Novel Gene-Repair Technology Under Development

OrphageniX Inc., a new biotechnology company founded by University of Delaware researchers, has been established in Wilmington to develop and commercialize UD-patented technologies for repairing genes that cause rare, hereditary diseases such as sickle cell anemia and spinal muscular atrophy. There are more than 5,000 rare or “orphan” diseases, so named because each affects fewer than 200,000 people nationwide. A number of these diseases are caused by a single-point mutation in a gene--which is like a spelling error, a single “letter” out of place, in its DNA code. The DNA nucleotide adenine (A), for example, might be replaced by guanine (G), cytosine (C) or thymidine (T). Kmiec and Parekh-Olmedo discovered a way to introduce a tiny fragment of DNA into a diseased cell to replace the defective portion, triggering the cell to heal itself. This method, which focuses on correcting a patient's genes to make their own proteins, offers a safer approach than treating a patient's genes with foreign genes or protein replacements, and eventually may lead to cures for rare diseases, according to Michael Herr, president and chief executive officer of OrphageniX.... Whole article"ScienceDaily":[ www.sciencedaily.com]

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