This comprehensive presentation explores oligonucleotide-based therapeutics, a revolutionary class of biopharmaceuticals that modulate gene expression to treat previously "undruggable" diseases. Key aspects covered include:
Mechanisms of Action:
Antisense oligonucleotides (ASOs) for mRNA degradation/splicing modulation
RNA interference (siRNA/miRNA) for gene silencing
Aptamers as "chemical antibodies" for targeted therapy
Clinical Applications:
Neurology: Nusinersen for spinal muscular atrophy
Metabolic Disorders: Mipomersen for hypercholesterolemia
Oncology: AS1411 aptamer in cancer trials
Ophthalmology: Macugen for macular degeneration
Technical Advancements:
Chemical modifications (2'-MOE, LNA, GalNAc conjugation)
Delivery systems overcoming cellular barriers
SELEX technology for aptamer development
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