Project Description

Targeted gene delivery into haematopoietic stem cells (HSCs) is an attractive strategy for gene therapy/gene editing of haematological diseases. Retroviruses and lentiviruses have been extensively used for gene transfer into HSCs. However, the risk of insertional mutagenesis/integration of proviral sequences into active genes of target cells outweighs the benefit of transgene expression. On the other hand, Adeno associated virus (AAV) vectors although safest, has its use limited in HSC due to incompletely understood transduction efficiency. Here we wish to understand the propensity of AAV to transduce cells such as HSCs. Based on such understanding, we propose to rationally engineer AAV capsids so as to facilitate their improved transduction efficiency.