Program Snapshot

The goal of the SCGE program is to accelerate the development of safer and more effective methods to edit the genomes of disease-relevant somatic cells and tissues in patients. For ethical, legal and safety reasons, the SCGE program does not support any research activities on genome editing in reproductive (germ) cells.

In The News

Francis Collins & David Liu paper accepted in Nature

Base editing successfully treats progeria in mice

Correcting the mutation that causes progeria with base editing leads to strong symptom reduction and longer lifespan in an animal model

Nobel Prize in Chemistry Awarded to Emmanuelle Charpentier and Jennifer A. Doudna

Emmanuelle Charpentier and Jennifer A. Doudna have discovered one of gene technology’s sharpest tools: the CRISPR/Cas9 genetic scissors. Using these, researchers can change the DNA of animals, plants and microorganisms with extremely high precision. This technology has had a revolutionary impact on the life sciences, is contributing to new cancer therapies and may make the dream of curing inherited diseases come true.

Methods Matter: Standard Production Platforms for Recombinant AAV Produce Chemically and Functionally Distinct Vectors

Different approaches are used in the production of recombinant adeno-associated virus (rAAV). The two leading approaches are transiently transfected human HEK293 cells and live baculovirus infection of Spodoptera frugiperda (Sf9) insect cells. Unexplained differences in vector performance have been seen clinically and preclinically.

New Insights into How COVID-19 Causes Heart Damage

COVID-19 was initially identified as a respiratory disease, but scientists now appreciate that it also affects several other organs in the body, including the heart.

mRNA Delivery Using Bioreducible Lipidoid Nanoparticles Facilitates Neural Differentiation of Human Mesenchymal Stem Cells

Mesenchymal stem cells (MSCs) are widely used in regenerative medicine and tissue engineering and delivering biological molecules into MSCs has been used to control stem cell behavior.

Important COVID-19 Updates from the NIH

COVID-19 is an emerging, rapidly evolving situation. Get the latest information here, including important prevention and mitigation strategies, and how to best protect you and you family.

Important information for laboratories can also be found here.

Thank you and stay safe!