Open AccessAntisense Oligonucleotide Rescue of Deep-Intronic Variants Activating Pseudoexons in the 6-Pyruvoyl-Tetrahydropterin Synthase Gene
Author(s) -
Ainhoa Martínez-Pizarro,
Fátima Leal,
Lise Lolle Holm,
Thomas Koed Doktor,
Ulrika Simone Spangsberg Petersen,
María Bueno,
Beat Thöny,
Belén Pérez,
Brage Storstein Andresen,
Lourdes R. Desviat
Publication year - 2022
Publication title -
nucleic acid therapeutics
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 2.255
H-Index - 67
eISSN - 2159-3345
pISSN - 2159-3337
DOI - 10.1089/nat.2021.0066
Subject(s) - exon , biology , splice , genetics , exon skipping , rna splicing , context (archaeology) , intron , gene , microbiology and biotechnology , oligonucleotide , alternative splicing , rna , paleontology