CRISPR/Cas9 was first successfully demonstrated as a tool for genome editing in human cells in 2013. Since then, several advances have enabled the use of this technology to shut down a faulty gene, change a single DNA letter in a gene sequence, or add a new gene to an existing genome sequence. This summer has...
The gene therapy field has seen several developments in recent months, especially around CRISPR, with both significant achievements and further controversies about the safety of the gene editing approach. CRISPR Cure for Duchenne’s Muscular Dystrophy (DMD) in Dogs Eric Olson’s team of researchers at the University of Texas Southwestern’s Harmon Center for Regenerative...
Back in April of this year, we wrote about the National Academies of Science and Medicine cautiously endorsing the possible use of gene editing at the germline level to correct serious diseases or medical conditions. Their report commented that scientists would likely be in a position to accomplish such activities safely within 5 to 10...
Sometime mid-month, the first clinical trial employing CRISPR gene editing technology within the human body will initiate in China, at Sun-Yat Sen University. Scientists there will seek to use CRISPR and TALEN, another gene editing method, to treat persistent HPV infection and disable the tumor growth mechanism in infected cells. In a non-invasive approach, scientists...
Duchenne Muscular Dystrophy (DMD) is a rare genetic disease characterized by progressive muscle degeneration and weakness. It is one of the most frequent genetic disorders, affecting approximately 1 of 3,600 (primarily) male children. The disease is caused by a mutation in the gene for dystrophin on the X chromosome that results in the absence of...
On September 21, Facebook CEO and billionaire, Mark Zuckerberg, and his pediatrician wife, Dr. Priscilla Chan, announced a new, privately funded big science effort. The couple pledged more than $3 billion toward a plan to “cure, prevent or manage all disease within our children’s lifetime.” The couple has pledged to donate over $3 billion...