Here, SCGE researchers report that an LNP formulated with a herringbone mixer led to 2-fold more heart delivery than the same LNP formulated with a bifurcating mixer. These data suggest that it is possible to increase heart delivery via nanoparticle processing. (2/2)
pubs.acs.org/doi/10.1021/...

3)And more!

View the platform website at scge.mcw.edu/platform/home

#RareDisease #Regulatory #GeneTherapy #FreeResource (3/3)

2) View documents generated through regulatory interactions between the U.S. Food and Drug Administration (FDA) and SCGE consortium projects. (2/3)

scge.mcw.edu/platform/pub...

The Cas9-d system provides a versatile approach to adjust Cas9 levels, demonstrating its potential as an experimental tool for controlling genome editing outcomes in vitro and ex vivo. With further development, it holds promise for enhancing somatic cell genome editing in vivo. (2/2)

Meet the Expert is a webinar series held by the Somatic Cell Genome Editing (SCGE) consortium to connect with experts involved in different aspects of gene therapy development and regulatory approval. Find more Meet the Expert webinars at scge.mcw.edu/meet-the-exp... (3/3)

#GeneTherapy #Webinar

Andy Holt is the Chief Commercial Officer at Viralgen. Jacob Smith is the Head of Technical Development & CMC Strategy at Viralgen. (2/3)

Collectively, their results highlight inflammation-inducible IL-1-targeted therapy using an rAAV vector as a long-lasting, pathophysiologic treatment for chronic inflammatory diseases. (4/4)

#GeneTherapy #BiomedicalResearch #RheumatoidArthritis

The rheumatoid arthritis model mice showed a significant reduction in circulating immune cells, expression of the genes associated with inflammatory responses, joint swelling, and bone destruction. (3/4)

In this study, SCGE researchers develop a recombinant adeno-associated virus (rAAV)-based gene therapy to deliver an inflammation-inducible, secreted human IL-1 receptor antagonist (sIL-1Ra) as a complementary approach to existing IL-1 blockers. (2/4)

www.sciencedirect.com/science/arti...

These studies provide methods and reagents to precisely control the dosage and half-life of CRISPR-based technologies, propelling their therapeutic development. (5/5)

#CRISPR #biotech #GeneTherapy