AUG 25, 2021 8:27 AM PDT

A Possible Treatment Strategy for Familial Alzheimer's is ID'ed

WRITTEN BY: Carmen Leitch

Some cases of Alzheimers disease (AD) are known as Familial Alzheimer's disease because they are due to genetic mutations. So far, mutations in three genes, APP,  PSEN1, and PSEN2 have been associated with familial AD. The disease burden is high in China, where an estimated 500,000 people have the disorder. An international team of researchers has now created a treatment approach that uses gene-editing in the brain to potentially reduce the symptoms of familial AD. This work, which was reported in Nature Biomedical Engineering, used a mouse model. The treatment strategy still has to be tested in humans.

Image credit: Pixnio

Many diseases that trace back to genetic mutations might be treatable if the genomes of affected cells could be edited to carry the correct version of whatever gene is mutated. While there are now reliable gene-editing tools, it's still a challenge to reach the right cells in time to prevent or treat disease. Gene-editing technology has worked in the human eye, for example, an organ that is easy to access. But the brain is a particularly sensitive and protected part of the body.

In this study, the researchers utilized a system that can cross the blood-brain barrier to make edits in cells throughout the brain. They modified a non-pathogenic viral delivery system known as an adeno-associated virus to ferry CRISPR-Cas9 components across the blood brain barrier. One intravenous administration was able to achieve that result in an animal model. Genetic mutations associated with familial AD could be corrected, and symptoms of the disorder were relieved.

In mouse models, the levels of a protein called amyloid that's been associated with AD-linked neurodegeneration stayed low for six months after they'd been treated; the gene editing technique appeared to have lasting impacts in an animal with a typical lifespan of about two years. The researchers did not detect any side effects in these animals either.

“As the first demonstration of efficient brain-wide genome editing to alleviate Alzheimer’s disease pathology throughout the whole brain, this is really an exciting development,” said study leader Professor Nancy Ip, Vice-President for Research and Development at HKUST. “Our work is an important milestone for the use of genome editing in treating hereditary brain diseases, and contributes to the development of precision medicine for inherited forms of neurodegenerative diseases.”

More research will be needed to demonstrate that this is a safe and effective treatment for people.

Sources: Hong Kong University of Science and Technology (HKUST), Nature Biomedical Engineering

About the Author
  • Experienced research scientist and technical expert with authorships on over 30 peer-reviewed publications, traveler to over 70 countries, published photographer and internationally-exhibited painter, volunteer trained in disaster-response, CPR and DV counseling.
You May Also Like
OCT 01, 2021
Genetics & Genomics
Making the Most of Your NGS Data: Understanding Metrics for Target-enriched NGS
OCT 01, 2021
Making the Most of Your NGS Data: Understanding Metrics for Target-enriched NGS
Introduction Targeted next-generation sequencing (NGS) is often performed using hybridization-based target enrichment, w ...
NOV 03, 2021
Genetics & Genomics
Gene Therapy Could Make Parkinson's Drug More Effective
NOV 03, 2021
Gene Therapy Could Make Parkinson's Drug More Effective
In Parkinson's disease, neurons that produce the neurotransmitter dopamine in a part of the brain called the substantial ...
NOV 11, 2021
Health & Medicine
The Taconic-Cyagen Academic Model Generation Alliance
NOV 11, 2021
The Taconic-Cyagen Academic Model Generation Alliance
With more than 15 years of experience in genetic engineering, Cyagen utilizes a highly efficient process, including prop ...
NOV 08, 2021
Genetics & Genomics
Revealing Gene Expression Networks in Human Embryonic Stem Cells
NOV 08, 2021
Revealing Gene Expression Networks in Human Embryonic Stem Cells
Advances in genetic and computational tools have enabled researchers to study complex networks of gene expression in ind ...
NOV 15, 2021
Microbiology
Is the Gut Microbiome - Autism Link Due to Diet?
NOV 15, 2021
Is the Gut Microbiome - Autism Link Due to Diet?
Research has shown that the gut microbiome, which consists of trillions of bacteria, fungi, viruses, and other microorga ...
DEC 05, 2021
Coronavirus
Researcher Who Alerted Us to Omicron Encourages Collaboration
DEC 05, 2021
Researcher Who Alerted Us to Omicron Encourages Collaboration
Many researchers have been working diligently to track the evolution of the pandemic virus SARS-CoV-2, which causes the ...
Loading Comments...