New approach to gene therapy can correct any disease-causing mutation within a gene. A new approach to gene editing using the CRISPR/ Cas9 system bypasses disease-causing mutations in a gene, enabling treatment of genetic diseases linked to a single gene, such as cystic fibrosis, certain types of sickle cell anemia, and other rare diseases. The method, developed and tested in mice and human tissue cultures by researchers at Penn State, involves inserting a new, fully functional copy of the gene that displaces the mutated gene.
A proof-of-concept for the approach is described in a paper appearing online April 20 in the journal Molecular Therapy.
The CRISPR/Cas9 system has allowed promising new gene therapies that can target and correct disease-causing mutations in a gene...
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