2 weeks ago
DNA damage may drive Huntington's disease, opening new treatment path
Huntington's disease is a very serious illness that a person is born with, and it slowly harms the brain, making it harder to move, think, and feel normally.
Scientists from a place called the Lawrence Berkeley National Laboratory studied this disease in mice.
They discovered that the sick brain cells have lots of tiny breaks in their DNA, which is like the instruction book for our bodies.
They found these DNA breaks happen before the disease even causes symptoms.
The scientists used a special medicine-like compound that acts like a shield against damage, and it fixed the DNA breaks and protected the brain cells.
The mice got better even though the gene that causes the disease was still there.
This is exciting because there is no cure for Huntington's yet, and current treatments only try to fix the gene itself.
The scientists say there are already similar compounds made for humans, so new treatments might be tested more quickly.
A study in Nature Communications found that increased breaks across DNA strands in the genome drive neurodegeneration in Huntington's disease.
Researchers at Lawrence Berkeley National Laboratory (Berkeley Lab) found DNA damage occurs before the onset of Huntington's symptoms.
An investigational antioxidant compound reversed the DNA damage and protected against neurodegeneration in mice without altering the mutated gene.
The finding follows nearly 30 years of research focused mainly on fixing the genetic mutation behind the disease.
Researchers say clinical agents that change DNA breaks already exist for humans, but the same disease mechanism must first be confirmed in human patients.
- Who
- Researchers Aris Polyzos and Cynthia McMurray, along with colleagues at Lawrence Berkeley National Laboratory's Biosciences Area, led the study.
- What
- The study identified DNA damage as a driver of neurodegeneration in Huntington's disease and showed an investigational antioxidant compound can reverse it in a mouse model.
- Where
- Lawrence Berkeley National Laboratory in the United States.
- When
- The article does not specify a date; the study was published in Nature Communications.
- Why
- Huntington's disease has no cure and only limited experimental treatments, so researchers are seeking new approaches that use existing compounds to target DNA damage.
Key facts
- Disease studied
- Huntington's disease (HD)
- Study publication
- Nature Communications
- Research institution
- Lawrence Berkeley National Laboratory
- Lead researchers
- Aris Polyzos and Cynthia McMurray
- Key finding
- Marked increase in breaks across DNA strands throughout the genome drives neurodegeneration
- Treatment tested
- Investigational antioxidant compound (in a mouse model)
- Treatment outcome
- Reversed DNA damage and protected neurons without altering or blocking the gene
- Next step
- Confirm the same disease mechanism occurs in human patients
Quotes
Cynthia McMurray
Senior lead and retiree affiliate in Berkeley Lab’s Biosciences Area
“We show that symptoms are preceded by DNA damage, and that this can be reversed using an investigational antioxidant compound, which also protects against neurodegeneration.”
theprint.in
“I believe we’re opening the door to a new way to treat Huntington’s patients. Clinical agents already exist for humans that are known to change these breaks.”
theprint.in
Sources
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