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Mucopolysaccharidosis-Plus Syndrome
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Genome Editing for Mucopolysaccharidoses

1
Gene Therapy Center, Hospital de Clinicas de Porto Alegre, Porto Alegre 90035-007, Brazil
2
Post-Graduate Program in Genetics and Molecular Biology, Universidade Federal do Rio Grande do Sul, Porto Alegre 91501-970, Brazil
3
Department of Pediatrics, Stanford University, Stanford, CA 94305, USA
*
Author to whom correspondence should be addressed.
Int. J. Mol. Sci. 2020, 21(2), 500; https://doi.org/10.3390/ijms21020500
Received: 1 November 2019 / Revised: 8 January 2020 / Accepted: 9 January 2020 / Published: 13 January 2020
(This article belongs to the Special Issue Mucopolysaccharidoses: Diagnosis, Treatment, and Management)
Genome editing holds the promise of one-off and potentially curative therapies for many patients with genetic diseases. This is especially true for patients affected by mucopolysaccharidoses as the disease pathophysiology is amenable to correction using multiple approaches. Ex vivo and in vivo genome editing platforms have been tested primarily on MSPI and MPSII, with in vivo approaches having reached clinical testing in both diseases. Though we still await proof of efficacy in humans, the therapeutic tools established for these two diseases should pave the way for other mucopolysaccharidoses. Herein, we review the current preclinical and clinical development studies, using genome editing as a therapeutic approach for these diseases. The development of new genome editing platforms and the variety of genetic modifications possible with each tool provide potential applications of genome editing for mucopolysaccharidoses, which vastly exceed the potential of current approaches. We expect that in a not-so-distant future, more genome editing-based strategies will be established, and individual diseases will be treated through multiple approaches. View Full-Text
Keywords: genome editing; gene therapy; mucopolysaccharidoses; lysosomal storage disease; CRISPR/Cas9; Hurler; Hunter; Zinc Finger Nucleases; viral vectors; non-viral vectors; hematopoietic stem cell transplantation genome editing; gene therapy; mucopolysaccharidoses; lysosomal storage disease; CRISPR/Cas9; Hurler; Hunter; Zinc Finger Nucleases; viral vectors; non-viral vectors; hematopoietic stem cell transplantation
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Poletto, E.; Baldo, G.; Gomez-Ospina, N. Genome Editing for Mucopolysaccharidoses. Int. J. Mol. Sci. 2020, 21, 500.

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