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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Retroviruses are single-stranded RNA viruses capable of converting RNA to cDNA by the action of reverse transcriptase, which is then amplified by the action of proteases such as DNA replicase, transcriptase, and translatorase. Researchers usually design replication-defective viruses based on certain properties of retroviruses to produce expression vectors capable of carrying specific target genes, known as retroviral vectors.

Retroviral vectors are currently the most widely used method for human gene transfer due to their relatively high efficiency of gene transfer and their ability to randomly insert and stably integrate exogenous genes into the host cell genome for sustained expression. Among these, lentiviral vectors and gamma-retroviral vectors have been widely used in gene therapy, for example, to introduce Chimeric Antigen Receptor (CAR) genes into T cells for the targeted killing of tumors by CAR-T cells. Gene therapy has gradually replaced currently available therapies for rare genetic diseases, such as enzyme replacement therapies, autologous transplantation, and antibody therapies.
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