Gene Therapy: Benefits and Worries
Since the 1960s, the idea of gene therapy has been under investigation. The ability to transfer genes from one animal to another via controlled techniques has been made possible thanks to continual improvements. Gene therapy in mice and humans, along with its correlation, is the most widely used method in this (Willett and Bennett, pg.5). Despite having benefits, this has encountered certain worries and disadvantages.
Concerns with the Therapeutic Notion
It is evident from the literature provided for reference that there are some concerns with the therapeutic notion. Despite the identification of the AAV gene which has been accredited with the advantage of its usability in both the human and other animal gene transfer, there are still some worries being presented. These fears are associated with the gene delivery and immune responses that are likely to be emitted (Mack, pg. 842).
Worries on Safe and Efficient Gene Delivery
There are worries on the safe and efficient delivery of the genes. There is a lack of a safe way to deliver the genes especially in the inner parts of the ear or eye in the case of audio or vision problems. In addition to this, there is the worry about the limited cloning capacity of the genes. The reason for this is the fact that there might be the requirement calling for the complete replacement of the therapeutic genes. In addition to this, there is no certainty on use with large genes (Pan, pg. 265). More into this, there is limited cloning which presents the genes for integration to double the capacity of the AAV vector. As a result of this, it is likely that the patient’s immune system might respond inappropriately thereby interfering with the treatment as a result of failed cloning and coding of the genes with the designed vector resulting from failed integration.
Investigator's Efforts to Address Concerns
However, the investigators have critically tried to eradicate these worries through some responses they have accrued. They were able to come up with a commendable design that would facilitate safe and efficient delivery of the AAV gene into the body. The investigators were able to come up with the rationally designed vector that would be used to deliver the gene with minimal risks. In addition to this, they were able to come up with the assurance that this therapy could be applied in a wide range of disease treatment including inherited diseases of the skeleton (Kapetanovic, pg. 2113).
Immune Responses and side effects
Moreover, their investigation proved that the likely immune responses that are likely to accrue as a result of the AAV gene integration are insufficient. This is because, the only immunogenicities produced are neutralized by the antibodies in the body (Brigande, pg. 217). Coupled with this is the concept that the gene is more predictable on where to integrate therefore the chances of causing a violent immune response is limited. Through this investigative outcomes, there is a lessened worry on likely outcomes that are associated with the therapeutic practice.
More into this, the investigators came up with the breakthrough that declared the gene therapy to possess no side effects associated with the integration. This was as a result of the AAV gene lacking pathogenicity thereby deeming it as safe for therapeutic functions (Landegger, pg.280-281 ). Coupled with its ability to infect nondividing cells and to integrate into the genome of the human chromosomes, it is possible to use it on any cell without fear due to its quick integration. Moreover, it does not affect the life of the host because the genome remains intact. To crown it all, the only likely immune responses that could accrue as a result of the gene integration are deemed to be short-lived. The maximum extension time is six (6) hours as opposed to other enteroviruses whose effects can be displayed for over twenty-four hours (Ruan, pg. 2).
Work Cited
Bifeng Pan, Gene therapy restores auditory and vestibular function in a mouse model of Usher syndrome type 1c, March 2017. Page 265
David L. Mack, Systemic AAV8-Mediated Gene Therapy Drives Whole-Body Correction of Myotubular Myopathy in Dogs, April 2017. Page 842
Jasmina Cehajic-Kapetanovic, Restoration of Vision with Ectopic Expression of Human Rod Opsin, August 7, 2015. Page 2113
John V Brigande, Hearing in the mouse of Usher, March 2017. Page 217
Keirnan Willett and Jean Bennett ,Immunology of AAV-mediated gene transfer in the eye, Department of Ophthalmology, Scheie Eye Institute, F.M. Kirby Center for Molecular Ophthalmology, University of Pennsylvania, Philadelphia, PA, USA, page 5.
Lukas D Landegger, A synthetic AAV vector enables safe and efficient gene transfer to the mammalian inner ear, March 2017. Page 280-281
Merry Z. C. Ruan, Proteoglycan 4 Expression Protects Against the Development of Osteoarthritis, March 2013. Page 2
