Let’s talk about Virus Like Particles… Here, I present part 3 from my video series on gene editing delivery.
Friends and Family!
How are we doing?
Summer is upon us in San Diego. The water is warming up, the air is warming up… and on the not so great side we have a great white shark nursery that’s up and running near Torrey Pines. We have seen a few recent shark attacks on swimmers. My recommendation for local swimmers is to find a spot that is at least 2-3 miles away from Torrey Pines.
Also… apologies to everyone that opened up an email I sent out a few weeks ago. It was just some random notes – the email should not have gone out. I will fire my marketing manager immediately… Oh wait, no that’s me, I’m the marketing manager…I guess I’m fired…
Alright, we’ve got all that out of the way.
In this newsletter, I am presenting a new video on the delivery of gene editing tools (i.e. Crispr/Cas9). This is the 3rd video in a series describing the challenge of delivering gene editing tools into the correct tissue in the human body.
Below, I summarize the use of VLPs (virus like particles) and then you can click into the YouTube video, and watch me discuss this topic.
VLPs for gene editing delivery
In order to advance genome editing therapies beyond blood or liver diseases, new technologies for targeted delivery are necessary.
The ideal gene editing delivery technology needs to be:
highly cell-type-specific
transient for controlled editing
in vivo administration
Viral vectors like AAV and LV are being tested for their ability to deliver gene editing cargo. However, there are concerns with current viral vectors.
Immunogenicity against the viral vector (AAV, ~10% of population have nAbs)
Not a transient approach. Long-term expression of CRISPR-Cas nucleases from integrated lentiviral or episomal AAV transgenes increases the risk of unintended off-target edits.
Virus Like Particles (VLPs) are just what they sound like… they behave similar to a viral vector but they are inherently safer as they don’t deliver a Cas9 enzyme into a cell’s genome.
VLPs can bind to a target cell and deliver RNA and proteins into those cells.
Think of a VLP like a Lipid Nanoparticle that relies on the surface structure of a viral vector for cell binding and transduction.
In terms of gene editing, guide RNA can be complexed with protein Cas9 and manufactured into a VLP. VLPs with pre-complexed guide RNA and Cas9 are currently being tested in animal models.
VLP features
VLPs promise the cell specific binding of lentiviral vectors, but without integration risks seen in LV or the cargo limiting AAV.
VLPs can be pseudotyped with different glycoproteins, enabling specific targeting of cell types of interest.
The future of VLP research…
In order for VLPs to target a range of cells in the human body for gene editing, we will need to compliment the natural glycoproteins, possibly with cell specific antibodies. Various lab are tweaking with linkers and glycoproteins to optimize editing efficiency and cell tropism.
Click below to watch a You Tube video on this subject…. and help me out and like, share and subscribe. I’ve been getting great feedback on these videos. As long as they are helpful, I will keep putting them together.
And….if you want to watch my whole series on gene editing delivery… Below you can see the first and second videos in this series.
Share these with any biotech students or entrepreneurs in your life!
We need the next generation of scientists to figure all this out! There are about 7000 genetic disorders remaining…