Delivery of Antisense Oligonucleotides to the Mouse Retina.
Antisense oligonucleotide
Drug delivery
Inherited retinal diseases
Intraocular injection
Intravitreal injection
Mouse
Retina
Journal
Methods in molecular biology (Clifton, N.J.)
ISSN: 1940-6029
Titre abrégé: Methods Mol Biol
Pays: United States
ID NLM: 9214969
Informations de publication
Date de publication:
2022
2022
Historique:
entrez:
25
2
2022
pubmed:
26
2
2022
medline:
3
3
2022
Statut:
ppublish
Résumé
The eye is the organ in charge of vision and, given its properties, has become an excellent organ to test genetic therapies, including antisense oligonucleotide (AON) technology. In fact, the first AON receiving FDA and EMA approval was meant to treat an eye condition. Currently, dozens of clinical trials are being conducted for a variety of subtypes of inherited retinal disease. Although most of them are based on gene augmentation therapies, a phase 3 and two phase 1/2 clinical trials using AONs are ongoing. Since the retina is a layered structure of nondividing cells, obtaining human retinal tissue and expanding it in the lab is not possible, unless induced pluripotent stem cell technology is used. Mouse models have helped to elucidate the function of many genes, and the retinal structure is quite similar to that of humans. Thus, drug delivery to the mouse eye can provide valuable information for further optimization of therapies. In this chapter, the protocol for intravitreal injections of AONs is described in detail.
Identifiants
pubmed: 35213028
doi: 10.1007/978-1-0716-2010-6_22
pmc: PMC9703207
doi:
Substances chimiques
Oligonucleotides
0
Oligonucleotides, Antisense
0
Types de publication
Journal Article
Research Support, Non-U.S. Gov't
Langues
eng
Sous-ensembles de citation
IM
Pagination
321-332Informations de copyright
© 2022. The Author(s).
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