Gapmers: Invention and Early History. (PubMed, Methods Mol Biol) - Oct 1, 2026 - "Gapmers have also successfully entered clinical trials for various genetic disorders, with two approved by the US Food and Drug Administration for the treatment of familial hypercholesterolemia and transthyretin amyloidosis-associated polyneuropathy, and one approved by the European Commission for the treatment of familial chylomicronemia syndrome. Here, we review the events surrounding the early development of gapmers, from conception to their maturity, and briefly conclude with perspectives on their use in therapy."
Journal • Review • Amyloidosis • Cardiac Amyloidosis • Dyslipidemia • Familial Chylomicronemia Syndrome • Familial Hypercholesterolemia • Genetic Disorders • Metabolic Disorders • Pain
|
|
Kenji Rowel Q Lim; Toshifumi Yokota
|