In Vivo Genome Editing of Stem Cells Induced by LNP-Based Delivery of mRNA
By Jonathan D. Grinstein,
Genetic Engineering & Biotechnology News
| 07. 27. 2023
Part of the job as a pediatric hematologist for Michael P. Triebwasser, MD, PhD, is to take care of patients during their bone marrow transplantation. Just last week, he took care of a patient with a severe combined immunodeficiency (SCID) disorder and another with a bone marrow disorder.
Even though there has been progress in the use of autologous cells for ex vivo gene therapies for hematopoietic disorders like sickle cell disease and beta thalassemia, Triebwasser warns that it still requires the invasive procedure of taking cells out of the body, putting electrophoresis on these cells, and “conditioning” the patients to get rid of their own hematopoietic stem cells (HSCs) to make room for the gene-edited ones. That’s why Triebwasser said that he and others have been on the hunt for “the holy grail of HSC gene therapy”—in vivo genome editing of HSCs.
“In theory, we can replace that gene or correct it in some way and offer them a curative therapy that doesn’t have some of the same issues that hematopoietic stem cell transplantation (HSCT) does, namely graft versus host disease,” Triebwasser...
Related Articles
By Mary Annette Pember, ICT News [cites CGS' Katie Hasson] | 04.18.2025
The sight of a room full of human cadavers can be off-putting for some, but not for Haley Omeasoo.
In fact, Omeasoo’s comfort level and lack of squeamishness convinced her to pursue studies in forensics and how DNA can be...
Gray wolf by Jessica Eirich via Unsplash
“I’m not a scarcity guy, I’m an abundance guy”
– Colossal co-founder and CEO Ben Lamm, The New Yorker, 4/14/25
Even the most casual consumers of news will have seen the run of recent headlines featuring the company Colossal Biosciences. On March 4, they announced with great fanfare the world’s first-ever woolly mice, as a first step toward creating a woolly mammoth. Then they topped that on April 7 by unveiling one...
By Katrina Northrop, The Washington Post | 04.06.2025
photo via Wikimedia Commons licensed under CC by 3.0
China's most infamous scientist is attempting a comeback. He Jiankui, who went to jail for three years after claiming he had created the world's first genetically altered babies, says he remains...
By Anumita Kaur [cites CGS’ Katie Hasson], The Washington Post | 03.25.2025
Genetic information company 23andMe has said that it is headed to bankruptcy court, raising questions for what happens to the DNA shared by millions of people with the company via saliva test kits.
Sunday’s announcement clears the way for a new...