Lentiviruses, a genus of the retroviridae family, are genetic vectors derived from the human immunodeficiency virus and are widely used for gene editing in eukaryotic cells such as neuronal cells and hematopoietic stem cells. Lentiviral vectors have been shown to be able to express multiple genes from a single vector and efficiently transduce postmitotic and quiescent cells. In addition, lentivirus vectors have the characteristics of a wide host range, low immunogenicity, large gene capacity, and long-term expression, making them widely used as gene vectors in basic research and clinical practice.
In the field of rare diseases, the use of lentiviral vectors in gene therapy may be applicable to many rare lysosomal diseases, and several lentiviral gene therapies for lysosomal diseases are being investigated. Lentiviral gene therapy has been shown to involve the insertion, modification, or deletion of genes using the family of lentiviruses that cause AIDS and other diseases. This therapy has already been approved for the treatment of metachromatic leukodystrophy (MLD) and beta thalassemia, and is being investigated for use in at least a dozen other rare diseases, such as Fabry disease, Gaucher type 1 disease, Hunter disease, and Sanfilippo syndrome.
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