A $14M grant will fund research on gene-editing therapies for rare metabolic diseases at the Perelman School of Medicine at the University of Pennsylvania (Penn) and Children's Hospital of ...
CRISPR-Cas9 genome editing exploits the CRISPR-Cas system to modify a genome in a targeted manner. Guided by RNA, the Cas9 endonuclease breaks DNA at a target sequence. Imprecise repair of the ...
Using a form of CRISPR technology, the ultimate vision of the four-year grant is to create a platform for rapid development of personalized gene-editing therapies for a wide range of rare genetic ...