Douglas R. Cavener, Penn State Professor of Biology and former Verne M. Willaman Dean of the Eberly College of Science, has been named Dorothy Foehr Huck and J. Lloyd Huck Distinguished Chair in Evolutionary Genetics by the Huck Institutes of the Life Sciences.
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Novel approach to gene editing bypasses disease-causing mutations in 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.
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UNIVERSITY PARK, Pa. 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. In this process, Cas9 a bacterial protein cuts DNA at a specific location, where the genetic sequence can then be edited, trimmed, or a new sequence inserted before the DNA is repaired. However, there are two main limitations to current repair strategies. First, th