1.
Indian Pediatr
;
2020 Feb; 57(2): 165-171
Artigo
| IMSEAR
| ID: sea-199481
RESUMO
Synthetic Antisense oligonucleotides (ASOs) are novel and efficient laboratory tools to regulate the expression of specific genes, andhave only recently come into clinical use. These are synthetic single-stranded DNA analogs, whose sequence is complementary to atarget nucleotide and alter protein synthesis by several mechanisms. We herein provide a primer on the topic for pediatricians, as thisgroup of drugs is likely to see many more drugs for previously incurable diseases.