| DEVELOPMENT | We are developing ZFPs for the targeted repair of genes that contain mutations in their sequence. By engineering ZFPs with an alternative functional domain, a Type II restriction endonuclease such as Fok I, we can make cuts at a precise location in the DNA sequence of a target gene that may carry a mutation and facilitate the substitution of a piece of DNA encoding the corrected sequence, a process known as homologous recombination. This approach may be useful for the treatment of conditions such as Sickle Cell Anemia, Severe Combined Immunodeficiency (SCID, or boy-in-a bubble disease) and Gaucher’s Disease in which correction of a mutation in a single gene would relieve symptoms of the disease |
| COMPANY | This data is not available for free |
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