When it comes to challenges in the genome editing space, all eyes are on delivery. Efficient editing of cells directly in an organism (rather than ex vivo) and targeting of relevant stem cells is still a hurdle to be cleared in the progress of genetic medicine.
Now, new work describes the optimization of lipid nanoparticles (LNPs) for delivering gene-editing reagents and delivery to all lung cell types, including stem cells. The approach, which was tested in patient-derived cells and in a mouse model of cystic fibrosis, demonstrated therapeutically relevant editing lasting for up to a year… Continue reading.
...As they grow, solid tumors surround themselves with a thick, hard-to-penetrate wall of molecular defenses. Getting drugs past that barricade is notoriously difficult. Now, scientists at UT Southwestern have developed nanoparticles that can break down the physical barriers around tumors to reach cancer cells. Once inside, the nanoparticles release their payload: a gene editing system that alters DNA inside the tumor, blocking its growth and activating the immune system.
The new nanoparticles, described in Nature Nanotechnology, effectively stopped the growth and spread of ovarian and liver tumors in mice. The system offers a new path forward for the use of the gene editing tool known as CRISPR-Cas9 in cancer treatment, said study leader Daniel Siegwart, Ph.D., Associate Professor of Biochemistry at UT Southwestern… Continue reading.
...WASHINGTON, D.C. — The American Institute for Medical and Biological Engineering (AIMBE) has announced the election of Daniel J. Siegwart, Ph.D., Associate Professor, Dept. of Biochemistry, University of Texas Southwestern Medical Center to its College of Fellows. Dr. Siegwart was nominated, reviewed, and elected by peers and members of the AIMBE College of Fellows for his outstanding contribution to lipid nanoparticles for nucleic acid delivery.
The College of Fellows is comprised of the top two percent of medical and biological engineers in the country. The most accomplished and distinguished engineering and medical school chairs, research directors, professors, innovators, and successful entrepreneurs comprise the College of Fellows. AIMBE Fellows are regularly recognized for their contributions in teaching, research, and innovation. AIMBE Fellows have been awarded the Nobel Prize, the Presidential Medal of Science and the Presidential Medal of Technology and Innovation, and many also are members of the National Academy of Engineering, National Academy of Medicine, and the National Academy of Sciences… Continue reading.
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