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Bioimpacts. 2018;8(3): 153-157. doi: 10.15171/bi.2018.17
PMID: 30211074        PMCID: PMC6128977

Editorial

Enzyme replacement therapies: what is the best option?

Azam Safary 1,2 ORCID, Mostafa Akbarzadeh Khiavi 3,2 ORCID, Rahimeh Mousavi 2, Jaleh Barar 2,4 ORCID, Mohammad A Rafi 5 *

Cited by CrossRef: 35

1- Sharifi M, Karim A, Mustafa Qadir Nanakali N, Salihi A, Aziz F, Hong J, Khan R, Saboury A, Hasan A, Abou-Zied O, Falahati M. Strategies of enzyme immobilization on nanomatrix supports and their intracellular delivery. Journal of Biomolecular Structure and Dynamics. 2020;38(9):2746 [Crossref]
2- Solano M, Fainboim A, Politei J, Porras-Hurtado G, Martins A, Souza C, Koch F, Amartino H, Satizábal J, Horovitz D, Medeiros P, Honjo R, Lourenço C. Enzyme replacement therapy interruption in patients with Mucopolysaccharidoses: Recommendations for distinct scenarios in Latin America. Molecular Genetics and Metabolism Reports. 2020;23:100572 [Crossref]
3- Safary A, Moniri R, Hamzeh-Mivehroud M, Dastmalchi S. Highly efficient novel recombinant L-asparaginase with no glutaminase activity from a new halo-thermotolerant Bacillus strain. Bioimpacts. 2018;9(1):15 [Crossref]
4- Akbarzadeh Khiavi M, Safary A, Aghanejad A, Barar J, Rasta S, Golchin A, Omidi Y, Somi M. Enzyme-conjugated gold nanoparticles for combined enzyme and photothermal therapy of colon cancer cells. Colloids and Surfaces A: Physicochemical and Engineering Aspects. 2019;572:333 [Crossref]
5- Lee H, McGinty G, Pearl P, Rotenberg A. Understanding the Molecular Mechanisms of Succinic Semialdehyde Dehydrogenase Deficiency (SSADHD): Towards the Development of SSADH-Targeted Medicine. IJMS. 2022;23(5):2606 [Crossref]
6- Del Grosso A, Carpi S, De Sarlo M, Scaccini L, Colagiorgio L, Alabed H, Angella L, Pellegrino R, Tonazzini I, Emiliani C, Cecchini M. Chronic Rapamycin administration via drinking water mitigates the pathological phenotype in a Krabbe disease mouse model through autophagy activation. Biomedicine & Pharmacotherapy. 2024;173:116351 [Crossref]
7- Akbarzadeh Khiavi M, Safary A, Barar J, Farzi-Khajeh H, Barzegari A, Mousavi R, Somi M, Omidi Y. PEGylated gold nanoparticles-ribonuclease induced oxidative stress and apoptosis in colorectal cancer cells. Bioimpacts. 2019;10(1):27 [Crossref]
8- Del Grosso A, Galliani M, Angella L, Santi M, Tonazzini I, Parlanti G, Signore G, Cecchini M. Brain-targeted enzyme-loaded nanoparticles: A breach through the blood-brain barrier for enzyme replacement therapy in Krabbe disease. Sci Adv. 2019;5(11) [Crossref]
9- Bouhamdani N, Comeau D, Turcotte S. A Compendium of Information on the Lysosome. Front Cell Dev Biol. 2021;9 [Crossref]
10- Cubero E, Ruano A, Delgado A, Barril X, Morales S, Trapero A, Leoni L, Bellotto M, Maj R, Guzmán B, Pérez-Carmona N, Garcia-Collazo A, Sahoo N. Discovery of allosteric regulators with clinical potential to stabilize alpha-L-iduronidase in mucopolysaccharidosis type I. PLoS ONE. 2024;19(5):e0303789 [Crossref]
11- Bonam S, Wang F, Muller S. Lysosomes as a therapeutic target. Nat Rev Drug Discov. 2019;18(12):923 [Crossref]
12- Safary A, Moghaddas-Sani H, Akbarzadeh-Khiavi M, Khabbazzi A, Rafi M, Omidi Y. Enzyme replacement combinational therapy: effective treatments for mucopolysaccharidoses. Expert Opinion on Biological Therapy. 2021;21(9):1181 [Crossref]
13- Fathi M, Safary A, Barar J. Therapeutic impacts of enzyme-responsive smart nanobiosystems. Bioimpacts. 2019;10(1):1 [Crossref]
14- Akbarzadeh Khiavi M, Safary A, Somi M. Recent advances in targeted therapy of colorectal cancer: impacts of monoclonal antibodies nanoconjugates. Bioimpacts. 2019;9(3):123 [Crossref]
15- Dumina M, Zhgun A, Pokrovskaya M, Aleksandrova S, Zhdanov D, Sokolov N, El’darov M. A Novel L-Asparaginase from Hyperthermophilic Archaeon Thermococcus sibiricus: Heterologous Expression and Characterization for Biotechnology Application. IJMS. 2021;22(18):9894 [Crossref]
16- Buchinskaya N, Kostik M, Kolobova O, Melnikova L. How Not to Miss the Mild Forms of Mucopolysaccharidosis Type I in Patients With Articular Manifestations of the Disease?. Vopr sovr pediatr. 2019;17(6):473 [Crossref]
17- Lu C, Kim J. Craniofacial Diseases Caused by Defects in Intracellular Trafficking. Genes. 2021;12(5):726 [Crossref]
18- Stiles A, Huggins E, Fierro L, Jung S, Balwani M, Kishnani P. The role of glucosylsphingosine as an early indicator of disease progression in early symptomatic type 1 Gaucher disease. Molecular Genetics and Metabolism Reports. 2021;27:100729 [Crossref]
19- Akhter S, Kaur H, Agrawal P, Raghava G. RareLSD: a manually curated database of lysosomal enzymes associated with rare diseases. 2019;2019 [Crossref]
20- Schaible P. Modifying enzyme replacement therapy – A perspective. J Cellular Molecular Medi. 2023;27(2):165 [Crossref]
21- Akbarzadeh Khiavi M, Safary A, Barar J, Ajoolabady A, Somi M, Omidi Y. Multifunctional nanomedicines for targeting epidermal growth factor receptor in colorectal cancer. Cell Mol Life Sci. 2020;77(6):997 [Crossref]
22- Safary A, Akbarzadeh Khiavi M, Omidi Y, Rafi M. Targeted enzyme delivery systems in lysosomal disorders: an innovative form of therapy for mucopolysaccharidosis. Cell Mol Life Sci. 2019;76(17):3363 [Crossref]
23- Carvill G, Matheny T, Hesselberth J, Demarest S. Haploinsufficiency, Dominant Negative, and Gain-of-Function Mechanisms in Epilepsy: Matching Therapeutic Approach to the Pathophysiology. Neurotherapeutics. 2021;18(3):1500 [Crossref]
24- Ghosh S, Brown A, Jenkins C, Campbell K. Viral Vector Systems for Gene Therapy: A Comprehensive Literature Review of Progress and Biosafety Challenges. Applied Biosafety. 2020;25(1):7 [Crossref]
25- Duskey J, da Ros F, Ottonelli I, Zambelli B, Vandelli M, Tosi G, Ruozi B. Enzyme Stability in Nanoparticle Preparations Part 1: Bovine Serum Albumin Improves Enzyme Function. Molecules. 2020;25(20):4593 [Crossref]
26- Mohapatra P, Gopikrishnan M, Doss C G, Chandrasekaran N. How Precise are Nanomedicines in Overcoming the Blood–Brain Barrier? A Comprehensive Review of the Literature. IJN. 2024;Volume 19:2441 [Crossref]
27- Abasolo I, Seras‐Franzoso J, Moltó‐Abad M, Díaz‐Riascos V, Corchero J, Pintos‐Morell G, Schwartz S. Nanotechnology‐based approaches for treating lysosomal storage disorders, a focus on Fabry disease. WIREs Nanomed Nanobiotechnol. 2021;13(3) [Crossref]
28- Seras‐Franzoso J, Díaz‐Riascos Z, Corchero J, González P, García‐Aranda N, Mandaña M, Riera R, Boullosa A, Mancilla S, Grayston A, Moltó‐Abad M, Garcia‐Fruitós E, Mendoza R, Pintos‐Morell G, Albertazzi L, Rosell A, Casas J, Villaverde A, Schwartz S, Abasolo I. Extracellular vesicles from recombinant cell factories improve the activity and efficacy of enzymes defective in lysosomal storage disorders. J of Extracellular Vesicle. 2021;10(5) [Crossref]
29- Riley R, Kashyap M, Billingsley M, White B, Alameh M, Bose S, Zoltick P, Li H, Zhang R, Cheng A, Weissman D, Peranteau W, Mitchell M. Ionizable lipid nanoparticles for in utero mRNA delivery. Sci Adv. 2021;7(3) [Crossref]
30- Dastmalchi M, Alizadeh M, Jamshidi-Kandjan O, Rezazadeh H, Hamzeh-Mivehroud M, Farajollahi M, Dastmalchi S. Expression and Biological Evaluation of an Engineered Recombinant L-asparaginase Designed by In Silico Method Based on Sequence of the Enzyme from Escherichia coli. Adv Pharm Bull. 2023;13(4):827 [Crossref]
31- Fraile-Gutiérrez I, Iglesias S, Acosta N, Revuelta J. Chitosan-based oral hydrogel formulations of β-galactosidase to improve enzyme supplementation therapy for lactose intolerance. International Journal of Biological Macromolecules. 2024;255:127755 [Crossref]
32- Chaudhary R, Rehman M, Agarwal V, Kumar A, Kaushik A, Srivastava S, Srivastava S, Verma R, Rajinikanth P, Mishra V. Terra incognita of glial cell dynamics in the etiology of leukodystrophies: Broadening disease and therapeutic perspectives. Life Sciences. 2024;354:122953 [Crossref]
33- Uzhytchak M, Smolková B, Lunova M, Frtús A, Jirsa M, Dejneka A, Lunov O. Lysosomal nanotoxicity: Impact of nanomedicines on lysosomal function. Advanced Drug Delivery Reviews. 2023;197:114828 [Crossref]

As a peer-reviewed international open-access journal, BioImpacts publishes articles on basic and translational aspects of pharmaceutical and biomedical sciences. 
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