The patient, KJ, reaching out after infusion of the CRISPR therapy, with a big smile! Photo credit Children's Hospital of Philadelphia

In a medical breakthrough, a team including IGI’s
@urnov.bsky.social & @giannikopoulosp.bsky.social created an on-demand #CRISPR therapy for an infant with a deadly gene mutation — developed, approved, and delivered to the patient in just 6 months.

Read more: ow.ly/G0Bg50VTonC

#RareDisease 🧬

A promising genetic treatment tailor-made for a baby born with a rare disorder For the first time, doctors have created a customized treatment using the revolutionary gene-editing technique known as CRISPR to treat a baby with a rare, life-threatening genetic disorder.

For the first time, doctors have created a customized treatment using the revolutionary gene-editing technique known as CRISPR to treat a baby with a rare, life-threatening genetic disorder.

I am presenting today at @asgct.bsky.social conference #ASGCT2025 on "Electroporation-free delivery of CRISPR enzymes for efficient multiplex genome editing of primary human immune cells and repair of patient mutations in the rare disease HLH".

If you are attending, stop by poster #779 tonight!

This was a beautifully put together talk, a recap of history in the making. I am so glad to have been in attendance to see this one in person.
Thanks to @isctglobal.bsky.social #isct2025

I am excited to share that I will be participating in ScaleReady’s breakfast panel session tomorrow, May 7, at the International Society for Cell & Gene Therapy conference.

Check out our new paper, out now in The CRISPR journal!

This paper is incredibly detailed and should provide the information you need to get PERC working well for RNP delivery ex vivo using Cas9 or Cas12a. The big idea is to omit the electroporator and instead mix your RNP enzyme with an inexpensive and easy-to-use peptide.

We also built an FAQ page with some complementary resources, including recommendations on sourcing protein & peptide. We will continue updating this page with new information. Check it out!
www.rosswilsonlab.org/perc

Peptide-enabled ribonucleoprotein delivery for CRISPR engineering (PERC) in primary human immune cells and hematopoietic stem cells - Nature Protocols Peptide-enabled ribonucleoprotein delivery for CRISPR engineering (PERC) uses an amphiphilic peptide reagent to mediate intracellular delivery of CRISPR enzymes for high-efficiency editing of stimulat...

I'm proud to share our protocol on CRISPR enzyme delivery in primary human cells using PERC, a non-viral & hardware-independent technology. We previously described PERC in T cells, and here we extend its use to hematopoietic stem/progenitor cells (HSPCs).
www.nature.com/articles/s41...

Happy to share my new (first author) protocol paper, out now in Nature Protocols @natprot.bsky.social detailing an elaborate step-by-step of how to use PERC to deliver CRISPR RNPs in immune cells for ex vivo genome editing.
@rosswilsonlab.org

Credit goes to everyone who contributed: Srishti Sahu, Lorena de Oñate, Bruno Solano, and especially lead author Christy George, who worked tirelessly on this review.

Ex vivo therapies have predominated so far because they sidestep the substantial challenges of in vivo delivery. As potent as cell therapies can be, transplant can limit access and delay treatment. Fortunately, off-the-shelf T cell therapies and in vivo delivery are poised to change this landscape.

Therapeutic genome editing of hematopoietic stem cells (HSCs) and T cells has been getting better and better over the last ~15 years, with progress greatly accelerated by the advent of CRISPR. Here's our timeline figure in presentation-friendly "landscape" format (an online exclusive 🤗)

Genome Editing Therapy for the Blood: Ex Vivo Success and In Vivo Prospects | The CRISPR Journal Hematopoietic stem cells (HSCs) provide the body with a continuous supply of healthy, functional blood cells. In patients with hematopoietic malignancies, immunodeficiencies, lysosomal storage disorde...

I'm proud to share our review on CRISPR therapies for the blood. It has been about a year since Casgevy was approved for treatment of sickle cell disease, and edited CAR-T cells are showing more and more promise. Key figures shared below; paper is open access here: www.liebertpub.com/doi/full/10....