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​References

  1. Medina A, Mahjoub Y, Shaver L, Pringsheim T. Prevalence and Incidence of Huntington's Disease: An Updated Systematic Review and Meta-Analysis. Movement Disorders. 2022;37(12):2327-35.

  2. Bates GP, Dorsey R, Gusella JF, Hayden MR, Kay C, Leavitt BR, et al. Huntington disease. Nat Rev Dis Primers. 2015;1:15005.

  3. MacDonald ME, Ambrose CM, Duyao MP, Myers RH, Lin C, Srinidhi L, et al. A novel gene containing a trinucleotide repeat that is expanded and unstable on Huntington's disease chromosomes. Cell. 1993;72(6):971-83.

  4. Tabrizi SJ, Estevez-Fraga C, van Roon-Mom WMC, Flower MD, Scahill RI, Wild EJ, et al. Potential disease-modifying therapies for Huntington's disease: lessons learned and future opportunities. The Lancet Neurology. 2022;21(7):645-58.

  5. Tabrizi SJ, Flower MD, Ross CA, Wild EJ. Huntington disease: new insights into molecular pathogenesis and therapeutic opportunities. Nature Reviews Neurology. 2020;16(10):529-46.

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  7. Roche. What is Huntington’s disease? [Internet]. www.youtube.com. 2019 [cited 2024 Jan 11]. Available from: https://www.youtube.com/watch?v=X89AjFBpmu0&t=202s

  8. Ferguson MW, Kennedy CJ, Palpagama TH, Waldvogel HJ, Faull RLM, Kwakowsky A. Current and Possible Future Therapeutic Options for Huntington’s Disease. Journal of Central Nervous System Disease. 2022;14:11795735221092517.

  9. Bennett CF, Swayze EE. RNA Targeting Therapeutics: Molecular Mechanisms of Antisense Oligonucleotides as a Therapeutic Platform. Annual Review of Pharmacology and Toxicology. 2010;50(1):259-93.

  10. Pfister EL, Kennington L, Straubhaar J, Wagh S, Liu W, DiFiglia M, et al. Five siRNAs Targeting Three SNPs May Provide Therapy for Three-Quarters of Huntington's Disease Patients. Current Biology. 2009;19(9):774-8.

  11. Kay C, Collins JA, Skotte NH, Southwell AL, Warby SC, Caron NS, et al. Huntingtin Haplotypes Provide Prioritized Target Panels for Allele-specific Silencing in Huntington Disease Patients of European Ancestry. Molecular Therapy. 2015;23(11):1759-71.

  12. Cerritelli SM, Crouch RJ. Ribonuclease H: the enzymes in eukaryotes. Febs j. 2009;276(6):1494-505.

  13. Kellum AH, Jr., Pallan PS, Nilforoushan A, Sturla SJ, Stone MP, Egli M. Conformation and Pairing Properties of an O6-Methyl-2′-deoxyguanosine-Directed Benzimidazole Nucleoside Analog in Duplex DNA. Chemical Research in Toxicology. 2022;35(10):1903-13.

  14. Rook ME, Southwell AL. Antisense Oligonucleotide Therapy: From Design to the Huntington Disease Clinic. BioDrugs. 2022;36(2):105-19.

  15. Kandasamy P, McClorey G, Shimizu M, Kothari N, Alam R, Iwamoto N, et al. Control of backbone chemistry and chirality boost oligonucleotide splice switching activity. Nucleic Acids Research. 2022;50(10):5443-66.

  16. Roberts TC, Langer R, Wood MJA. Advances in oligonucleotide drug delivery. Nature Reviews Drug Discovery. 2020;19(10):673-94.

  17. Pediatrician MA. INTRATHECAL DRUG ADMINISTRATION AND CSF SAMPLE | ANIMATION | [Internet]. www.youtube.com. 2023 [cited 2024 Jan 11]. Available from: https://www.youtube.com/watch?v=2VRGiKtt6pM

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Some images (i.e., the location of SNP rs362307, the chemical modification of the ASO67) are created by BioRender and ChemDraw 20.1. 

Other images and videos are from Wix or the Pixabay - in which case licences have been obtained for each and they are free to use.​

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© 2024 Ariella Pharmaceuticals

 

Disclaimer: Ariella Pharmaceuticals is a fictional company and Nominsen is a fictitious product. They were both created for University College London's BIOC0015 coursework. Any similarity to real businesses, individuals, or other technologies is coincidental. 

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