ReviewGene therapy2003
Gene therapy progress and prospects: gene therapy of lysosomal storage disorders.
Review in Gene therapy, 2003. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 28 papers.
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.
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
28 citing papers in PubMed, 116 citations in OpenAlex.
- Advances in AAV-mediated gene replacement therapy for pediatric monogenic neurological disorders.Molecular therapy. Methods & clinical development · 2024Review
- Review
- Polymer-based drug delivery systems under investigation for enzyme replacement and other therapies of lysosomal storage disorders.Advanced drug delivery reviews · 2023Review
- IgG Fusion Proteins for Brain Delivery of Biologics via Blood-Brain Barrier Receptor-Mediated Transport.Pharmaceutics · 2022Review
- Gene Therapy for Lysosomal Storage Disorders: Ongoing Studies and Clinical Development.Biomolecules · 2021Review
- Modeling Mucopolysaccharidosis Type II in the Fruit Fly by Using the RNA Interference Approach.Life (Basel, Switzerland) · 2020Article
- The Configuration of the Perivascular System Transporting Macromolecules in the CNS.Frontiers in neuroscience · 2019Article
- Gene therapy for the neurological manifestations in lysosomal storage disorders.Journal of lipid research · 2014Review
- SERCA2a gene therapy in heart failure: an anti-arrhythmic positive inotrope.British journal of pharmacology · 2014Review
- Physiology of the intrathecal bolus: the leptomeningeal route for macromolecule and particle delivery to CNS.Molecular pharmaceutics · 2013Review
- Current and emerging treatments and surgical interventions for Morquio A syndrome: a review.Research and reports in endocrine disorders · 2012Article
- Secondary alterations of sphingolipid metabolism in lysosomal storage diseases.Neurochemical research · 2011Review
- Lessons learnt from animal models: pathophysiology of neuropathic lysosomal storage disorders.Journal of inherited metabolic disease · 2010Article
- Deregulated sphingolipid metabolism and membrane organization in neurodegenerative disorders.Molecular neurobiology · 2010Review
- AGT-181: expression in CHO cells and pharmacokinetics, safety, and plasma iduronidase enzyme activity in Rhesus monkeys.Journal of biotechnology · 2009Article
- Effect of elongation factor 1alpha promoter and SUMF1 over in vitro expression of N-acetylgalactosamine-6-sulfate sulfatase.Molecular biology reports · 2009Article
- Widespread biochemical correction of murine mucopolysaccharidosis type VII pathology by liver hydrodynamic plasmid delivery.Gene therapy · 2009Article
- Medical bioremediation of age-related diseases.Microbial cell factories · 2009Article
- Gene therapy for lysosomal storage diseases (LSDs) in large animal models.ILAR journal · 2009Review
- In vitro correction of ARSA deficiency in human skin fibroblasts from metachromatic leukodystrophy patients after treatment with microencapsulated recombinant cells.Metabolic brain disease · 2008Article
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.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Despite disappointments with early clinical studies, there is continued interest in the development of gene therapy for the group of metabolic diseases referred to as lysosomal storage disorders (LSDs). The LSDs are monogenic and several small and large, representative animal models of the human diseases are available. Further, the successful reconstitution of only low and unregulated tissue levels of the affected lysosomal enzymes are expected to be sufficient to correct the disease at least in the case of some of the LSDs. For these reasons, they are perceived as good models for the evaluation of different gene delivery vectors and of different strategies for treating chronic genetic diseases by gene transfer. In this review, we will highlight the progress that has been made over the past 2 years in preclinical research for this group of disorders and speculate on future prospects.
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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.