Intracellular Delivery of β-Galactosidase Enzyme Using Arginase-Responsive Dextran Sulfate/Poly-l-arginine Capsule for Lysosomal Storage Disorder

ACS Omega
Meenakshi GuptaSri Sivakumar

Abstract

β-Galactosidase (β-gal) is one of the important lysosomal enzymes that is involved in the breakdown of glycosphingolipids (e.g., GM1 ganglioside), and its deficiency leads to GM1 Gangliosidosis, a lysosomal storage disorder (LSD). Intracellular delivery of β-gal is one of the preferable methods to treat this kind of LSDs. However, it cannot permeate the cell membrane due to its intricate macromolecular nature, low stability, and degradation by endogenous proteases. To this end, we report efficient intracellular delivery of β-gal via arginase-responsive dextran sulfate/poly-l-arginine polymer capsules (DS/PA capsules). The therapeutic activity of β-gal enzyme has been assessed in two gene-deficient diseased cell lines, SV (β-galactosidase gene-deficient mouse fibroblast) and R201C (deficient human β-galactosidase gene-introduced mouse fibroblast), and in wild-type mouse fibroblast immortalized cell lines. The activity of β-gal enzyme has been estimated within cells by using fluorescein isothiocyanate-cholera toxin B as a florescent probe that illustrates the level of GM1 ganglioside, the β-gal substrate. We found 1.8-, 3.4-, and 2.8-fold reduction in the substrate level in R201C, SV, and wild-type mouse fibroblast, respectively,...Continue Reading

References

Oct 25, 2001·Proceedings of the National Academy of Sciences of the United States of America·Y SugimotoK Ohno
Jun 14, 2003·Philosophical Transactions of the Royal Society of London. Series B, Biological Sciences·Frances M PlattTerry D Butters
Oct 7, 2003·Journal of Colloid and Interface Science·D L GreenM T Harris
Dec 17, 2003·Proceedings of the National Academy of Sciences of the United States of America·Junichiro MatsudaYoshiyuki Suzuki
Sep 25, 2004·Movement Disorders : Official Journal of the Movement Disorder Society·Uday MuthaneSrikanth Subbamma Govindappa
May 7, 2005·Langmuir : the ACS Journal of Surfaces and Colloids·Dmitry V VolodkinGleb B Sukhorukov
Jun 4, 2005·Biotechnology Progress·Alexander I PetrovGleb B Sukhorukov
Oct 20, 2005·Journal of Bioscience and Bioengineering·M OndaT Kunitake
Jun 10, 2006·Journal of Inherited Metabolic Disease·J E Wraith
Dec 13, 2006·FEMS Microbiology Letters·Daniel J-F ChinnapenWayne I Lencer
Dec 14, 2006·Molecular Therapy : the Journal of the American Society of Gene Therapy·M L D BroekmanM Sena-Esteves
Feb 28, 2007·Immunology and Cell Biology·Norbert BlankHanns-Martin Lorenz
Apr 5, 2008·Molecular Genetics and Metabolism·Elena Elliot-SmithFrances M Platt
Jun 24, 2008·European Journal of Paediatric Neurology : EJPN : Official Journal of the European Paediatric Neurology Society·Anna CaciottiAmelia Morrone
Dec 2, 2009·The Journal of Immunology : Official Journal of the American Association of Immunologists·Stefaan De KokerJohan Grooten
Feb 6, 2010·Journal of the American Chemical Society·Partha GhoshVincent M Rotello
Sep 10, 2010·Molecular Genetics and Metabolism·Linjing LiEiji Nanba
Mar 8, 2011·Biomacromolecules·Marie-Luce De TemmermanStefaan C De Smedt
Apr 23, 2011·Advanced Drug Delivery Reviews·Katsuhiko ArigaJonathan P Hill
Jun 21, 2013·The Journal of Neuroscience : the Official Journal of the Society for Neuroscience·Konrad Sandhoff, Klaus Harzer
Apr 18, 2014·Journal of Pharmaceutical Sciences·Lina Herrera EstradaJulie A Champion
May 13, 2014·Proceedings of the Japan Academy. Series B, Physical and Biological Sciences·Yoshiyuki Suzuki
Apr 9, 2015·The Journal of Pharmacy and Pharmacology·Yuki MaedaHidetoshi Arima
Jun 6, 2015·Annals of Translational Medicine·Carl HaywardAlexander R Lyon
Nov 21, 2015·Journal of the American Chemical Society·Jeffrey D BrodinChad A Mirkin
Feb 26, 2016·International Journal of Molecular Sciences·Ana DincaMichael T Chin

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Citations

Apr 17, 2021·The Application of Clinical Genetics·Allisandra K RhaDouglas R Martin

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Datasets Mentioned

BETA
GM1

Methods Mentioned

BETA
enzyme replacement therapy
dynamic light scattering
transmission
scanning electron microscopy
confocal microscopy

Software Mentioned

Image J

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