Work

194. Lymphatic Transport Mediates Systemic Leakage of Peripherally Administered AAV9 Vectors

Year: 2015

Type: article

Abstract: Adeno-associated viruses (AAV) have emerged as promising vector candidates for gene therapy of central nervous system (CNS) disorders. Preclinical studies in rodents as well as large animal models such as cats and non-human primates have shown that AAV9 mediates robust and widespread gene expression in the brain and spinal cord. In particular, after intracranial or intrathecal administration, leakage into the systemic circulation and accumulation of vector genomes in off-target organs such as the liver or spleen presents a potential safety concern.

Source: Molecular Therapy

DOI: 10.1016/s1525-0016(16)33799-6

Authors: Ruth M. Castellanos Rivera, Lavanya Rao, Giridhar Murlidharan, Travis Corriher, Aravind Asokan

Institution: University of North Carolina at Chapel Hill

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