{"schema_version":"1.0","canonical_url":"https://patentable.app/patents/US-9795687","patent":{"patent_number":"US-9795687","title":"Modularly assembled small molecules for the treatment of myotonic dystrophy type 1","assignee":null,"inventors":[],"filing_date":"2014-08-29T00:00:00.000Z","publication_date":"2017-10-24T00:00:00.000Z","cpc_codes":["G05B","G06F","G06F","G06F","H02J","H02J"],"num_claims":1,"abstract":"To study RNA function using small molecules, we designed bioactive, modularly assembled small molecules that target the noncoding expanded RNA repeat that causes myotonic dystrophy type 1 (Dm1), r(CUG)exp. Different modular assembly scaffolds were investigated including polyamines, alpha-peptides, beta-peptides, and peptide tertiary amides (PT As). Based on activity as assessed by improvement of DM1 -associated defects, stability against proteases, cellular permeability, and toxicity, we discovered that constrained backbones, namely PT As, are optimal."},"analysis":{"summary":null,"layman_explanation":null,"technical_analysis":null,"business_analysis":null,"faqs":null,"topics":[],"tech_cluster":null},"seo":{"title":"Modularly assembled small molecules for the treatment of myotonic dystrophy type 1","description":"To study RNA function using small molecules, we designed bioactive, modularly assembled small molecules that target the noncoding expanded RNA repeat that causes myotonic dystrophy type 1 (Dm1), r(CUG","keywords":[]},"attribution":{"source":"Patentable","source_url":"https://patentable.app","canonical_url":"https://patentable.app/patents/US-9795687","license":"CC-BY-4.0-like","license_terms":"AI-generated analysis on this page (summary, layman_explanation, technical_analysis, business_analysis, faqs) may be reused with attribution and a visible link back to the canonical URL above. Patent abstracts, claims, and bibliographic data are USPTO public domain.","required_link":"https://patentable.app/patents/US-9795687","citation_suggestion":"Patentable. \"Modularly assembled small molecules for the treatment of myotonic dystrophy type 1\" (US-9795687). https://patentable.app/patents/US-9795687","copyright_holder":"Nomic Interactive Technology LLC"},"links":{"html":"https://patentable.app/patents/US-9795687","json":"https://patentable.app/api/llm-context/US-9795687","site":"https://patentable.app","llms_txt":"https://patentable.app/llms.txt"},"generated_at":"2026-06-06T05:36:49.557Z"}