Articles on Spinal Muscular Atrophy research
23 Apr 2007
OrphageniX Inc., a new biotechnology company founded by University of Delaware researchers, has been established in Wilmington to develop and commercialize University of Delaware-patented technologies for repairing genes that cause rare, hereditary diseases such as sickle cell anemia and spinal muscular atrophy. The announcement was made in a news release issued by the company on April 13.
Eric Kmiec, professor of biological sciences, and Hetal Parekh-Olmedo, senior research associate, both in the UD College of Arts and Sciences, co-founded and incubated OrphageniX at UD’s Delaware Biotechnology Institute in the Delaware Technology Park in 2005. 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).
Read the full press release
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