Modified Oligonucleotides Targeting miR-17 for Cyst Growth Control
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Solution Overview
Problem
Polycystic kidney disease is characterized by the accumulation of fluid-filled cysts that compress surrounding tissue, leading to a decline in kidney function and eventual end-stage renal disease, with no effective treatments to halt cyst growth or improve kidney function.
Innovation Solution
A modified oligonucleotide with a specific nucleoside pattern and nucleobase sequence is administered to inhibit the activity of the miR-17 family, thereby reducing cyst growth and improving kidney function in subjects with polycystic kidney disease.
Engineering Contradictions & Design Principles
Engineering Contradiction Analysis
1Reliability
If conventional treatments are used for polycystic kidney disease, then current standard of care is maintained, but cyst growth continues and kidney function declines
Solution Approach 1:
The patent employs antisense oligonucleotides that change the molecular parameters of miR-17 family members through complementary base pairing, preventing their binding to target mRNAs. This molecular-level parameter change inhibits cyst growth while preserving kidney function, resolving the contradiction between maintaining current care standards and preventing disease progression.
Solution Approach 2:
The antisense oligonucleotides act as intermediary molecules that bind to miR-17 family members, preventing them from exerting their harmful effects on kidney tissue. These oligonucleotide intermediaries specifically target the pathological process without disrupting normal kidney function, thereby addressing both aspects of the contradiction.
2Reliability
If no effective treatment is available, then disease progression is unmanaged, but introducing new therapies carries risks and complexity
Solution Approach 1:
The patent replaces mechanical/surgical interventions with a molecular-level therapeutic approach using antisense oligonucleotides. This substitution delivers precise disease progression control through biochemical mechanisms rather than complex surgical procedures, reducing treatment complexity while improving reliability of disease management.
Solution Approach 2:
The antisense oligonucleotides are designed to preemptively block miR-17 family member activity before it can cause significant cyst growth and kidney damage. This preliminary molecular action prevents disease progression at an early stage, avoiding the need for more complex interventions later.
Data Source
AI summary
Provided herein are methods for the treatment of polycystic kidney disease, including autosomal dominant polycystic kidney disease, using modified oligonucleotides targeted to miR-17.