Troy Rohn and colleagues targeted 5HT-2A, a serotonin receptor known to play a role in anxiety and depression. The authors used a vector based on an inactivated adeno-associated virus to deliver the vector through the nose. The vector delivers a guide RNA to neurons. The guide RNA binds to the target receptor gene, HTR2A, which is then cut at a specific location by the enzyme Cas9. The work is published in the journal PNAS Nexus.
Five weeks after intranasal delivery of the vector and package, 75 mice were tested with standard behavioral assays measuring mouse anxiety. For example, anxious mice will choose to spend more time in dark areas and tend to bury unfamiliar objects such as marbles in sawdust rather than letting them be.
Treated mice spent 35.7% more time in light areas than controls and showed a 14.8% decrease in the number of marbles buried compared with controls. Mice treated with the CRISPR package showed an 8.47-fold decrease in HTR2A expression in their brains, compared with control mice. According to the authors, noninvasive, intranasal delivery of CRISPR/Cas9 therapeutics may help patients who exhibit treatment-resistant anxiety.
PackGene is a CRO & CDMO technology company that specializes in packaging recombinant adeno-associated virus (rAAV) vectors. Since its establishment in 2014, PackGene has been a leader in the AAV vector CRO service field, providing tens of thousands of custom batches of AAV samples to customers in over 20 countries. PackGene offers a one-stop CMC solution for the early development, pre-clinical development, clinical trials, and drug approval of rAAV vector drugs for cell and gene therapy (CGT) companies that is fast, cost-effective, high-quality, and scalable. Additionally, the company provides compliant services for the GMP-scale production of AAVs and plasmids for pharmaceutical companies, utilizing five technology platforms, including the π-Alpha™ 293 cell AAV high-yield platform and the π-Omega™ plasmid high-yield platform. PackGene’s mission is to make gene therapy affordable and accelerate the launch of innovative gene drugs. The company aims to simplify the challenging aspects of gene therapy development and industrialization processes and provide stable, efficient, and economical rAAV Fast Services to accelerate gene and cell therapy development efforts from discovery phase to commercialization.
Related News
Sangamo Therapeutics Secures Accelerated Approval Pathway for Gene Therapy in Fabry Disease
Sangamo Therapeutics has announced a major advancement in its gene therapy program for Fabry disease, as the U.S. FDA has provided a clear pathway for Accelerated Approval. This decision could potentially speed up approval timelines by three years, with a Biologics...
[2024/10/18] Gene and Cell Therapy- weekly digest from PackGene
FeaturedNewsArticlesPackGene's NewsletterReceive the latest news and insights to your inbox.About PackGenePackGene Biotech is a world-leading CRO and CDMO, excelling in AAV vectors, mRNA, plasmid DNA, and lentiviral vector solutions. Our comprehensive offerings span...
GSK sues Moderna over mRNA vaccine patents, seeks ‘reasonable royalty’
GSK on Tuesday unveiled a lawsuit filed against Moderna in Delaware federal court, alleging that its patented inventions provide the “foundation” for Moderna’s mRNA vaccine portfolio. GSK said it’s looking to recover “a reasonable royalty” for Moderna’s tens of...
Gene Therapy Automatically Converts Omega-6 to Omega-3 Fatty Acids in the Body
Shriners Children's Develops New Technology to Prevent Childhood Obesity ST. LOUIS, Oct. 16, 2024 /PRNewswire/ -- According to the Centers for Disease Control, nearly 20% of children and teens are considered obese. Research shows it can have a dramatic impact on a...
Related Services
AAV Packaging - NHP Grade
READ MORE
Off the Shelf AAV Products
We offer a wide catalogue of pre-made AAVs for a multitude of research needs.
READ MORE
AAV Capsid Engineering
Proven technology paving your path to effective therapies for cancer or genetic disorder
READ MORE