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Development and Clinical Applications of Antisense Oligonucleotide Gapmers
Leanna Chan1,2, Toshifumi Yokota3,4,5
1Department of Medical Genetics, Faculty of Medicine and Dentistry, University of Alberta, Edmonton, AB, Canada.
Methods in Molecular Biology (Clifton, N.J.)
|September 1, 2020
Summary
Antisense oligonucleotides, a type of DNA-like molecule, regulate gene expression for genetic disease treatment. Gapmer chemistry, a specific type, shows promise in treating conditions like familial hypercholesterolemia and hereditary transthyretin amyloidosis.
Area of Science:
- Molecular Biology
- Genetics
- Pharmacology
Background:
- Antisense oligonucleotides (ASOs) are DNA-like molecules that regulate gene expression.
- They are crucial for suppressing mutated genes that disrupt essential cellular pathways.
- Advancements in ASO chemistry aim to enhance therapeutic efficacy, potency, and safety.
Observation:
- Chimeric ASO structures, known as gapmers, combine deoxyribonucleotides with modified nucleotides.
- Gapmers have demonstrated significant potential in treating various genetic disorders.
- Approved drugs like mipomersen and inotersen exemplify successful gapmer applications.
Findings:
- Mipomersen and inotersen are FDA-approved for familial hypercholesterolemia and hereditary transthyretin amyloidosis.
- Volanesorsen received conditional EU approval for familial chylomicronemia syndrome.
- Numerous other gapmer therapies are undergoing clinical trials and preclinical development.
Implications:
- Gapmer technology represents a significant advancement in treating genetic diseases.
- Ongoing research explores gapmer applications in diverse conditions including cancer and neurological disorders.
- The development of novel ASO chemistries like gapmers expands therapeutic options for unmet medical needs.
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