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Personalized gene-editing

KJ Muldoon (infant) is the first person in the world who received  in vivo gene-editing therapy making medical history in 2025. It was made at Children’s Hospital of Philadelphia/Penn Medicine. 

KJ was born with a rare metabolic disorder known as severe carbamoyl phosphate synthetase 1 (CPS1) deficiency, a life-threatening metabolic disorder that impairs the body’s ability to clear ammonia. 

Genetic Analysis:

Genetic analysis revealed that KJ inherited two distinct truncating (nonsense premature stop) variants in the CPS1 gene: Q335X (it means Glutamine into Stop codon at the site of CDS 335) and E714X (glutamic acid into stop codon at CDS 714). If you want to see the variant details about this mutation, click here:

Gene-Editing Treatment: 

A team led by Drs. Rebecca Ahrens-Nicklas and Kiran Musunuru developed a therapy delivered via lipid nanoparticles to the liver that uses a base-editing CRISPR. The first dose was administered in February 2025 (at 6-7 months old) and followed by further escalating doses in March and April (7-8 months old). 

Gene-Editing Design

The team chose to target one of the two mutants (rather than both), and selected the Q335X variant for the adenine base editing (A—>G conversion). Maybe this is a good strategy because of off-targets and bystand targets.

Outcome: 

Doctors reported that the treatment was successful in correcting the genetic defect and reducing ammonia levels in KJ’s blood. He was able to go home from the hospital in June 2025 and is currently thriving. 

We do not know editing efficiency, off-target and by-stand target rate, but saved a life. 

For more detailed news, please click here

(biotech news) Personalized CRISPR startup, Aurora, Jan 9 2026

Poem Book—Returning to Origins—Free

Last time when I visited Korea, one of my sunbaes (college seniors) told me that even startups need practice (she is a CEO of Biotech CRO) and practice with any kind of business. I thought about what I could do. That was making books, since I already had personal notes. However, I did not know how to get ISBNs, how to calculate cover thicknesses, how to choose papers, or where to print and sell books.

In Korea, I used Indipub for distribution. Actually, I arranged for the printed book to be delivered directly to the Indipub’s office. In the US, I used Amazon KDP to publish a poem book, both as an eBook and in print. Printing price is cheaper in the US than in Korea, but the quality and variety are limited in the US.

This year, I tried creating children’s picture books using Pages and JPEG images by exporting as ePub. While I worked on them, I realized that JPEG (file size) is smaller than PNG, and iBooks does not support any more ePub previews, so I used Cradle instead of iBooks. I just published two books through Amazon KDP and Barnes & Noble. One book was by my first daughter, and the other by my second daughter. I hope to continue supporting other kids to publish their own books. Writing and publishing seem to help them grow and become more thoughtful about the world around them.

Furthermore, working together with my kids makes me happy and helps me learn more and allows me to better understand how the world works and how the world changes quickly. Today, I discovered how to make eBooks available for free. At Amazon KDP, authors can not offer their books for free all the time, but through KDP, we can make them free 5 days within a 90-day period.

From today, Sep27 to Sep 30, the book, Returning to Origins, is free : https://a.co/d/cIdHYP5

Thanks

Published a book of poetry, returning to origins

Last summer, I had English class at MiraCosta. The lecture/class was led by Professor Donna Fazio DiBenedetto.

The class started with a few quotes. One of them was:

“The scariest moment is always just before you start. After that, things can only get better.” ~ Stephen King

“I do believe writing is thinking. Sometimes we can’t untangle what’s happening in our brains, but we get our pen moving and all of a sudden, as we write, we figure it out.” —Elizabeth AcevedoLinks to an external site.

One of the main books that we should read was “Siddahartha by Herman Hesse”.

While taking classes, I looked back myself, turned to my past, and started looking through old poems that I wrote quite long ago, and finally, I decided to share them with others.

In Korea, I published a book titled 원점 at a small independent bookstore, INDIEPUB.

In the USA, I published it as an ebook and a paper book, titled Returning to Origins at Amazon

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