Antisense oligonucleotide (AON) therapy for Stargardt disease type 1 (STGD1) provides a proof of concept with effective restoration of correct ABCA4 transcript splicing.
Researchers at ProQR Therapeutics, an RNA company based in Leiden, the Netherlands, and with researchers at Radboud University Medical Center in Nijmegen, have reported results… Read More »Antisense oligonucleotide (AON) therapy for Stargardt disease type 1 (STGD1) provides a proof of concept with effective restoration of correct ABCA4 transcript splicing.