Editing Nucleotide (section)

==Medical applications of synthetic nucleotides==
The applications of synthetic nucleotides vary widely and include disease diagnosis, treatment, or precision medicine. 

# '''Antiviral or Antiretroviral agents:''' several nucleotide derivatives have been used in the treatment against infection with [[Hepatitis]] and [[HIV]].<ref>{{cite journal | vauthors = Ramesh D, Vijayakumar BG, Kannan T | title = Therapeutic potential of uracil and its derivatives in countering pathogenic and physiological disorders | journal = European Journal of Medicinal Chemistry | volume = 207 | pages = 112801 | date = December 2020 | pmid = 32927231 | doi = 10.1016/j.ejmech.2020.112801 | s2cid = 221724578 }}</ref><ref>{{cite journal | vauthors = Ramesh D, Vijayakumar BG, Kannan T | title = Advances in Nucleoside and Nucleotide Analogues in Tackling Human Immunodeficiency Virus and Hepatitis Virus Infections | journal = ChemMedChem | volume = 16 | issue = 9 | pages = 1403–1419 | date = May 2021 | pmid = 33427377 | doi = 10.1002/cmdc.202000849 | url = https://chemistry-europe.onlinelibrary.wiley.com/doi/epdf/10.1002/cmdc.202000849 | access-date = 13 March 2021 | url-status = dead | s2cid = 231576801 | archive-url = https://web.archive.org/web/20211214220544/https://chemistry-europe.onlinelibrary.wiley.com/doi/epdf/10.1002/cmdc.202000849 | archive-date = 14 December 2021 | url-access = subscription }}</ref> Examples of direct nucleoside analog reverse-transcriptase inhibitors ([[Reverse-transcriptase inhibitor|NRTIs]]) include [[Tenofovir disoproxil]], [[Tenofovir alafenamide]], and [[Sofosbuvir]]. On the other hand, agents such as [[Mericitabine]], [[Lamivudine]], [[Entecavir]] and [[Telbivudine]] must first undergo metabolization via phosphorylation to become activated. 
# '''Antisense oligonucleotides (ASO)''': synthetic [[Oligonucleotide|oligonucleotides]] have been used in the treatment of rare heritable diseases since they can bind specific [[RNA]] transcripts and ultimately modulate protein expression. [[Spinal muscular atrophy]], [[ALS|amyotrophic lateral sclerosis]], [[Familial hypercholesterolemia|homozygous familial hypercholesterolemia]], and [[Primary hyperoxaluria|primary hyperoxaluria type 1]] are all amenable to ASO-based therapy.<ref>{{cite journal | vauthors = Lauffer MC, van Roon-Mom W, Aartsma-Rus A | title = Possibilities and limitations of antisense oligonucleotide therapies for the treatment of monogenic disorders | journal = Communications Medicine | volume = 4 | issue = 1 | pages = 6 | date = January 2024 | pmid = 38182878 | pmc = 10770028 | doi = 10.1038/s43856-023-00419-1 }}</ref> The application of oligonucleotides is a new frontier in precision medicine and management of conditions which are untreatable.
# '''Synthetic guide RNA (gRNA)''': synthetic nucleotides can be used to design [[Guide RNA|gRNA]] which are essential for the proper function of gene-editing technologies such as [[CRISPR gene editing|CRISPR-Cas9]].