The Jackson Laboratory in Bar Harbor has been awarded a $22 million federal grant to develop new gene editing therapies to cure rare neurological disorders.
Dr. Cat Lutz is spearheading the effort which will research gene editing to treat disorders such as spinal muscular atrophy and Huntington's disease.
"In gene editing, you're just going into the cell and you're fixing the mutation. A lot of people like to call it the one and done, because once it's fixed, it's fixed and you don't have to go back and worry about anything else again," Lutz says.
Lutz will collaborate with several other institutions on the five-year grant, including Massachusetts General Hospital and Boston Children’s Hospital.
Lutz says developing therapies for rare disease has been a challenge due to a small number of patients, high costs, and regulatory barriers.
