18307559. TREATMENT OF POLYCYSTIC DISEASES WITH AN HDAC6 INHIBITOR simplified abstract (Mayo Foundation for Medical Education and Research)

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TREATMENT OF POLYCYSTIC DISEASES WITH AN HDAC6 INHIBITOR

Organization Name

Mayo Foundation for Medical Education and Research

Inventor(s)

Sergio A. Gradilone of Rochester MN (US)

Nicholas F. Larusso of Rochester MN (US)

TREATMENT OF POLYCYSTIC DISEASES WITH AN HDAC6 INHIBITOR - A simplified explanation of the abstract

This abstract first appeared for US patent application 18307559 titled 'TREATMENT OF POLYCYSTIC DISEASES WITH AN HDAC6 INHIBITOR

Simplified Explanation

The abstract describes the use of an HDAC6-specific inhibitor to reduce the pathogenesis associated with polycystic liver disease. This inhibitor has shown to attenuate symptoms such as cyst formation, cyst growth, and cholangiocyte proliferation. Additionally, treatment with the inhibitor increased the acetylation of tubulin and phosphorylation/acetylation of β-catenin while reducing the synthesis of β-catenin in bile ducts.

  • HDAC6-specific inhibitor shown to reduce symptoms of polycystic liver disease
  • Inhibitor decreases cyst formation, growth, and cholangiocyte proliferation
  • Treatment increases acetylation of tubulin and phosphorylation/acetylation of β-catenin
  • Reduces synthesis of β-catenin in bile ducts

Potential Applications

The technology could potentially be used in the treatment of polycystic liver disease and other related conditions.

Problems Solved

This technology addresses the pathogenesis associated with polycystic liver disease by reducing cyst formation, growth, and cholangiocyte proliferation.

Benefits

The use of an HDAC6-specific inhibitor offers a potential therapeutic approach to managing polycystic liver disease and improving patient outcomes.

Potential Commercial Applications

The technology could be developed into a pharmaceutical product for the treatment of polycystic liver disease, providing a new option for patients.

Possible Prior Art

Prior research may have explored the role of HDAC6 inhibitors in other disease conditions, but specific applications for polycystic liver disease may be novel.

Unanswered Questions

How does the HDAC6-specific inhibitor affect other organs or systems in the body?

The abstract focuses on the impact of the inhibitor on polycystic liver disease, but it does not mention potential effects on other organs or systems.

What are the potential side effects or limitations of using an HDAC6-specific inhibitor for the treatment of polycystic liver disease?

The abstract does not provide information on any potential side effects or limitations associated with the use of the inhibitor in treating polycystic liver disease.


Original Abstract Submitted

An HDAC6-specific inhibitor (i.e., a compound of Formula I or II) is shown to reduce the pathogenesis associated with polycystic disease. Administration of an HDAC6-specific inhibitor attenuated many of the symptoms characteristic of polycystic liver disease including cyst formation, cyst growth and cholangiocyte proliferation. Treatment with a HDAC6-specific inhibitor also increased the amount of bile duct acetylated tubulin and β-catenin phosphorylation and/or acetylation while reducing bile duct β-catenin synthesis. These results demonstrate that HDAC6 is overexpressed in cystic cholangiocytes and that its pharmacological inhibition reduces cholangiocyte proliferation and cyst growth.