Our Proposal
Our Future Technology:
Although significant strides have been made in the advancement of gene editing in the past few years with the discovery of CRISPR, scientists are still limited by the inability to deliver Cas9 to all cells. Our future technology is to advance gene editing by developing a viral vector, (possibly the Adeno Associated Virus,) capable of carrying Cas9 to all of the cells in the human body and thus, moving the science from the laboratory to implementation in the prevention and cure of human health and disease. (1)
Breakthroughs:
There are a few scientific breakthroughs that will have to occur in order for our future technology to become a reality. The CRISPR technology needs to be improved so that there is very little chance of off target effects, which would cause the misplacement of the technology in a strand of DNA.(2) The transmission of the technology also needs to be improved. Our proposal includes using a Adeno Associated Virus ( AAV), to transmit the CRISPR technology into human cells. This virus has to be compatible with the patient so that the patient's immune system does not immediately fight it off.
A series of tests can be conducted to improve our technology. Researches at Nanjing University have successfully inserted the CRISPR technology into a macaque embryo and edited the monkeys genome.(3) The monkeys were born safely and their genetic material was modified. Instead of injecting genetic material into an embryo we could use our application method of an AAV and test that on primates such as the macaque. This would allow use to safely test our technology and correct any issues before it was applied to humans.
(1) "The World of Nucleases." Nuclease-Based Genome Editing. N.p., n.d. Web. 25 Jan. 2015
(2) "Vector Biolabs: Introduction to AAV." Vector Biolabs: Introduction to AAV. N.p., n.d. Web. 25 Jan. 2015.
(3) Rojahn, Susan Young. "Monkeys Modified with Genome Editing." N.p., n.d. Web. 25 Jan. 2015.
Although significant strides have been made in the advancement of gene editing in the past few years with the discovery of CRISPR, scientists are still limited by the inability to deliver Cas9 to all cells. Our future technology is to advance gene editing by developing a viral vector, (possibly the Adeno Associated Virus,) capable of carrying Cas9 to all of the cells in the human body and thus, moving the science from the laboratory to implementation in the prevention and cure of human health and disease. (1)
Breakthroughs:
There are a few scientific breakthroughs that will have to occur in order for our future technology to become a reality. The CRISPR technology needs to be improved so that there is very little chance of off target effects, which would cause the misplacement of the technology in a strand of DNA.(2) The transmission of the technology also needs to be improved. Our proposal includes using a Adeno Associated Virus ( AAV), to transmit the CRISPR technology into human cells. This virus has to be compatible with the patient so that the patient's immune system does not immediately fight it off.
A series of tests can be conducted to improve our technology. Researches at Nanjing University have successfully inserted the CRISPR technology into a macaque embryo and edited the monkeys genome.(3) The monkeys were born safely and their genetic material was modified. Instead of injecting genetic material into an embryo we could use our application method of an AAV and test that on primates such as the macaque. This would allow use to safely test our technology and correct any issues before it was applied to humans.
(1) "The World of Nucleases." Nuclease-Based Genome Editing. N.p., n.d. Web. 25 Jan. 2015
(2) "Vector Biolabs: Introduction to AAV." Vector Biolabs: Introduction to AAV. N.p., n.d. Web. 25 Jan. 2015.
(3) Rojahn, Susan Young. "Monkeys Modified with Genome Editing." N.p., n.d. Web. 25 Jan. 2015.