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Expert Opinion on Drug Delivery Volume 11, 2014 - Issue 3
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Gene delivery strategies for the treatment of mucopolysaccharidoses

Guilherme BaldoCentro de Terapia Gênica- Hospital de Clinicas de Porto Alegre, Ramiro Barcelos, 2350, Porto Alegre, RS 90035-903, Brazil
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Roberto GiuglianiCentro de Terapia Gênica- Hospital de Clinicas de Porto Alegre, Ramiro Barcelos, 2350, Porto Alegre, RS 90035-903, Brazil;Programa de Pós-Graduação em genética e Biologia Molecular, UFRGS, Ramiro Barcelos, 2350, Porto Alegre, RS 90035-903, Brazil+55 513 359 8010; [email protected]
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Ursula MatteCentro de Terapia Gênica- Hospital de Clinicas de Porto Alegre, Ramiro Barcelos, 2350, Porto Alegre, RS 90035-903, Brazil
Pages 449-459 | Published online: 23 Jan 2014

Citations (13)

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Read on this site (2)

Roselena S. Schuh, Guilherme Baldo & Helder F. Teixeira. (2016) Nanotechnology applied to treatment of mucopolysaccharidoses. Expert Opinion on Drug Delivery 13:12, pages 1709-1718.
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Roberto Giugliani, Andressa Federhen, Filippo Vairo, Cláudia Vanzella, Gabriela Pasqualim, Letícia Machado Rosa da Silva, Luciana Giugliani, Ana Paula Kurz de Boer, Carolina Fishinger Moura de Souza, Ursula Matte & Guilherme Baldo. (2016) Emerging drugs for the treatment of mucopolysaccharidoses. Expert Opinion on Emerging Drugs 21:1, pages 9-26.
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Articles from other publishers (11)

Alexis David, Thibault Chazeirat, Ahlame Saidi, Gilles Lalmanach & Fabien Lecaille. (2023) The Interplay of Glycosaminoglycans and Cysteine Cathepsins in Mucopolysaccharidosis. Biomedicines 11:3, pages 810.
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Edina Poletto, Guilherme Baldo & Natalia Gomez-Ospina. (2020) Genome Editing for Mucopolysaccharidoses. International Journal of Molecular Sciences 21:2, pages 500.
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Tran Kiem Hao, Nguyen Thi Diem Chi, Nguyen Thi Hong Duc & Nguyen Thi Kim Hoa. (2020) A case study of three patients with mucopolysaccharidoses in Hue Central Hospital. SAGE Open Medical Case Reports 8, pages 2050313X2093824.
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Florian B. Lagler. 2020. Pediatric Pharmacotherapy. Pediatric Pharmacotherapy 39 56 .
Roselena Silvestri Schuh, Édina Poletto, Gabriela Pasqualim, Angela Maria Vicente Tavares, Fabíola Shons Meyer, Esteban Alberto Gonzalez, Roberto Giugliani, Ursula Matte, Helder Ferreira Teixeira & Guilherme Baldo. (2018) In vivo genome editing of mucopolysaccharidosis I mice using the CRISPR/Cas9 system. Journal of Controlled Release 288, pages 23-33.
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Juliana Bidone, Roselena Silvestri Schuh, Mirian Farinon, Édina Poletto, Gabriela Pasqualim, Patrícia Gnieslaw de Oliveira, Michelle Fraga, Ricardo Machado Xavier, Guilherme Baldo, Helder Ferreira Teixeira & Ursula Matte. (2018) Intra-articular nonviral gene therapy in mucopolysaccharidosis I mice. International Journal of Pharmaceutics 548:1, pages 151-158.
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Roselena Silvestri Schuh, Talita Giacomet de Carvalho, Roberto Giugliani, Ursula Matte, Guilherme Baldo & Helder Ferreira Teixeira. (2018) Gene editing of MPS I human fibroblasts by co-delivery of a CRISPR/Cas9 plasmid and a donor oligonucleotide using nanoemulsions as nonviral carriers. European Journal of Pharmaceutics and Biopharmaceutics 122, pages 158-166.
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Valeska Lizzi Lagranha, Barbara Zambiasi Martinelli, Guilherme Baldo, Giuseppe Ávila Testa, Talita Giacomet de Carvalho, Roberto Giugliani & Ursula Matte. (2017) Subcutaneous implantation of microencapsulated cells overexpressing α-L-iduronidase for mucopolysaccharidosis type I treatment. Journal of Materials Science: Materials in Medicine 28:3.
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Esteban Alberto Gonzalez & Guilherme Baldo. (2017) Gene Therapy for Lysosomal Storage Disorders. Journal of Inborn Errors of Metabolism and Screening 5, pages 232640981668978.
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Talita Giacomet de Carvalho, Ursula da Silveira Matte, Roberto Giugliani & Guilherme Baldo. (2015) Genome Editing: Potential Treatment for Lysosomal Storage Diseases. Current Stem Cell Reports 1:1, pages 9-15.
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Shunji Tomatsu, Tsutomu Shimada, Robert Mason, Adriana Montaño, Joan Kelly, William LaMarr, Francyne Kubaski, Roberto Giugliani, Aratrik Guha, Eriko Yasuda, William Mackenzie, Seiji Yamaguchi, Yasuyuki Suzuki & Tadao Orii. (2014) Establishment of Glycosaminoglycan Assays for Mucopolysaccharidoses. Metabolites 4:3, pages 655-679.
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