Temple University researchers have successfully cured an HIV infection from laboratory mice. They have accomplished this using the gene editing technique “CRISPR.” This successful test marks the first time that the gene technology has been used in such a way, and may hold promises for the future. Though this newest accomplishment is encouraging and highlights the promise of gene editing, challenges remain to adapting this sort of treatment for humans.
Paul Volberding, head of the amfAR Institute for HIV Cure Research and director of the UCSF AIDS Research Institute said in a statement to CBS News:
“The basic science community in HIV research is now very focused on finding a cure. It still feels a long way off but the tools we now have definitely including the gene editing used in this report is accelerating our work and raising optimism. The cure field is in very close contact and collaborations are active world wide. It’s really quite exciting!”
“Gene editing is a potent and still rather new tool in HIV research and many other areas as well,” he said. “It faces a challenge in scalability — getting the technology simplified and inexpensive — but is certainly worth following.”
CRISPR is a method of genetic editing, and stands for “clustered regularly interspaced short palindromic repeats.” According to YourGenome.org, the CRISPR system consists of two key molcules that introduce a mutation into targeted DNA: an enzyme, called Cas9, and a piece of RNA, called guide RNA or gRNA.
John Meyer in Consumers’ Research magazine writes:
“Thus, CRISPR allows easy, extensive, and accurate editing of the genome, marking a quantum leap in the ease and affordability of the development of practical genetic engineering of animals and humans, while making the already established genetic engineering processes in plants and bacteria much more versatile and efficient. For example, it used to take approximately one million tries to get a mouse cell to accurately include a desired new characteristic; CRISPR can attain the same result in about 10 tries. CRISPR both makes established genetic engineering processes orders of magnitude more efficient and opens up many new areas that were not feasible with prior techniques.”
Gene editing via CRISPR enables genetic engineering of animals other than mice, because it is more accurate than traditional editing. Due to this, mice have traditionally been the only animals used in genetic modeling of human disease.
There are a myriad of other applications of this genetic editing technology; according to YourGenome.org CRISPR has the potential for treating cancer, Hepatitis B, high cholesterol, and other conditions that have a genetic component. Other non-medical applications could include editing reproductive cells, also known as germline cells. This mutation would be passed down, so editing of germline cells is very controversial – in fact, it is illegal in many countries. Editing of non-reproductive, or somatic cells, is fairly uncontroversial.