Investigating gene therapy techniques aimed at correcting Alzheimer’s mutations

Investigating gene therapy techniques aimed at correcting Alzheimer’s mutations

### Investigating Gene Therapy Techniques for Alzheimer’s

Alzheimer’s disease is a complex condition that affects millions of people worldwide. While current treatments focus on managing symptoms, researchers are exploring new ways to prevent the disease by targeting its root causes at the genetic level. One promising approach is gene therapy, which aims to correct the genetic mutations that contribute to Alzheimer’s.

#### What is Gene Therapy?

Gene therapy involves making changes to the DNA in our cells to prevent or treat diseases. This can be done by replacing harmful genes, silencing certain genes, or introducing new genetic material that has a positive effect. For Alzheimer’s, scientists are looking at how genes play a role in the development of the disease.

#### The Role of Genes in Alzheimer’s

Alzheimer’s is a multifactorial disease, but genes are a significant part of the puzzle. One of the strongest genetic risk factors is the APOE gene. People inherit one version of this gene from each parent, and some versions, like APOE4, increase the risk of developing Alzheimer’s. On the other hand, the APOE2 version seems to protect against the disease.

#### Current Research

Several gene therapies are currently in clinical trials for Alzheimer’s. These trials aim to prevent the abnormal proteins that build up in the brain, which is a hallmark of the disease. For example, a recent trial by Eli Lilly is investigating an investigational monoclonal antibody called remternetug. This trial involves young adults from families with a history of Alzheimer’s mutations, as they are at a higher risk of developing the disease.

Another trial at Washington University School of Medicine is also focusing on preventing Alzheimer’s in young adults with a family history of the disease. This trial aims to determine if remternetug can remove or prevent amyloid plaques from building up in the brain, which is a key factor in the progression of Alzheimer’s.

#### How Gene Therapy Works

Gene therapy for Alzheimer’s works by targeting specific genes that contribute to the disease. For instance, scientists might use gene editing techniques to replace the harmful APOE4 gene with a protective version like APOE2. Alternatively, they might introduce new genetic material that helps to clear out amyloid plaques or reduce inflammation in the brain.

#### Future Prospects

While gene therapy is still in its early stages for Alzheimer’s, the potential is vast. If successful, these treatments could prevent the onset of the disease or slow its progression significantly. This could mean a better quality of life for millions of people and a substantial reduction in the economic burden of Alzheimer’s on society.

In summary, gene therapy offers a promising avenue for preventing and treating Alzheimer’s by addressing the genetic mutations that contribute to the disease. Ongoing research and clinical trials are crucial steps towards making this therapy a reality, potentially offering new hope for those affected by Alzheimer’s.