McClintock B. The origin and behavior of mutable loci in maize. Proc Natl Acad Sci U S A 1950, 36: 344–355.
Article CAS PubMed Central PubMed Google Scholar
Siguier P, Gourbeyre E, Chandler M. Bacterial insertion sequences: their genomic impact and diversity. FEMS Microbiol Rev 2014, 38: 865–891.
Article CAS PubMed Google Scholar
Durrant MG, Perry NT, Pai JJ, Jangid AR, Athukoralage JS, Hiraizumi M. Bridge RNAs direct programmable recombination of target and donor DNA. Nature 2024, 630: 984–993.
Article CAS PubMed Central PubMed Google Scholar
Hoersten J, Ruiz-Gómez G, Paszkowski-Rogacz M, Gilioli G, Guillem-Gloria PM, Lansing F, et al. Engineering spacer specificity of the Cre/loxP system. Nucleic Acids Res 2024, 52: 8017–8031.
Article CAS PubMed Central PubMed Google Scholar
Qin Y, Li S, Li XJ, Yang S. CRISPR-based genome-editing tools for Huntington’s disease research and therapy. Neurosci Bull 2022, 38: 1397–1408.
Article PubMed Central PubMed Google Scholar
Yu J, Li T, Chen K, Tang Q, Zhu J. Stereopure AIMer: a promising RNA base-editing tool for monogenic neurological diseases. Neurosci Bull 2023, 39: 353–355.
Article CAS PubMed Google Scholar
Doman JL, Pandey S, Neugebauer ME, An M, Davis JR, Randolph PB, et al. Phage-assisted evolution and protein engineering yield compact, efficient prime editors. Cell 2023, 186: 3983-4002.e26.
Article CAS PubMed Central PubMed Google Scholar
Hiraizumi M, Perry NT, Durrant MG, Soma T, Nagahata N, Okazaki S, et al. Structural mechanism of bridge RNA-guided recombination. Nature 2024, 630: 994–1002.
Article CAS PubMed Central PubMed Google Scholar
Zain R, Edvard Smith CI. Targeted oligonucleotides for treating neurodegenerative tandem repeat diseases. Neurotherapeutics 2019, 16: 248–262.
Article CAS PubMed Central PubMed Google Scholar
Li HL, Zhang YB, Wu ZY. Development of research on Huntington disease in China. Neurosci Bull 2017, 33: 312–316.
Duan D, Goemans N, Takeda S, Mercuri E, Aartsma-Rus A. Duchenne muscular dystrophy. Nat Rev Dis Primers 2021, 7: 13.
Article PubMed Central PubMed Google Scholar
Paulson HL, Shakkottai VG, Brent Clark H, Orr HT. Polyglutamine spinocerebellar ataxias—from genes to potential treatments. Nat Rev Neurosci 2017, 18: 613–626.
Article CAS PubMed Central PubMed Google Scholar
Campuzano V, Montermini L, Moltò MD, Pianese L, Cossée M, Cavalcanti F, et al. Friedreich’s Ataxia: autosomal recessive disease caused by an intronic GAA triplet repeat expansion. Science 1996, 271: 1423–1427.
Article CAS PubMed Google Scholar
Berry-Kravis EM, Lindemann L, Jønch AE, Apostol G, Bear MF, Carpenter RL, et al. Drug development for neurodevelopmental disorders: lessons learned from fragile X syndrome. Nat Rev Drug Discov 2018, 17: 280–299.
Article CAS PubMed Google Scholar
Chen PJ, Hussmann JA, Yan J, Knipping F, Ravisankar P, Chen PF, et al. Enhanced prime editing systems by manipulating cellular determinants of editing outcomes. Cell 2021, 184: 5635-5652.e29.
Article CAS PubMed Central PubMed Google Scholar
Yang J, Luly KM, Green JJ. Nonviral nanoparticle gene delivery into the CNS for neurological disorders and brain cancer applications. WIREs Nanomed Nanobiotechnol 2023, 15: e1853.
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