Techniques of gene replacement have been found to be quite complex in the four decades this kind of research has been going on. This complexity is because it is difficult to control the targeted genes in their environment. Scientists however, have taken up the challenge and good progress has been made with promising clinical trials and results. It has been found that there are genes that are so big that delivery vectors cannot be used to transfer them. It is expensive to edit genes even as scientists try to achieve various modifications in genome sequences with great regards for precision and target orientation.
Biotech firms and pharmaceuticals are investing heavily in the various gene editing therapies presented. The potential rewards they offer in addition to the many diseases they promise to cure in the future push them forward. One of these biotech firms is Biogen. Based in the United States, they are collaborating with the University of Pennsylvania in order to move gene therapy research as well as gene editing technology forward. They have also invested heavily in therapeutic divisions of immunology, hematology and neurology. The current agreement will center on developing targeted therapies for the central nervous system, skeletal muscles and the eye.
Another similar collaboration is between Avellino Labs and Ulster University of Northern Ireland. They are looking to develop novel gene editing techniques as well as create therapies for a variety of ophthalmic conditions. They will be using CRISPR genome editing platform.
This platform that is pronounced as crisper is used to alter DNA. It was discovered by a team from the University of California in 2012. The team was headed by Professor Jennifer Doudna a molecular biologist. They were studying how a bacterium puts up a defense when faced by a viral infection when they discovered CRISPR. From that time interest in the technique by both academic and corporate entities has been aroused.
A study carried out at Temple University, led by Professor Kamel Khalili in May 2016, cut out from the genes of an animal infected with HIV a portion of HIV DNA. He observed that using gene editing technology one is able to cut out large fragments and multiple organs of viral DNA from the cell genome of the host at once. This kind of demonstration is one that is regarded as being just a step away from the discovery of a permanent-cureing-for-AIDS.
With the ability to splice HIV RNA and DNA, CRISPR has become even more credible seeing as antiretroviral medication is challenged when the virus hides in the various reservoirs found in the body. Additionally, healthcare practitioners also find themselves significantly challenged since halting the medication leads to a rebound of HIV replication.
This new technique could actually be the first step in a worthwhile cause in the 40 years of combating HIV. Should everything go as planned, the data collected during clinical trials could provide the relief that is much needed by those in populations where the growth of HIV is faster than medical initiatives and awareness.