Article in Science advances, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 2 papers.
0numbers the graph read from it
0cells of the map it votes in
2citing 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.
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
26 authors.
Judit Tomsen-MeleroCentro de Investigación Biomédica en Red de Bioingeniería, Biomateriales y Nanomedicina, Instituto de Salud Carlos III, Madrid, Spain.ORCID 0000-0002-2837-8107
Marc Moltó-AbadCentro de Investigación Biomédica en Red de Bioingeniería, Biomateriales y Nanomedicina, Instituto de Salud Carlos III, Madrid, Spain.ORCID 0000-0002-6364-4570
Josep Merlo-MasNanomol Technologies SL, Campus de la UAB, 08193 Bellaterra, Spain.ORCID 0000-0002-3698-6655
Zamira V Díaz-RiascosCentro de Investigación Biomédica en Red de Bioingeniería, Biomateriales y Nanomedicina, Instituto de Salud Carlos III, Madrid, Spain.ORCID 0000-0003-1595-618X
Edgar Cristóbal-LecinaCentro de Investigación Biomédica en Red de Bioingeniería, Biomateriales y Nanomedicina, Instituto de Salud Carlos III, Madrid, Spain.ORCID 0000-0003-3270-9340
Thomas Altendorfer-KroathJOANNEUM RESEARCH-Institute for Biomedical Research and Technologies (HEALTH), Neue Stiftingtalstraße 2, 8010 Graz, Austria.ORCID 0000-0001-9531-9505
Dganit DaninoCryo-EM Laboratory of Soft Matter, Faculty of Biotechnology and Food Engineering, Technion-Israel Institute of Technology, 32000 Haifa, Israel.
Inbal IonitaCryo-EM Laboratory of Soft Matter, Faculty of Biotechnology and Food Engineering, Technion-Israel Institute of Technology, 32000 Haifa, Israel.ORCID 0000-0001-7359-0142
Jan Skov PedersenDepartment of Chemistry and Interdisciplinary Nanoscience Center (iNANO), Aarhus University, DK-8000 Aarhus C, Denmark.ORCID 0000-0002-7768-0206
Lyndsey SnellingLabcorp Drug Development, Harrogate HG3 IPY, UK.
Hazel ClayLabcorp Drug Development, Harrogate HG3 IPY, UK.
Aida CarreñoCentro de Investigación Biomédica en Red de Bioingeniería, Biomateriales y Nanomedicina, Instituto de Salud Carlos III, Madrid, Spain.ORCID 0000-0001-7725-7515
José L CorcheroCentro de Investigación Biomédica en Red de Bioingeniería, Biomateriales y Nanomedicina, Instituto de Salud Carlos III, Madrid, Spain.ORCID 0000-0002-6109-144X
Daniel PulidoCentro de Investigación Biomédica en Red de Bioingeniería, Biomateriales y Nanomedicina, Instituto de Salud Carlos III, Madrid, Spain.ORCID 0000-0002-2841-194X
Josefina CasasInstitut de Química Avançada de Catalunya (IQAC-CSIC), c/ Jordi Girona 18-26, 08034 Barcelona, Spain.ORCID 0000-0002-7926-5209
Jaume VecianaCentro de Investigación Biomédica en Red de Bioingeniería, Biomateriales y Nanomedicina, Instituto de Salud Carlos III, Madrid, Spain.ORCID 0000-0003-1023-9923
Simó SchwartzCentro de Investigación Biomédica en Red de Bioingeniería, Biomateriales y Nanomedicina, Instituto de Salud Carlos III, Madrid, Spain.ORCID 0000-0001-8297-7971
Santi SalaNanomol Technologies SL, Campus de la UAB, 08193 Bellaterra, Spain.ORCID 0009-0008-4560-0882
Albert FontLeanbio SL, 08028 Barcelona, Spain.
Thomas BirngruberJOANNEUM RESEARCH-Institute for Biomedical Research and Technologies (HEALTH), Neue Stiftingtalstraße 2, 8010 Graz, Austria.ORCID 0000-0001-8827-4660
Miriam RoyoCentro de Investigación Biomédica en Red de Bioingeniería, Biomateriales y Nanomedicina, Instituto de Salud Carlos III, Madrid, Spain.ORCID 0000-0001-5292-0819
Alba CórdobaNanomol Technologies SL, Campus de la UAB, 08193 Bellaterra, Spain.ORCID 0000-0002-2726-5119
Nora VentosaCentro de Investigación Biomédica en Red de Bioingeniería, Biomateriales y Nanomedicina, Instituto de Salud Carlos III, Madrid, Spain.ORCID 0000-0002-8008-4974
Ibane AbasoloCentro de Investigación Biomédica en Red de Bioingeniería, Biomateriales y Nanomedicina, Instituto de Salud Carlos III, Madrid, Spain.ORCID 0000-0001-5970-6276
Elisabet González-MiraCentro de Investigación Biomédica en Red de Bioingeniería, Biomateriales y Nanomedicina, Instituto de Salud Carlos III, Madrid, Spain.ORCID 0000-0003-2502-225X
Funding
No grant is acknowledged in the PubMed record.
6 · The paper itself
Abstract
The central nervous system represents a major target tissue for therapeutic approach of numerous lysosomal storage disorders. Fabry disease arises from the lack or dysfunction of the lysosomal alpha-galactosidase A (GLA) enzyme, resulting in substrate accumulation and multisystemic clinical manifestations. Current enzyme replacement therapies (ERTs) face limited effectiveness due to poor enzyme biodistribution in target tissues and inability to reach the brain. We present an innovative drug delivery strategy centered on a peptide-targeted nanoliposomal formulation, designated as nanoGLA, engineered to selectively deliver a recombinant human GLA (rhGLA) to target tissues. In a Fabry mouse model, nanoGLA demonstrated improved efficacy, inducing a notable reduction in Gb3 deposits in contrast to non-nanoformulated GLA, even in the brain, highlighting the potential of the nanoGLA to address both systemic and cerebrovascular manifestations of Fabry disease. The EMA has granted the Orphan Drug Designation to this product, underscoring the potential clinical superiority of nanoGLA over authorized ERTs and encouraging to advance it toward clinical translation.
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.
Targeted nanoliposomes to improve enzyme replacement therapy of Fabry disease. · full record | OpenQuestion