Project information

Zebrafish Screen for drugs to treat Lowe Syndrome

The idea is to Screen for drugs on Zebrafish deficient in OCRL1 which exhibit several characteristic features of Lowe Syndrome to better understand how these defects seen in Lowe Syndrome (LS) are brought about.

36 months

Charity information: The Lowe Syndrome Trust

The Lowe Syndrome Trust logo
  • Need

    Need

    The current problem is the lack of understanding of how the eye, kidney and neurological defects seen in Lowe Syndrome (LS) are brought about, and thus lack of effective treatments or preventative measures available.

    Solution

    To better understand how these defects seen in Lowe Syndrome (LS) are brought about it is important we have an animal model that is amenable to experimentation. Zebrafish embryos are an excellent model for drug screening, as Zebrafish deficient in OCRL1 exhibit several characteristic features of LS. Thus it will allow us to screen for drugs that will correct the symptoms of LS. in Lowe Syndrome disease (LS).

  • Aims

    Aim 1

    To find Successful FDA Approved compounds for treating Zebrafish

    Activities

    » reat Zebrafish with Compounds from Enzo life sciences and GlaxoSmithKline kinase inhibitor libraries.

    Success will be...Determined by Lowe syndrome Researchers


  • Impact

    Impact

    This assay will therefore serve as an important resource for the future development of drugs for Lowe Syndrome patients, including drugs that could help with seizures, cystic brain legions and defective kidney functions. Successful hits in Zebrafish is the first step to demonstrating their success, as Zebrafish deficient in OCRL1 exhibit several characteristic features of Lowe Syndrome.

    Risk

    None Reported

    Reporting

    Researchers will regularly update the Lowe Syndrome Trust which will report to donors via regular emails.

  • Budget

    Budget - Project Cost: £120,000

    Loading graph....
      Amount Heading Description
      £60,000 Salary Phd Student
      £60,000 Consumables Reagents and small equipment
  • Background

    Location

    Manchester University Laboratories

    Beneficiaries

    Lowe Syndrome Patients, Medical Researchers, Health Professionals

  • Why Us?

    Why Us?

    The Lowe Trust is the only UK based charity that offers funding into medical research investigating Lowe Syndrome, education and support to the families and sufferers of the disease. It also is the main funder of the research into this disease worldwide.

    Read more about the Charity running this project.

    People

    Dr Martin Lowe

    Supervisor