In one paragraphArticle in International journal of molecular sciences, 2025. 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
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 registryThe 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 literatureWho cites it
0 citing papers in PubMed.
No citing paper in PubMed yet.
4 · The recordCorrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
5 · Who and what moneyAuthors and funding
14 authors.
Ana Carolina CoelhoDepartment of Genetics, Ribeirão Preto Medical School, University of São Paulo, Ribeirão Preto 14049-900, SP, Brazil.ORCID 0009-0005-0307-1503 Claudia Emília Vieira WiezelDepartment of Genetics, Ribeirão Preto Medical School, University of São Paulo, Ribeirão Preto 14049-900, SP, Brazil.ORCID 0000-0002-4381-0834 Alline Cristina de CamposDepartment of Pharmacology, Ribeirão Preto Medical School, University of São Paulo, Ribeirão Preto 14049-900, SP, Brazil.ORCID 0000-0003-4258-3198 Lílian Louise Souza FigueiredoDepartment of Genetics, Ribeirão Preto Medical School, University of São Paulo, Ribeirão Preto 14049-900, SP, Brazil.
Gabriela Aparecida Marcondes SuardiDepartment of Genetics, Ribeirão Preto Medical School, University of São Paulo, Ribeirão Preto 14049-900, SP, Brazil.ORCID 0000-0002-2735-2570 Juliana de Paula BernardesDepartment of Genetics, Ribeirão Preto Medical School, University of São Paulo, Ribeirão Preto 14049-900, SP, Brazil.ORCID 0009-0001-9542-5495 Daniela Pretti da Cunha TirapelliDepartment of Surgery and Anatomy, Ribeirão Preto Medical School, University of São Paulo, Ribeirão Preto 14049-900, SP, Brazil.
Vitor Marcel FaçaDepartment of Biochemistry and Immunology, Ribeirão Preto Medical School, University of São Paulo, Ribeirão Preto 14049-900, SP, Brazil.ORCID 0000-0003-3205-7944 Kuruvilla Joseph AbrahamDepartment of Computer Science, Institute of Mathematics and Computer Science, University of São Paulo, São Carlos 13566-590, SP, Brazil.ORCID 0000-0002-0520-4665 Carlos Gilberto Carlotti-JúniorDivision of Neurology, São Paulo Medical School, University of São Paulo, São Paulo 05403-000, SP, Brazil.
Velia SicilianoSynthetic and Systems Biology Laboratory for Biomedicine, Istituto Italiano di Tecnologia-IIT, Largo Barsanti e Matteucci, 80125 Naples, Italy.ORCID 0000-0001-7734-9153 Ron WeissSynthetic Biology Center, Department of Biological Engineering, Massachusetts Institute of Technology, Cambridge, MA 02129-4307, USA.
Stanton GersonSchool of Medicine, University Hospital Medical Center, Case Western Reserve University, Cleveland, OH 44106, USA.ORCID 0000-0003-3106-2549 Aparecida Maria FontesDepartment of Genetics, Ribeirão Preto Medical School, University of São Paulo, Ribeirão Preto 14049-900, SP, Brazil.ORCID 0000-0002-4878-417X Funding
Coordenação de Aperfeicoamento de Pessoal de Nível Superior 001Fundação de Amparo à Pesquisa do Estado de São Paulo 2013/50450-2Fundação de Amparo à Pesquisa do Estado de São Paulo 2013/50764-7Fundação de Amparo à Pesquisa do Estado de São Paulo 2014/15846-5Fundação de Apoio ao Ensino, Pesquisa e Assistência do Hospital das Clínicas da Faculdade de Medicina de Ribeirão Preto da Universidade de São Paulo 001National Council for Scientific and Technological Development 001
6 · The paper itselfAbstract
Gaucher disease (GD) is an autosomal recessive disorder caused by the deficient activity of the lysosomal enzyme glucocerebrosidase (GCase). Although enzyme replacement therapy (ERT) remains the standard of care for non-neuropathic GD patients, its high cost significantly limits accessibility. To enhance production efficiency, we developed a lentiviral system encoding a codon-optimized GCase gene driven by the human elongation factor 1a (hEF1α) promoter for stable production in human cell lines. A functional lentiviral vector, LV_EF1α_GBA_Opt, was generated at a titer of 7.88 × 10
Indexed as
Gaucher DiseaseGenetic VectorsGlucosylceramidaseLentivirusCell LineEnzyme Replacement TherapyHEK293 CellsHumansPeptide Elongation Factor 1Promoter Regions, GeneticRecombinant ProteinsGlucosylceramidasePeptide Elongation Factor 1Recombinant Proteins293FT cellscodon optimizationGaucher diseaseglucocerebrosidasehEF1α promoterlentiviral vector
Identifiers
PMID40806226
PMCPMC12346726
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