Citations (34)
Keep up to date with the latest research on this topic with citation updates for this article.
Read on this site (3)
Azam Safary, Hakimeh Moghaddas-Sani, Mostafa Akbarzadeh-Khiavi, Alireza Khabbazzi, Mohammad A. Rafi & Yadollah Omidi. (2021) Enzyme replacement combinational therapy: effective treatments for mucopolysaccharidoses. Expert Opinion on Biological Therapy 21:9, pages 1181-1197.
Read now
Read now
Shawn C. Aylward, Jonathan Pindrik, Nicolas J. Abreu, W. Bruce Cherny, Matthew O’Neal & Emily de Los Reyes. (2020) Cerliponase alfa for CLN2 disease, a promising therapy. Expert Opinion on Orphan Drugs 8:11, pages 445-454.
Read now
Read now
Anna Tylki-Szymańska & Agnieszka Jurecka. (2015) Prospective therapies for mucopolysaccharidoses. Expert Opinion on Orphan Drugs 3:11, pages 1241-1253.
Read now
Read now
Articles from other publishers (31)
Claire O’Leary, Gabriella Forte, Nadia L. Mitchell, Amir Saam Youshani, Adam Dyer, Martin P. Wellby, Katharina N. Russell, Samantha J. Murray, Nelly Jolinon, Simon A Jones, Kevin Stacey, Daniel M. Davis, Els Henckaerts, David N. Palmer, Ian Kamaly-Asl & Brian W. Bigger. (2023) Intraparenchymal convection enhanced delivery of AAV in sheep to treat Mucopolysaccharidosis IIIC. Journal of Translational Medicine 21:1.
Crossref
Crossref
Weijun Sun, Tianming Liu, Haojun Lei, Xinrong Wei, Lixia Li, Hongxing Chen, Dan Li & Lingtian Xie. (2023)
Evaluation of Cyclophosphamide on the Behavior and Reproduction of
Daphnia magna
. ACS ES&T Water 3:4, pages 996-1006.
Crossref
Crossref
Oscar González-Davis, Maria V. Villagrana-Escareño, Mario A. Trujillo, Pedro Gama, Kanchan Chauhan & Rafael Vazquez-Duhalt. (2023) Virus-like nanoparticles as enzyme carriers for Enzyme Replacement Therapy (ERT). Virology 580, pages 73-87.
Crossref
Crossref
Wei Zhan, Manish Muhuri, Phillip W. L. Tai & Guangping Gao. (2021) Vectored Immunotherapeutics for Infectious Diseases: Can rAAVs Be The Game Changers for Fighting Transmissible Pathogens?. Frontiers in Immunology 12.
Crossref
Crossref
Jennifer T. Saville, Ainslie L.K. Derrick-RobertsChantelle McIntyreMaria Fuller. (2021) Systemic scAAV9.U1a.hSGSH Delivery Corrects Brain Biochemistry in Mucopolysaccharidosis Type IIIA at Early and Later Stages of Disease. Human Gene Therapy 32:7-8, pages 420-430.
Crossref
Crossref
Françoise Piguet, Timothée de Saint Denis, Emilie Audouard, Kevin Beccaria, Arthur André, Guillaume Wurtz, Raphael Schatz, Sandro Alves, Caroline SevinMichel Zerah & Nathalie Cartier. (2021) The Challenge of Gene Therapy for Neurological Diseases: Strategies and Tools to Achieve Efficient Delivery to the Central Nervous System. Human Gene Therapy 32:7-8, pages 349-374.
Crossref
Crossref
Esmee Oussoren, Margreet A.E.M. Wagenmakers, Bianca Link, Jan C. van der Meijden, W.W.M. Pim Pijnappel, George J.G. Ruijter, Mirjam Langeveld & Ans T. van der Ploeg. (2021) Hip disease in Mucopolysaccharidoses and Mucolipidoses: A review of mechanisms, interventions and future perspectives. Bone 143, pages 115729.
Crossref
Crossref
Lalitha R. Belur, Kelly M. Podetz-Pedersen, Thuy An Tran, Joshua A. Mesick, Nathaniel M. Singh, Maureen Riedl, Lucy Vulchanova, Karen F. Kozarsky & R. Scott McIvor. (2020) Intravenous delivery for treatment of mucopolysaccharidosis type I: A comparison of AAV serotypes 9 and rh10. Molecular Genetics and Metabolism Reports 24, pages 100604.
Crossref
Crossref
Joost van Haasteren, Jie Li, Olivia J. Scheideler, Niren Murthy & David V. Schaffer. (2020) The delivery challenge: fulfilling the promise of therapeutic genome editing. Nature Biotechnology 38:7, pages 845-855.
Crossref
Crossref
Tamer A. Mansour, Kevin D. Woolard, Karen L. Vernau, Devin M. Ancona, Sara M. Thomasy, Lionel Sebbag, Bret A. Moore, Marguerite F. Knipe, Haitham A. Seada, Tina M. Cowan, Miriam Aguilar, C. Titus Brown & Danika L. Bannasch. (2020) Whole genome sequencing for mutation discovery in a single case of lysosomal storage disease (MPS type 1) in the dog. Scientific Reports 10:1.
Crossref
Crossref
Marcos Almeida Matos, Fábio Ferri-de-Barros & Roberto Guarniero. (2018) Quality of life evaluation in patients with mucopolysaccharidosis using PedsQL. Journal of Child Health Care 23:2, pages 278-285.
Crossref
Crossref
Cinzia M. Bellettato & Maurizio Scarpa. (2018) Possible strategies to cross the blood–brain barrier. Italian Journal of Pediatrics 44:S2.
Crossref
Crossref
Rita Barone, Alessandra Pellico, Annarita Pittalà & Serena Gasperini. (2018) Neurobehavioral phenotypes of neuronopathic mucopolysaccharidoses. Italian Journal of Pediatrics 44:S2.
Crossref
Crossref
Veronica Jimenez, Claudia Jambrina, Estefania Casana, Victor Sacristan, Sergio Muñoz, Sara Darriba, Jordi Rodó, Cristina Mallol, Miquel Garcia, Xavier León, Sara Marcó, Albert Ribera, Ivet Elias, Alba Casellas, Ignasi Grass, Gemma Elias, Tura Ferré, Sandra Motas, Sylvie Franckhauser, Francisca Mulero, Marc Navarro, Virginia Haurigot, Jesus Ruberte & Fatima Bosch. (2018) FGF21 gene therapy as treatment for obesity and insulin resistance. EMBO Molecular Medicine 10:8.
Crossref
Crossref
Josefine Radke, Carsten G. Bönnemann, Werner Stenzel & Hans-H. Goebel. 2018. Developmental Neuropathology. Developmental Neuropathology
293
311
.
Joost M Costerus, Matthijs C Brouwer & Diederik van de Beek. (2018) Technological advances and changing indications for lumbar puncture in neurological disorders. The Lancet Neurology 17:3, pages 268-278.
Crossref
Crossref
Dirnete Diel, Valeska Lizzi Lagranha, Roselena Silvestri Schuh, Fernanda Bruxel, Ursula Matte & Helder Ferreira Teixeira. (2018) Optimization of alginate microcapsules containing cells overexpressing α-l-iduronidase using Box-Behnken design. European Journal of Pharmaceutical Sciences 111, pages 29-37.
Crossref
Crossref
Wenyong Tong, Chrissa A. Dwyer, Bryan E. Thacker, Charles A. Glass, Jillian R. Brown, Kristina Hamill, Kelley W. Moremen, Stéphane Sarrazin, Philip L.S.M. Gordts, Lara E. Dozier, Gentry N. Patrick, Yitzhak Tor & Jeffrey D. Esko. (2017) Guanidinylated Neomycin Conjugation Enhances Intranasal Enzyme Replacement in the Brain. Molecular Therapy 25:12, pages 2743-2752.
Crossref
Crossref
Belal Azab, Zain Dardas, Mohannad Hamarsheh, Mohammad Alsalem, Zaid Kilani, Farah Kilani, Abdalla Awidi, Hanan Jafar & Sami Amr. (2017) Novel frameshift variant in the IDUA gene underlies Mucopolysaccharidoses type I in a consanguineous Yemeni pedigree. Molecular Genetics and Metabolism Reports 12, pages 76-79.
Crossref
Crossref
Melani Solomon & Silvia Muro. (2017) Lysosomal enzyme replacement therapies: Historical development, clinical outcomes, and future perspectives. Advanced Drug Delivery Reviews 118, pages 109-134.
Crossref
Crossref
Lalitha R. Belur, Alexa Temme, Kelly M. Podetz-Pedersen, Maureen Riedl, Lucy Vulchanova, Nicholas Robinson, Leah R. Hanson, Karen F. KozarskyPaul J. Orchard, William H. FreyIIII, Walter C. Low & R. Scott McIvor. (2017) Intranasal Adeno-Associated Virus Mediated Gene Delivery and Expression of Human Iduronidase in the Central Nervous System: A Noninvasive and Effective Approach for Prevention of Neurologic Disease in Mucopolysaccharidosis Type I. Human Gene Therapy 28:7, pages 576-587.
Crossref
Crossref
Natalie S. Rodriguez, Lisa Yanuaria, Kevin Murphy R. Parducho, Irving M. Garcia, Bino A. Varghese, Brendan H. Grubbs & Toshio Miki. (2017) Liver-Directed Human Amniotic Epithelial Cell Transplantation Improves Systemic Disease Phenotype in Hurler Syndrome Mouse Model. Stem Cells Translational Medicine 6:7, pages 1583-1594.
Crossref
Crossref
Carles Roca, Sandra Motas, Sara Marcó, Albert Ribera, Víctor Sánchez, Xavier Sánchez, Joan Bertolin, Xavier León, Jennifer Pérez, Miguel Garcia, Pilar Villacampa, Jesús Ruberte, Anna Pujol, Virginia Haurigot & Fatima Bosch. (2017) Disease correction by AAV-mediated gene therapy in a new mouse model of mucopolysaccharidosis type IIID. Human Molecular Genetics 26:8, pages 1535-1551.
Crossref
Crossref
Yasunori Matsuzaki, Ayumu Konno, Ryo Mukai, Fumiaki Honda, Masafumi Hirato, Yuhei Yoshimoto & Hirokazu Hirai. (2016) Transduction Profile of the Marmoset Central Nervous System Using Adeno-Associated Virus Serotype 9 Vectors. Molecular Neurobiology 54:3, pages 1745-1758.
Crossref
Crossref
Esteban Alberto Gonzalez & Guilherme Baldo. (2017) Gene Therapy for Lysosomal Storage Disorders. Journal of Inborn Errors of Metabolism and Screening 5, pages 232640981668978.
Crossref
Crossref
Gregory A. Grabowski & Chester Whitley. (2017) Ten plus one challenges in diseases of the lysosomal system. Molecular Genetics and Metabolism 120:1-2, pages 38-46.
Crossref
Crossref
J.D. García Díaz, J.M. Mesa Latorre, D. Corps Fernández & A. Valbuena Parra. (2016) Enfermedades por depósito lisosomal. Medicine - Programa de Formación Médica Continuada Acreditado 12:19, pages 1072-1081.
Crossref
Crossref
F. Feillet, A. Wiedemann, E. Jeannesson, R. Jaussaud & P. Journeau. (2016) Mucopolisacaridosis. EMC - Pediatría 51:3, pages 1-14.
Crossref
Crossref
Sandra Motas, Virginia Haurigot, Miguel Garcia, Sara Marc?Albert Ribera, Carles Roca, Xavier S?nchez, V?ctor S?nchez, Maria Molas, Joan Bertolin, Luca Maggioni, Xavier Le?n, Jes?s Ruberte & Fatima Bosch. (2016) CNS-directed gene therapy for the treatment of neurologic and somatic mucopolysaccharidosis type II (Hunter syndrome). JCI Insight 1:9.
Crossref
Crossref
Megan E. Bosch & Tammy Kielian. (2015) Neuroinflammatory paradigms in lysosomal storage diseases. Frontiers in Neuroscience 9.
Crossref
Crossref
Qiu-Juan Liang, Mei Jiang, Xin-Hong Wang, Li-Li Le, Meng Xiang, Ning Sun, Dan Meng & Si-Feng Chen. (2015) Pre-existing interleukin 10 in cerebral arteries attenuates subsequent brain injury caused by ischemia/reperfusion. IUBMB Life 67:9, pages 710-719.
Crossref
Crossref