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Editorial
. 2023 Aug;65(8):998.
doi: 10.1111/dmcn.15584. Epub 2023 Apr 27.

The continuing challenges facing gene-targeted therapies

Nina F Schor  1
Affiliations
Free article
Editorial

The continuing challenges facing gene-targeted therapies

Nina F Schor. Dev Med Child Neurol. 2023 Aug.
Free article
No abstract available

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References

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    1. Jiang S, Huang Z, Li Y, et al. Single-cell chromatin accessibility and transcriptome atlas of mouse embryos. Cell Rep 2023; 42: 112210.
    1. Amanat M, Nemeth CL, Fine AS, Leung DG, Fatemi A. Antisense oligonucleotide therapy for the nervous system: From bench to bedside with emphasis on pediatric neurology. Pharmaceutics 2022; 14: 2389.
    1. Lekstrom-Himes J, Brooks PJ, Koeberl DD, et al. Moving away from one disease at a time: Screening, trial design, and regulatory implications of novel platform technologies. Am J Med Genet C Semin Med Genet 2023; 193: 30-43.

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