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Cell and Gene Therapies for Mucopolysaccharidoses: Base Editing and Therapeutic Delivery to the CNS

Department of Biology, Centre for Biomedical Research, University of Victoria, 3800 Finnerty Rd., Victoria, BC V8P 5C2, Canada
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Diseases 2019, 7(3), 47; https://doi.org/10.3390/diseases7030047
Received: 11 June 2019 / Revised: 21 June 2019 / Accepted: 25 June 2019 / Published: 26 June 2019
(This article belongs to the Collection Lysosomal Storage Diseases)
Although individually uncommon, rare diseases collectively account for a considerable proportion of disease impact worldwide. A group of rare genetic diseases called the mucopolysaccharidoses (MPSs) are characterized by accumulation of partially degraded glycosaminoglycans cellularly. MPS results in varied systemic symptoms and in some forms of the disease, neurodegeneration. Lack of treatment options for MPS with neurological involvement necessitates new avenues of therapeutic investigation. Cell and gene therapies provide putative alternatives and when coupled with genome editing technologies may provide long term or curative treatment. Clustered regularly interspaced short palindromic repeats (CRISPR)-based genome editing technology and, more recently, advances in genome editing research, have allowed for the addition of base editors to the repertoire of CRISPR-based editing tools. The latest versions of base editors are highly efficient on-targeting deoxyribonucleic acid (DNA) editors. Here, we describe a number of putative guide ribonucleic acid (RNA) designs for precision correction of known causative mutations for 10 of the MPSs. In this review, we discuss advances in base editing technologies and current techniques for delivery of cell and gene therapies to the site of global degeneration in patients with severe neurological forms of MPS, the central nervous system, including ultrasound-mediated blood-brain barrier disruption. View Full-Text
Keywords: mucopolysaccharidosis; base editing; therapeutic; central nervous system; clustered regularly interspaced short palindromic repeats; gene therapies; lysosomal disease; blood-brain barrier; molecular Trojan horse; ultrasound-mediated blood-brain barrier disruption mucopolysaccharidosis; base editing; therapeutic; central nervous system; clustered regularly interspaced short palindromic repeats; gene therapies; lysosomal disease; blood-brain barrier; molecular Trojan horse; ultrasound-mediated blood-brain barrier disruption
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Christensen, C.L.; Ashmead, R.E.; Choy, F.Y.M. Cell and Gene Therapies for Mucopolysaccharidoses: Base Editing and Therapeutic Delivery to the CNS. Diseases 2019, 7, 47.

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