Targeting the Brain with New MD Therapies
In people with DMD, part of the gene responsible for dystrophin production is missing, resulting in an inability to make a functional form of the protein. Researchers have discovered that introducing pieces of genetic material called oligonucleotides into affected cells causes the cells to skip over the damaged part of the gene and produce functional dystrophin. The introduction of oligonucleotides has proven effective at treating the symptoms of DMD.
One of the biggest challenges for scientists working on oligonucleotide therapies has been finding a way to get the therapeutic molecules into cells. One successful approach has been to create compounds of oligonucleotides bound together with amino acids. The amino acids act as transport vehicles that deliver the oligonucleotides inside the cells. These compounds are called enhanced delivery oligonucleotides (EDOs).
EDOs work well for getting oligonucleotides into muscle cells, but getting them into affected brain cells is more complicated. The brain has a very effective system for keeping foreign (and potentially harmful) molecules from entering the brain, a layer of cells called the blood-brain barrier. The barrier does an excellent job of repelling dangerous substances, but it also easily blocks molecules (such as EDOs) meant to treat brain diseases.
Fortunately, new research may have discovered a solution to the problem. Researchers have focused on a compound called a heteroduplex oligonucleotide (HDO). These compounds consist of oligonucleotides bound to a fatty molecule on one end. In animal tests, HDOs were better able to cross the blood-brain barrier than other kinds of therapeutic oligonucleotides. HDO research is in its early stages, but if it lives up to its promise, new treatments for the brain-related symptoms of muscular dystrophy could be on the horizon.
To learn more about our mission to find a cure for brain disorders worldwide, please visit our website at The United Brain Association. If you’d like to receive updates, news, scheduled events, or more information about our ongoing donor-funded research projects, sign up for our email newsletter by clicking here. Together we can find a cure for brain disorders.