Antisense Oligonucleotides (ASO)

Describes Antisense Oligonucleotide (ASO) technology and how it can be used to treat a variety of genetic disorders with no previous effective conventional therapy. Spinal Muscular Atrophy (SMA) and Lipoprotein (a) or Lp(a) are presented as examples.

[@WmLatin]

Very clear explanations- even for an old-non-biologist !

[@mariebarker4499]

Thank you!

[@hirosewill893]

Fantastic!

[@kimboxdorfer7010]

Would this work on familial CJD with the E200K-129M mutation?

[@dailydoseofmedicinee]

💯