Adeno-associated viruses in gene therapy for spinal muscular atrophies: trend or triumph?

Abstract

The development of gene therapy in the 21st century is largely based on application of viral vectors, which have shown their effectiveness along with a fairly high safety profile. Among the vector systems, one of the leading places was taken by adeno-associated viruses (AAV), on the basis of which drugs were created for the treatment of severe hereditary monogenic diseases, including spinal muscular atrophies (SMA). Their use, on the one hand, is justified by the flexibility of AAV as a platform for the creation of gene therapy drugs, and on the other hand, it is often perceived as a kind of trend that has significant limitations. In this review, the focus is on two main aspects: AAV as a vector for the treatment of diseases from the SMA group and possible directions of development in this area and in gene therapy in general.
 The review operates with recent data published after clinical trials and experimental studies during last decade, and also critically examines the possibilities of gene therapy using AAV, mentioning other existing approaches, incl. medical therapy for SMA.
 Attention is also paid to the situation in the field of using AAV for the treatment of other hereditary diseases and the most acute problems faced by the use of drugs created on the basis of this promising vector system.