ArticleLipids1996
Enzyme replacement and gene therapy for Gaucher's disease.
Article in Lipids, 1996. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 4 papers.
What it found
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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.
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.
Who cites it
4 citing papers in PubMed, 14 citations in OpenAlex.
- Bone marrow involvement in Gaucher disease at MRI : what long-term evolution can we expect under enzyme replacement therapy?European radiology · 2015Article
- Generation of polyclonal antibodies against recombinant human glucocerebrosidase produced in Escherichia coli.Molecular biotechnology · 2010Article
- Chemically modified beta-glucuronidase crosses blood-brain barrier and clears neuronal storage in murine mucopolysaccharidosis VII.Proceedings of the National Academy of Sciences of the United States of America · 2008Article
- Enzyme therapy in mannose receptor-null mucopolysaccharidosis VII mice defines roles for the mannose 6-phosphate and mannose receptors.Proceedings of the National Academy of Sciences of the United States of America · 2006Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
2 authors at 1 institution in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
The lipid storage disorders have long been considered primary candidates for enzyme replacement therapy. This goal has been achieved with a remarkable degree of success in Gaucher's disease. Among the accomplishments that were important to obtain clinical benefit were the development of a large-scale procedure to purify human placental glucocerebrosidase and a method to target this enzyme to lipid-storing macrophages through glycoform modification. In addition, the effectiveness of recombinantly produced macrophage-targeted glucocerebrosidase has recently been demonstrated. Because macrophages originate from stem cells in the bone marrow, ex vivo transduction of these cells with retroviral vectors containing the cDNA for human glucocerebrosidase is being explored for the genetic therapy of Gaucher's disease.
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What OpenQuestion holds
Registered trials
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.