Combining Engineered U1 snRNA and Antisense Oligonucleotides to Improve the Treatment of a BBS1 Splice Site Mutation

• Những tác giả chính: Saskia Breuel (Tác giả), Mariann Vorm (Tác giả), Anja U. Bräuer (Tác giả), Marta Owczarek-Lipska (Tác giả), John Neidhardt (Tác giả)
• Định dạng: Sách
• Được phát hành: Elsevier, 2019-12-01T00:00:00Z.
• Những chủ đề: article
• Truy cập trực tuyến: Connect to this object online.

Manipulation of pre-mRNA processing is a promising approach toward overcoming disease-causing mutations and treating human diseases. We show that a combined treatment applying two splice-manipulating technologies improves therapeutic efficacies to correct mutation-induced splice defects. Previously,...