Gene Therapy
Author:
Publisher
Elsevier
Reference166 articles.
1. Therapeutic in vivo gene transfer for genetic disease using AAV: progress and challenges;Mingozzi;Nat Rev Genet.,2011
2. The burden of genetic disease on inpatient care in a children's hospital;McCandless;Am J Hum Genet.,2004
3. Long-term sex-biased correction of circulating propionic acidemia disease markers by adeno-associated virus vectors;Guenzel;Hum Gene Ther.,2015
4. Self-complementary adeno-associated virus vectors containing a novel liver-specific human factor IX expression cassette enable highly efficient transduction of murine and nonhuman primate liver;Nathwani;Blood.,2006
5. Long-term safety and efficacy of factor IX gene therapy in hemophilia B;Nathwani;N Engl J Med.,2014
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