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
GeneWatch UK has prepared a briefing on the genetic modification of nature for the International Union for Conservation of Nature (IUCN) Congress in October 2025
The upcoming Congress claims to be “where the world comes together to set priorities and drive conservation and sustainable development action.” A major concern for those on the outside is that the Congress may advance plans to develop and encourage the use of synthetic biology in nature conservation. This could at first glance sound like...
By Aaron Ginn, The Washington Post | 09.12.2025
Earlier this year, I had dinner in D.C. with Jensen Huang, the president and chief executive of Nvidia. At one point, he said something that struck me: “Why is everyone here so negative?”
He wasn’t referring to the economy...
By Roni Caryn Rabin, The New York Times | 08.25.2025
Scientists have dreamed for centuries about using animal organs to treat ailing humans. In recent years, those efforts have begun to bear fruit: Researchers have begun transplanting the hearts and kidneys of genetically modified pigs into patients, with varying degrees...
The Center for Genetics and Society is delighted to recommend the current edition of GMWatch Review – Number 589. UK-based GMWatch, a long-standing ally, was founded in 1998 by Jonathan Matthews as an independent organization seeking to counter the enormous corporate political power and propaganda of the GMO industry and its supporters. Matthews and Claire Robinson are its directors and managing editors.
CGS works to ensure that social justice, equity, human rights, and democratic governance are front...