Editing Human genetic enhancement (section)

=== Gene therapy ===
{{Main|Gene therapy}}

Modification of human genes in order to treat genetic diseases is referred to as [[gene therapy]]. Gene therapy is a medical procedure that involves inserting genetic material into a patient's cells to repair or fix a malfunctioning gene in order to treat hereditary illnesses. Between 1989 and December 2018, over 2,900 [[clinical trial]]s of gene therapies were conducted, with more than half of them in [[Phases of clinical research|phase I]].<ref name="JGenMed Database">{{cite web |date=June 2016 |title=Gene Therapy Clinical Trials Worldwide Database |url=https://a873679.fmphost.com/fmi/webd/GTCT |work=The Journal of Gene Medicine }}s</ref> Since that time, many gene therapy based drugs became available, such as [[Zolgensma]] and [[Patisiran]]. Most of these approaches utilize [[viral vector]]s, such as [[adeno-associated virus]]es (AAVs), adenoviruses (AV) and [[lentivirus]]es (LV), for inserting or replacing [[transgene]]s ''[[in vivo]]'' or ''[[ex vivo]]''.<ref>{{cite journal | vauthors = Li X, Le Y, Zhang Z, Nian X, Liu B, Yang X | title = Viral Vector-Based Gene Therapy | journal = International Journal of Molecular Sciences | volume = 24 | issue = 9 | page = 7736 | date = April 2023 | pmid = 37175441 | pmc = 10177981 | doi = 10.3390/ijms24097736 | doi-access = free }}</ref><ref>{{cite journal | vauthors = Lundstrom K | title = Viral Vectors in Gene Therapy: Where Do We Stand in 2023? | journal = Viruses | volume = 15 | issue = 3 | page = 698 | date = March 2023 | pmid = 36992407 | pmc = 10059137 | doi = 10.3390/v15030698 | doi-access = free }}</ref>

In 2023, [[nanoparticle]]s that act similarly to viral vectors were created. These nanoparticles, called ''bioorthgonal engineered virus-like recombinant biosomes'', display strong and rapid binding capabilities to [[LDL receptor]]s on cell surfaces, allowing them to enter cells efficiently and [[gene delivery|deliver genes]] to specific target areas, such as [[Neoplasm|tumor]] and [[Arthritis|arthritic tissues]].<ref>{{cite journal | vauthors = Bao CJ, Duan JL, Xie Y, Feng XP, Cui W, Chen SY, Li PS, Liu YX, Wang JL, Wang GL, Lu WL | display-authors = 6 | title = Bioorthogonal Engineered Virus-Like Nanoparticles for Efficient Gene Therapy | journal = Nano-Micro Letters | volume = 15 | issue = 1 | pages = 197 | date = August 2023 | pmid = 37572220 | pmc = 10423197 | doi = 10.1007/s40820-023-01153-y | bibcode = 2023NML....15..197B }}</ref>

[[RNA interference]]-based agents, such as [[zilebesiran]], contain [[Small interfering RNA|siRNA]] which binds with mRNA of the target cells, modifying gene expression.<ref>{{cite journal | vauthors = Desai AS, Webb DJ, Taubel J, Casey S, Cheng Y, Robbie GJ, Foster D, Huang SA, Rhyee S, Sweetser MT, Bakris GL | display-authors = 6 | title = Zilebesiran, an RNA Interference Therapeutic Agent for Hypertension | journal = The New England Journal of Medicine | volume = 389 | issue = 3 | pages = 228–238 | date = July 2023 | pmid = 37467498 | doi = 10.1056/NEJMoa2208391 | hdl = 20.500.11820/9ec1c393-058a-4fe7-8e8f-df207dcdfb85 | s2cid = 259995680 | url = https://openaccess.sgul.ac.uk/id/eprint/115616/1/nejmoa2208391.pdf | hdl-access = free }}</ref>