ArticleBioImpacts : BI2018
Enzyme replacement therapies: what is the best option?
Article in BioImpacts : BI, 2018. 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, 53 citations in OpenAlex.
- Advances in β-Galactosidase Research: A Systematic Review from Molecular Mechanisms to Enzyme Delivery Systems.Pharmaceutics · 2025Review
- Discovery of allosteric regulators with clinical potential to stabilize alpha-L-iduronidase in mucopolysaccharidosis type I.PloS one · 2024Article
- How Precise are Nanomedicines in Overcoming the Blood-Brain Barrier? A Comprehensive Review of the Literature.International journal of nanomedicine · 2024Review
- Expression and Biological Evaluation of an Engineered Recombinant L-asparaginase Designed byAdvanced pharmaceutical bulletin · 2023Article
- Modifying enzyme replacement therapy - A perspective.Journal of cellular and molecular medicine · 2023Review
- Understanding the Molecular Mechanisms of Succinic Semialdehyde Dehydrogenase Deficiency (SSADHD): Towards the Development of SSADH-Targeted Medicine.International journal of molecular sciences · 2022Review
- A kaleidoscopic view of extracellular vesicles in lysosomal storage disorders.Extracellular vesicles and circulating nucleic acids · 2022Review
- A Novel L-Asparaginase from Hyperthermophilic ArchaeonInternational journal of molecular sciences · 2021Article
- Haploinsufficiency, Dominant Negative, and Gain-of-Function Mechanisms in Epilepsy: Matching Therapeutic Approach to the Pathophysiology.Neurotherapeutics : the journal of the American Society for Experimental NeuroTherapeutics · 2021Review
- The role of glucosylsphingosine as an early indicator of disease progression in early symptomatic type 1 Gaucher disease.Molecular genetics and metabolism reports · 2021Article
- Review
- Extracellular vesicles from recombinant cell factories improve the activity and efficacy of enzymes defective in lysosomal storage disorders.Journal of extracellular vesicles · 2021Article
- A Compendium of Information on the Lysosome.Frontiers in cell and developmental biology · 2021Review
- Achievements and beyond: Scientific trajectory of Professor Mohammad A. Rafi.BioImpacts : BI · 2021Article
- Ionizable lipid nanoparticles for in utero mRNA delivery.Science advances · 2021Article
- Enzyme Stability in Nanoparticle Preparations Part 1: Bovine Serum Albumin Improves Enzyme Function.Molecules (Basel, Switzerland) · 2020Article
- Enzyme replacement therapy interruption in patients with Mucopolysaccharidoses: Recommendations for distinct scenarios in Latin America.Molecular genetics and metabolism reports · 2020Article
- Viral Vector Systems for Gene Therapy: A Comprehensive Literature Review of Progress and Biosafety Challenges.Applied biosafety : journal of the American Biological Safety Association · 2020Review
- Multifunctional nanomedicines for targeting epidermal growth factor receptor in colorectal cancer.Cellular and molecular life sciences : CMLS · 2020Review
- Therapeutic impacts of enzyme-responsive smart nanobiosystems.BioImpacts : BI · 2020Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
5 authors at 2 institutions in 2 countries.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Despite many beneficial outcomes of the conventional enzyme replacement therapy (ERT), several limitations such as the high-cost of the treatment and various inadvertent side effects including the occurrence of an immunological response against the infused enzyme and development of resistance to enzymes persist. These issues may limit the desired therapeutic outcomes of a majority of the lysosomal storage diseases (LSDs). Furthermore, the biodistribution of the recombinant enzymes into the target cells within the central nervous system (CNS), bone, cartilage, cornea, and heart still remain unresolved. All these shortcomings necessitate the development of more effective diagnosis and treatment modalities against LSDs. Taken all, maximizing the therapeutic response with minimal undesired side effects might be attainable by the development of targeted enzyme delivery systems (EDSs) as a promising alternative to the LSDs treatments, including different types of mucopolysaccharidoses ( MPSs ) as well as Fabry, Krabbe, Gaucher and Pompe diseases.
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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.