Therapeutic applications of trans-splicing.
Br Med Bull
; 136(1): 4-20, 2020 12 15.
Article
en En
| MEDLINE
| ID: mdl-33010155
ABSTRACT
BACKGROUND:
RNA trans-splicing joins exons from different pre-mRNA transcripts to generate a chimeric product. Trans-splicing can also occur at the protein level, with split inteins mediating the ligation of separate gene products to generate a mature protein. SOURCES OF DATA Comprehensive literature search of published research papers and reviews using Pubmed. AREAS OF AGREEMENT Trans-splicing techniques have been used to target a wide range of diseases in both in vitro and in vivo models, resulting in RNA, protein and functional correction. AREAS OF CONTROVERSY Off-target effects can lead to therapeutically undesirable consequences. In vivo efficacy is typically low, and delivery issues remain a challenge. GROWING POINTS Trans-splicing provides a promising avenue for developing novel therapeutic approaches. However, much more research needs to be done before developing towards preclinical studies. AREAS TIMELY FOR DEVELOPING RESEARCH Increasing trans-splicing efficacy and specificity by rational design, screening and competitive inhibition of endogenous cis-splicing.Palabras clave
Texto completo:
1
Colección:
01-internacional
Base de datos:
MEDLINE
Asunto principal:
Trans-Empalme
/
Inteínas
Límite:
Humans
Idioma:
En
Revista:
Br Med Bull
Año:
2020
Tipo del documento:
Article
País de afiliación:
Reino Unido