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
Exploring Tau Protein’s Role in Glaucoma: New Insights and Therapeutic Potential
Glaucoma, a chronic neurodegenerative disorder, leads to irreversible vision loss by damaging retinal ganglion cells (RGCs) and the optic nerve, often associated with increased intraocular pressure (IOP). Despite the benefits of IOP-lowering treatments, the underlying...
FDA-mandated CAR-T monitoring period could be halved, say researchers
In patients with diffuse large B-cell non-Hodgkin lymphoma (DLBCL), the two hallmark post-chimeric antigen receptor (CAR)-T therapy toxicities are extremely rare after two weeks, supporting a shorter, more flexible toxicity monitoring period, according to a study...
Ancestral CRISPR-Cas13 Ribonucleases Discovered: Implications for Genome Editing
In a pioneering study published in *Science*, a team of researchers led by Peter H. Yoon and Jennifer A. Doudna from the University of California, Berkeley, has made a remarkable discovery in the realm of CRISPR technology. The team has identified an ancestral clade...
KBI Biopharma Expands Manufacturing Contract with Global Pharmaceutical Company
KBI Biopharma Inc., a JSR Life Sciences company and global cGMP contract development and manufacturing organization (CDMO), has extended and expanded its manufacturing contract with a leading global pharmaceutical company. Originally initiated in 2020, the renewed...
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
