Summary
Orphagenix is using a family of patented techniques designed to correct point mutations, developed by researchers led by molecular biologist Eric Kmiec of the University of Delaware in Newark. Referring to Kmiec as a "pioneer," Herr says Orphagenix is applying Kmiec's techniques to four point-mutation diseases: sickle-cell anemia, spinal muscular atrophy (floppy baby syndrome), juvenile macular degeneration (an eye disease), and alpha-1 antitrypsin deficiency (can cause lung and liver failure).
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Extract
Fixing Genetic Errors
Orphagenix uses targeted gene alteration to correct point mutations by repairing mismatches.
The paradigmatic gene therapy technique involves slipping new genes into cells on the backs of modified virus...See the full content of this document
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