Lipid Replacement Therapy: Dr. Catherine Clarke (UCLA)
Barth syndrome is uniquely associated with a deficiency and alterations in cardiolipin, a phospholipid that is an important component of the inner mitochondrial membrane. This deficiency and alterations in cardiolipin result in damage to the cell. Providing a “disease-resistant” cardiolipin to increase and replace the usual molecules may be therapeutic for individuals with Barth syndrome.
To test this hypothesis, Dr. Catherine Clarke of UCLA will apply this unique therapeutic idea by using chemically modified lipids to reduce the amount of cellular damage produced by deficient and altered cardiolipin.
If this novel specific lipid-replacement therapy preserves mitochondrial function, it could protect cells against the oxidative stress conditions known to exist in people with Barth syndrome.
Learn more about the Clarke lab.
The cardiolipin molecule which has four fatty acid chains is altered in Barth syndrome