Published in Gene Therapy Weekly, June 5th, 2008
of human gene therapy, announced that it obtained a license from La Sapienza University in Rome, Italy, to their advanced small nuclear RNA (snRNA)-based exon-skipping technology for the treatment of Duchenne muscular dystrophy (DMD). The combination with AMT's proprietary adeno-associated virus (AAV) gene therapy platform potentially makes up a long-term treatment for this seriously debilitating disease with a single administration of the product.
Keywords: Biotechnology, Duchenne Muscular Dystrophy, Gene Therapy, Genetics, Genomics, Molecular Research, Molecular Therapies,...
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