Research into Treatments

There are currently no clinical treatments for bestrophinopathies. However, researchers are currently developing new approaches, such as gene therapy and genome editing to treat these inherited diseases.

Image showing the addition of a functioning gene into a defective cell.

Gene Therapy

Gene therapy supplies healthy (non-mutated) copies of the gene into a cell. Find out how this is done and how it works.

Image showing scissors cutting DNA to remove a mutation and replace with the correct DNA

Gene Editing

This is a method that allows researchers to make specific changes to the DNA of living cells, modifying, removing or adding to the DNA sequence to correct the defect. Find out how we do this and how it works.