Toshiyuki Kurai

Global diffuse distribution in the brain and efficient gene delivery to the dorsal root ganglia by intrathecal injection of adeno-associated viral vector serotype 1

The success of gene therapy for inherited neurodegenerative diseases such as metachromatic leukodystrophy (MLD) depends on the development of efficient gene delivery throughout the brain guarded by the blood–brain barrier and achieves distribution of the deficient enzyme throughout the brain. Direct injection of viral vector into the brain parenchyma is too invasive and may not be sufficient to treat the entire brain. As an alternative approach, we examined the feasibility of intrathecal (IT) injection of adeno‐associated viral vector serotype 1 (AAV1).

1071. Potential Utility of Adipose-Derived Stromal Cells (ASCs) as a Carrier for Ex Vivo Gene Therapy |[ndash]| Gene Transfer Efficiency of ASCs

Background: Immature pluripotent cells are important targets for tissue engineering, regenerative medicine, and gene therapy. In addition to bone marrow derived stromal cells (BSCs), we and others have demonstrated that adipose tissue derived stromal cells (ASCs) contain mesenchymal stem cells. Compared to well characterized BSCs, ASCs are easy to obtain, have relatively low donor site morbidity, and are available for harvest in large numbers. It was previously demonstrated that ASCs differentiate into a variety of cell lineages both in vitro and in vivo. In this report, we studied the gene transfer efficiency of ASCs to evaluate their potential utility as a carrier for cell-based gene therapy. Hereinafter, the term |[ldquo]|adipose-derived stromal cells|[rdquo]| is abbreviated to |[ldquo]|ASCs|[rdquo]|, and |[ldquo]|bone marrow-derived stromal cells|[rdquo]| to |[ldquo]|BSCs|[rdquo]|. Both types are considered to include mesenchymal stem cells (MSCs).