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

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dc.contributor.authorChristensen, Chloe L.
dc.contributor.authorAshmead, Rhea E.
dc.contributor.authorChoy, Francis Y. M.
dc.date.accessioned2019-07-22T16:18:22Z
dc.date.available2019-07-22T16:18:22Z
dc.date.copyright2019en_US
dc.date.issued2019
dc.description.abstractAlthough 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.en_US
dc.description.reviewstatusRevieweden_US
dc.description.scholarlevelFacultyen_US
dc.description.sponsorshipThis research was funded by an operating grant from the Sanfilippo Children’s Research Foundation, and the BC Children’s Hospital Foundation and Rare Disease Foundation, grant number 2326.en_US
dc.identifier.citationChristensen, C.L., Ashmead, R.E. & Choy, F.Y.M. (2019). Cell and Gene Therapies for Mucopolysaccharidoses: Base Editing and Therapeutic Delivery to the CNS. Diseases, 7(3), 47. https://doi.org/10.3390/diseases7030047en_US
dc.identifier.urihttps://doi.org/10.3390/diseases7030047
dc.identifier.urihttp://hdl.handle.net/1828/10981
dc.language.isoenen_US
dc.publisherDiseasesen_US
dc.subjectmucopolysaccharidosis
dc.subjectbase editing
dc.subjecttherapeutic
dc.subjectcentral nervous system
dc.subjectclustered regularly interspaced short palindromic repeats
dc.subjectgene therapies
dc.subjectlysosomal disease
dc.subjectblood-brain barrier
dc.subjectmolecular Trojan horse
dc.subjectultrasound-mediated blood-brain barrier disruption
dc.subjectCentre for Biomedical Research
dc.subject.departmentDepartment of Biology
dc.titleCell and Gene Therapies for Mucopolysaccharidoses: Base Editing and Therapeutic Delivery to the CNSen_US
dc.typeArticleen_US

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