New biotech company to commercialize novel UD gene-repair technology: "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. ...
Kmiec holds 14 UD patents for gene-editing technologies and is widely regarded as a pioneer in the field.
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)."
