

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Gene therapy is becoming an increasingly valuable tool to treat many genetic diseases with no or limited treatment options. This is the case for hundreds of monogenic metabolic disorders of hepatic origin, for which liver transplantation remains the only cure. Furthermore, the liver contains 10-15% of the body's total blood volume, making it ideal for use as a factory to secrete proteins into the circulation. In recent decades, an expanding toolbox has become available for liver-directed gene delivery. Although viral vectors have long been the preferred approach to target hepatocytes, an increasing number of non-viral vectors are emerging as highly efficient vehicles for the delivery of genetic material. Herein, we review advances in gene delivery vectors targeting the liver and more specifically hepatocytes, covering strategies based on gene addition and gene editing, as well as the exciting results obtained with the use of RNA as a therapeutic molecule. Moreover, we will briefly summarise some of the limitations of current liver-directed gene therapy approaches and potential ways of overcoming them.

Indexed as

AAT, α1-antitrypsinAAV, adeno-associated virusAd, adenovirusAHP, acute hepatic porphyriasAIP, acute intermittent porphyriaALAS1, aminolevulic synthase 1APCs, antigen-presenting cellsapoB/E, apolipoprotein B/EASGCT, American Society of Gene and Cell TherapyASGPR, asialoglycoprotein receptorASOs, antisense oligonucleotidesCBS, cystathionine β-synthaseCN, Crigel-NajjarCRISPR/Cas9, CRISPR associated protein 9CRISPR, clustered regularly interspaced short palindromic repeatsdCas9, dead Cas9DSBs, double-strand breaksefficacyERT, enzyme replacement therapyFH, familial hypercholesterolemiaFSP27, fat-specific protein 27GalNAc, N-acetyl-D-galactosaminegene additiongene editinggene silencingGO, glycolate oxidaseGSD1a, glycogen storage disorder 1aGT, gene therapyGUSB, β-glucuronidaseHDAd, helper-dependent adenovirusHDR, homology-directed repairHemA/B, haemophilia A/BhepatocytesHT, hereditary tyrosinemiaIDS, iduronate 2-sulfataseIDUA, α-L-iduronidaseIMLD, inherited metabolic liver diseasesimmune responseITR, inverted terminal repetitionLDH, lactate dehydrogenaseLDLR, low-density lipoprotein receptorlncRNA, long non-coding RNALNP, Lipid nanoparticlesLTR, long terminal repeatLV, lentivirusmiRNAs, microRNAsMMA, methylmalonic acidemiaMPR, metabolic pathway reprogramingMPS, mucopolysaccharidosisMPS type I, MPSIMPS type VII, MPSVIINASH, non-alcoholic steatohepatitisNHEJ, non-homologous end joiningNHPs, non-human primatesNon-viral vectorsOLT, orthotopic liver transplantationOTC, ornithine transcarbamylasePA, propionic acidemiaPB, piggyBacPCSK9, proprotein convertase subtilisin/kexin type 9PEG, polyethylene glycolPEI, polyethyleneiminePFIC3, progressive familial cholestasis type 3PH1, Primary hyperoxaluria type 1PKU, phenylketonuriaRV, retrovirusSB, Sleeping BeautysiRNA, small-interfering RNAS/MAR, scaffold matrix attachment regionsSRT, substrate reduction therapySTK25, serine/threonine protein kinase 25TALEN, transcription activator-like effector nucleasestoxicityTTR, transthyretinUCD, urea cycle disordersviral vectorsVLDLR, very-low-density lipoprotein receptorWD, Wilson’s diseaseZFN, zinc finger nucleases

Identifiers

PMID34159305
PMCPMC8203845
OpenAlexW3157192672

What OpenQuestion holds

Textmetadata
LicenceCC BY-NC-ND
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.