Publications by authors named "Lawrence C T Tam"
Article Synopsis
- The challenge of intragenic heterogeneity in dominant disease-causing genes complicates the development of effective and affordable treatments, as seen in autosomal dominant retinitis pigmentosa caused by numerous rhodopsin mutations.
- An RNA interference (RNAi)-based method is proposed that can target the problematic native transcripts without affecting engineered replacement genes, suggesting a mutation-independent therapeutic strategy.
- Experiments show that this RNAi approach can reduce murine rhodopsin levels by up to 90% while still allowing the expression of the replacement genes, validating its effectiveness in cell cultures and live mouse models.
