Work

96. Genomic Integration Profile of AAVHSC Vectors in Human CD34+ Long-Term Engrafted Hematopoietic Stem Cell Xenografts in Immune-Deficient Mice

Year: 2015

Type: article

Abstract: Adeno-associated virus vectors (AAV) are proving to be promising and powerful for gene delivery due to their ability to safely transduce both non-dividing and dividing cells. While AAV mainly persists in episomal copies in non-dividing cells, genomic integration has been reported including in CD34+ hematopoietic stem cells (HSCs). Characterizing the vector integration profile is important to ensure safety since genomic integration has the potential for inducing insertional mutagenesis and genotoxicity.

Source: Molecular Therapy

DOI: 10.1016/s1525-0016(16)33701-7

Authors: Manasa Chandra, Tahira Ul-Hasan, Laura J. Smith, K.K. Wong, Saswati Chatterjee

Institutions: City Of Hope National Medical Center, Beckman Research Institute, City of Hope

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