Latest Developments in Gene Transfer Technology: Achievements, Perspectives, and Controversies over Therapeutic Applications

Abstract

ABSTRACT

Genetically engineered Sertoli cells are able to survive allogeneic transplantation

“…Most notable are the recent reports identifying the negative issues using adenoviral vectors as a means of inserting genes into cells. 22,27 Other vectors have had similar unacceptable safety profiles, 21,22 not to mention that the current vectors are limited as to the size of the gene constructs and hence, the types of proteins that can be generated.…”

“…Traditionally, these therapies have involved the use of viral vectors or other methods to produce the expression (often transient) of the gene of interest, raising questions of safety and efficacy. [20][21][22] Cell-based gene therapy has been proposed as one means of overcoming these issues but has itself been hampered by the lack of an abundant, safe and immunologically acceptable source of the tissue. 21 Theoretically, transgenic animals can be designed to produce specific therapeutic proteins overcoming the issues of transient expression and safety associated with viral vectors, but this is hampered by immune rejection of the allogeneic or xenogeneic tissue.…”

“…[20][21][22] Cell-based gene therapy has been proposed as one means of overcoming these issues but has itself been hampered by the lack of an abundant, safe and immunologically acceptable source of the tissue. 21 Theoretically, transgenic animals can be designed to produce specific therapeutic proteins overcoming the issues of transient expression and safety associated with viral vectors, but this is hampered by immune rejection of the allogeneic or xenogeneic tissue. The use of transgenic SCs could overcome this issue by providing long-term gene expression in an immunoprivileged environment allowing the efficacious use of allogeneic or even xenogeneic cells.…”

Viral vectors for use in the development of biodefense vaccines

“…Adenovirus (Ad) was first discovered in 1954 when scientists were trying to establish cells lines derived from tonsil and adenoidal tissues and has recently been utilized extensively as a vector for gene therapy [96,97]. Human Ad belongs to the Adenoviridae family which is composed of two genera [98].…”

Synthesis of a novel series of cationic lipids that can act as efficient gene delivery vehicles through systematic heterocyclic substitution of cholesterol derivatives

“…[1][2][3][4] However, one of the most difficult hurdles in achieving effective gene therapy is the requirement for the use of efficient vehicles to deliver the gene of interest into target cells. A diverse spectrum of gene delivery vehicles ranging from replication-incompetent viruses to DNA formulated with various delivery vehicles has been utilized.…”