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Posts tagged “CRISPR-Cas9

Stem cell gene therapy could be key to treating Duchenne muscular dystrophy

crispr cas9 art

crispr cas9 art

Scientists at the UCLA Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research and Center for Duchenne Muscular Dystrophy at UCLA have developed a new approach that could eventually be used to treat Duchenne muscular dystrophy. The stem cell gene therapy could be applicable for 60 percent of people with Duchenne, which affects approximately 1 in 5,000 boys in the U.S. and is the most common fatal childhood genetic disease.

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Scientists discover new system for human genome editing

Bacteriophages attacking bacteria, TEM

CRISPR systems help bacteria defend against viral attack (shown here).
These systems have been adapted for use as genome editing tools in
human cells.
Image credit goes to: Ami Images/Science Photo Library.

A team including the scientist who first harnessed the revolutionary CRISPR-Cas9 system for mammalian genome editing has now identified a different CRISPR system with the potential for even simpler and more precise genome engineering. In the study researchers describe the unexpected biological features of this new system and demonstrate that it can be engineered to edit the genomes of human cells.

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