ReviewExpert opinion on biological therapy2007
Gene therapy for mucopolysaccharidosis.
Review in Expert opinion on biological therapy, 2007. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 43 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
43 citing papers in PubMed, 92 citations in OpenAlex.
- Functional gene delivery to and across brain vasculature of systemic AAVs with endothelial-specific tropism in rodents and broad tropism in primates.Nature communications · 2023Article
- Functional gene delivery to and across brain vasculature of systemic AAVs with endothelial-specific tropism in rodents and broad tropism in primates.bioRxiv : the preprint server for biology · 2023Article
- Mucopolysaccharidoses type I gene therapy.Journal of inherited metabolic disease · 2021Review
- Research and Application of Chondroitin Sulfate/Dermatan Sulfate-Degrading Enzymes.Frontiers in cell and developmental biology · 2020Review
- Article
- Prolonged Expression of Secreted Enzymes in Dogs After Liver-Directed Delivery of Sleeping Beauty Transposons: Implications for Non-Viral Gene Therapy of Systemic Disease.Human gene therapy · 2017Article
- Open issues in Mucopolysaccharidosis type I-Hurler.Orphanet journal of rare diseases · 2017Review
- A quarter century of progress in paediatric clinical pharmacology: A personal view.British journal of clinical pharmacology · 2016Article
- Evaluation of AAV-mediated Gene Therapy for Central Nervous System Disease in Canine Mucopolysaccharidosis VII.Molecular therapy : the journal of the American Society of Gene Therapy · 2016Article
- Genome-wide RNAi screening identifies host restriction factors critical for in vivo AAV transduction.Proceedings of the National Academy of Sciences of the United States of America · 2015Article
- Lysosomal storage disease: gene therapy on both sides of the blood-brain barrier.Molecular genetics and metabolism · 2015Review
- The effect of Tlr4 and/or C3 deficiency and of neonatal gene therapy on skeletal disease in mucopolysaccharidosis VII mice.Molecular genetics and metabolism · 2015Article
- Therapies for the bone in mucopolysaccharidoses.Molecular genetics and metabolism · 2015Review
- Gene therapy for neurologic manifestations of mucopolysaccharidoses.Expert opinion on drug delivery · 2015Review
- The Murine Model of Mucopolysaccharidosis IIIB Develops Cardiopathies over Time Leading to Heart Failure.PloS one · 2015Article
- A novel role for 12/15-lipoxygenase in regulating autophagy.Redox biology · 2015Article
- The phytoestrogen genistein modulates lysosomal metabolism and transcription factor EB (TFEB) activation.The Journal of biological chemistry · 2014Article
- Pathogenesis of mitral valve disease in mucopolysaccharidosis VII dogs.Molecular genetics and metabolism · 2013Article
- The effect of neonatal gene therapy with a gamma retroviral vector on cardiac valve disease in mucopolysaccharidosis VII dogs after a decade.Molecular genetics and metabolism · 2013Article
- Therapies of mucopolysaccharidosis IVA (Morquio A syndrome).Expert opinion on orphan drugs · 2013Article
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 2 institutions in 1 country.
Funding
Abstract
Mucopolysaccharidoses (MPS) are due to deficiencies in activities of lysosomal enzymes that degrade glycosaminoglycans. Some attempts at gene therapy for MPS in animal models have involved intravenous injection of vectors derived from an adeno-associated virus (AAV), adenovirus, retrovirus or a plasmid, which primarily results in expression in liver and secretion of the relevant enzyme into blood. Most vectors can correct disease in liver and spleen, although correction in other organs including the brain requires high enzyme activity in the blood. Alternative approaches are to transduce hematopoietic stem cells, or to inject a vector locally into difficult-to-reach sites such as the brain. Gene therapy holds great promise for providing a long-lasting therapeutic effect for MPS if safety issues can be resolved.
Indexed as
Identifiers
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.