Splicing Alteration TRIZ Cases
- Antisense Oligonucleotides Modulating LRP8 Splicing
Chemically modified antisense compounds increase exon 19 inclusion in LRP8 mRNA to restore signaling pathways disrupted in Alzheimer's Disease.
- Antisense Modulation of Aberrant Gene Expression in Precursor Cells
Antisense oligonucleotides normalize aberrant gene expression in precursor cells, correcting secondary defects that limit transplantation success.