Therapeutic targeting of RNA for neurological and neuromuscular disease.
Genes Dev
; 38(15-16): 698-717, 2024 Sep 19.
Article
en En
| MEDLINE
| ID: mdl-39142832
ABSTRACT
Neurological and neuromuscular diseases resulting from familial, sporadic, or de novo mutations have devasting personal, familial, and societal impacts. As the initial product of DNA transcription, RNA transcripts and their associated ribonucleoprotein complexes provide attractive targets for modulation by increasing wild-type or blocking mutant allele expression, thus relieving downstream pathological consequences. Therefore, it is unsurprising that many existing and under-development therapeutics have focused on targeting disease-associated RNA transcripts as a frontline drug strategy for these genetic disorders. This review focuses on the current range of RNA targeting modalities using examples of both dominant and recessive neurological and neuromuscular diseases.
Palabras clave
Texto completo:
1
Bases de datos:
MEDLINE
Asunto principal:
ARN
/
Enfermedades del Sistema Nervioso
/
Enfermedades Neuromusculares
Límite:
Animals
/
Humans
Idioma:
En
Revista:
Genes Dev
Asunto de la revista:
BIOLOGIA MOLECULAR
Año:
2024
Tipo del documento:
Article
País de afiliación:
Estados Unidos