… of gene therapy. Zolgensma involves the use of an adenoassociated virus (AAV) vector, one
of the leading approaches to gene therapy… issues associated with viral vectors including the …

… A major obstacle in cancer gene therapy is … gene transfer by replication-defective retrovirus
vectors in current use. We had also developed replication-competent retroviral (RCR) vectors…

… Small scale adeno-associated virusvector production for preclinical gene delivery based
on chloroform precipitation Gene therapy aims to introduce genetic information into a cell-type …

… safety of gene therapy are very dependent on the vectors. Many studies and developments
have … The neutralizing antibodies against AAV are considered as a major obstacle. The pre-…

… Although the BBB represents a major obstacle for drug delivery … gene therapy); viral or
non-viral vectors carrying the gene for NGF or NGF analogs/ receptor ligands (in vivo gene therapy…

… barriers to systemic delivery of siRNA limit the use of naked siRNA; therefore, siRNAvectors
developed to protect and deliver these therapies … the potential for gene therapy for various …

… of Vector and compound, effects on apical endocytosis, activation of unscheduled DNA
synthesis, etc.) will be discussed. A major barrier to successful AAV and lentiviral gene therapy in …

… ABSTRACT Human gene therapy needs to express exogenous … gene therapy with the GAD
gene via an rAAV vector. … overcome some of the obstacles faced with gene therapy , such as …

… corrected through a transposon vector and then transplanted … a major obstacle for the
engraftment of primary human cells, … -based gene therapy approach should be developed to treat …

… specifically the brain, is another challenge in the development of gene therapy. Transfer of
… facilitating genetic treatment by AAV. Transgene expression of vectors including the gene of …