Work

105. A Single Administration of Low Dosage (5 x1010 vp/kg) hFX/HDAd Complex Achieves Saturated hFVIII Levels in the Blood of Mice

Year: 2015

Type: article

Abstract: The development of helper-dependent adenoviral vectors (HDAds) devoid of all functional viral genes has largely overcome cellmediated host immune responses associated with Ad viral proteins. However, a dose-related acute toxicity associated with systemic delivery has been a major obstacle to the clinical translation of Adbased vectors. Since HDAd has a large transgene cargo (up to 34 kb), we recently succeeded in producing an HDAd encoding two codon-optimized hFVIII variant N6 expression cassettes (HDAd2xcoN6).

Source: Molecular Therapy

DOI: 10.1016/s1525-0016(16)33710-8

Authors: Masataka Suzuki, Michele Martinez, Alice Tarantal, Brendan Lee

Institution: Baylor College of Medicine

Language: English

Topic: Virus-based gene therapy research

Subfield: Genetics

Field: Biochemistry, Genetics and Molecular Biology

Domain: Life Sciences

SDG: Good health and well-being

Open Access status: hybrid

Estimated APC paid: $4,500