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Cell and Gene Therapies for Mucopolysaccharidoses: Base Editing and Therapeutic Delivery to the CNS.
Diseases. 2019 Jun 26;7(3):47. doi: 10.3390/diseases7030047.
Diseases. 2019.
PMID: 31248000
Free PMC article.
Review.
A Prospective Treatment Option for Lysosomal Storage Diseases: CRISPR/Cas9 Gene Editing Technology for Mutation Correction in Induced Pluripotent Stem Cells.
Christensen CL, Choy FYM.
Christensen CL, et al.
Diseases. 2017 Feb 24;5(1):6. doi: 10.3390/diseases5010006.
Diseases. 2017.
PMID: 28933359
Free PMC article.
Review.
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Base editing corrects the common Salla disease SLC17A5 c.115C>T variant.
Harb JF, Christensen CL, Kan SH, Rha AK, Andrade-Heckman P, Pollard L, Steet R, Huang JY, Wang RY.
Harb JF, et al. Among authors: christensen cl.
Mol Ther Nucleic Acids. 2023 Aug 26;34:102022. doi: 10.1016/j.omtn.2023.08.024. eCollection 2023 Dec 12.
Mol Ther Nucleic Acids. 2023.
PMID: 37727271
Free PMC article.
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Progranulin as a Novel Factor in Gaucher Disease.
Choy FYM, Christensen CL.
Choy FYM, et al. Among authors: christensen cl.
EBioMedicine. 2016 Nov;13:13-14. doi: 10.1016/j.ebiom.2016.11.006. Epub 2016 Nov 5.
EBioMedicine. 2016.
PMID: 27836396
Free PMC article.
No abstract available.
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