What are antisense therapies?

Antisense therapies (also called ASOs, antisense oligonucleotides) are short, specially designed genetic molecules that can influence how a gene is read inside cells. For LAMA2-RD, they are being explored as a potential way to increase production of functional laminin alpha-2 in patients with partial deficiency.

Why is there currently no cure for LAMA2-RD? What research studies are currently underway? How long does it take to develop a new treatment? What are the stages of drug development? What is a natural history study? Why are natural history studies important? What is a patient registry? Why should I participate in a patient registry? Which universities and institutions are researching LAMA2-RD? Which companies are developing therapies? What are the biggest scientific challenges? Which treatment approaches are being investigated? What is gene therapy? What is CRISPR gene editing? What are stem cell therapies?

What are antisense therapies?

Related questions

The driving forces behind Lama2.com