Preclinical development of antisense conjugates for the treatment of myotonic dystrophy 1

Thesis

Myotonic dystrophy type 1 (DM1) is a complex autosomal dominant neuromuscular disorder that affects approximately 1 in 8,000 individuals. DM1 impacts multiple organ systems, including skeletal muscle, heart and central nervous system (CNS), leading to a diverse range of symptoms. Despite ongoing research efforts, no curative treatments are currently approved and readily available to patients. Antisense oligonucleotide (ASO) based therapies are some of the most advanced treatments being tes...

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Authors: Stoodley, J

• DOI: 10.5287/ora-bmqk6ymgy
• Type of award: DPhil
• Level of award: Doctoral
• Awarding institution: University of Oxford
• Language: English
• Keywords: antibody drug conjugates; antisense oligonucleotides (ASOS); myotonic dystrophy 1 (DM1); preclinical development; heart drug delivery; skeletal muscle drug delivery; transferrin receptor 1 (TFR1); cell penetrating peptides; neuromuscular disease; antisense oligonucleotide conjugates; central nervous system drug delivery
• Subjects: Medical sciences