Article
Selective elimination of mitochondrial mutations in the germline by genome editing.
Cell - 23 Apr 2015
Reddy Pradeep, Ocampo Alejandro, Suzuki Keiichiro, Luo Jinping, Bacman Sandra R, Williams Sion L, Sugawara Atsushi, Okamura Daiji, Tsunekawa Yuji, Wu Jun, Lam David, Xiong Xiong, Montserrat Nuria, Esteban Concepcion Rodriguez, Liu Guang-Hui, Sancho-Martinez Ignacio, Manau Dolors, Civico Salva, Cardellach Francesc, Del Mar O'Callaghan Maria, Campistol Jaime, Zhao Huimin, Campistol Josep M, Moraes Carlos T, Izpisua Belmonte Juan Carlos
Abstract excerpt
Mitochondrial diseases include a group of maternally inherited genetic disorders caused by mutations in mtDNA. In most of these patients, mutated mtDNA coexists with wild-type mtDNA, a situation known as mtDNA heteroplasmy. Here, we report on a strategy toward preventing germline transmission of mitochondrial diseases by inducing mtDNA heteroplasmy shift through the selective elimination of mutated mtDNA. As a...
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