Table of Contents

I. Introduction
II. Complexity of Gene Replacement Techniques
III. Progress in Gene Therapy Research and Gene Editing Technologies
IV. Collaboration of Biogen and University of Pennsylvania for Gene Therapy Research
V. Avellino Labs and Ulster University Alliance for Gene Editing Techniques in Ophthalmology
VI. Potential of CRISPR Technology in Combating HIV
VII. Conclusion

Over four decades of research has shown that gene replacement techniques are more complex than initially perceived. The complexity mainly stems from the inability to control the target genetic material in its environment. However, scientists have taken this challenge to their heart and made sizeable progress during this time and have shown promising clinical results. Also, some genes are too large to be transferred via delivery vectors. Gene editing methods are costly as it aims to achieve modifications to genome sequences with the utmost regard for target orientation and precision.Large numbers of Pharmaceutical and Biotechnology firms are continuing its investments in gene editing therapies due to the potential reward it offers besides the vast array of diseases it promises to cure in the years ahead. Biogen, one of the oldest biotechnology firms in the United States, continues to collaborate with University of Pennsylvania to advance gene therapy research and gene editing technologies. The firm has a significant presence in therapeutic segments of neurology, hematology and immunology; the new agreement will focus on the development of targeted therapies for the eye, skeletal muscles, and central nervous system.


Likewise, Avellino Labs is in a tie-up with Ulster University located in Northern Ireland to develop new gene editing techniques and create therapies for different ophthalmic conditions. The alliance aims to use CRISPR genome editing as a new therapeutic platform in ophthalmology.

Pronounced as crisper, CRISPR is a biological system for altering DNA; CRISPR was discovered in 2012 by a team at the University of California headed by molecular biologist Professor Jennifer Doudna when studying bacterial defense mechanism against viral infection. Since then, the technique has aroused interest in both corporate and academic circles.

In May 2016, the scientists used the technology to cut a segment of HIV DNA from the genes of an infected animal. Professor Kamel Khalili who led the study at Temple University observed gene-editing technology can be delivered to multiple organs and large fragments of viral DNA can be excised at once from the host cell genome. Currently, HIV infections are treated by a combination of antiretroviral drugs. This demonstration of the concept is widely regarded as a step away from finding a permanent cure for HIV that had plagued the humankind for almost four decades now. Importantly, CRISPR has been in the limelight for all the right reasons in the medical community since its discovery.

The Splicing of HIV DNA and RNA adds to the much-needed credibility for CRISPR as antiretroviral drugs face significant challenges and roadblocks when it comes to taking on the virus that hides in other reservoirs in the body. Also, there is a significant challenge faced by healthcare practitioners as HIV replication rebounds when the treatment is halted.

The technique could be the beginning of something worthwhile in the medical field after a long time in combating HIV. If everything goes well, the clinical trials data could provide much-needed relief to the selective population where HIV growth outpaces the awareness and medical initiatives.