Research into CRISPR gene editing technology continues to expand the amount of potential applications, while shrinking the actual size of proteins used in treatments. By cutting the number of amino acids in a cas protein in half, scientists claim to have turned a non-working CRISPR into a working one for the first time.
At the same time, a heightened interest into RNA is changing the way we view genomics. New research promises to utilize human proteins that can minimize adverse immune responses, eliminate viruses like HIV and Zika, and even re-order the “letters” of the misspelled RNA that leads to muscular dystrophy.
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Many researchers studying gene editing technologies like clustered regularly interspaced short palindromic repeats (CRISPR) are now focusing more closely on RNA.
A new system, dubbed SEND by Dr. Feng Zhang and his colleagues at Harvard and MIT’s Broad Institute, leverages the ability of a human protein called PEG10 to bind to its own mRNA and form a protective capsule around it. In a new study published in Science, Zhang and colleagues engineered PEG10 to take on RNA cargoes of their choice and successfully delivered the system to mouse and human cell. Because SEND uses a protein that’s produced naturally in the body, Fierce Biotech notes that it may not trigger the kind of immune responses that can render gene therapies ineffective.
Dr. Zhang’s lab has been an incubator for some of the top CRISPR researchers that have emerged in recent years. Sherlock Bioscience founders Omar Abudayyeh and Jonathan Gootenberg (each having worked at Zhang’s Broad Institute lab), for instance, have just rolled out research on their new CRISPR cas7-11 system that could forever change the way we treat diseases like Huntington’s and muscular dystrophy, each caused by spelling errors in RNA.
CRISPR Weaponized Against Viruses
As Biospace.com writes, all CRISPR proteins discovered thus far are used by bacteria as a defense against viruses. The protein stores a memory of the pathogen in its genetic material, and guided by a small piece of RNA, it targets and destroys the infection. As such, they are a natural breeding ground for potential therapeutic tools.
Essentially, a focus on RNA could soon open up a gene editing offensive on viruses. MRP has highlighted CRISPR’s virus fighting potential since 2019.
Excision BioTherapeutics – licensing a technology from the Lewis Katz School of Medicine at Temple University – has combined CRISPR with antiretroviral therapy, to work on eliminating HIV, the virus that causes AIDS. As Massive Science wrote, scientists can use artificially-created guide RNAs to direct cas9 protein to the targets of interest. In that particular HIV study, after suppressing the HIV infection with LASER ART, a treatment effective in stopping the production and insertion of new copies of the virus, the researchers treated the mice with edited guide RNAs that excised the viral DNA from the genome — actually “curing” the mice of the chronic disease.
Although the editing was effective in fewer than half the mice, and some worry that the HIV has the potential to mutate and avoid further CRISPR treatments, we will finally have some definitive results from human trials soon as Excision secured $60 million earlier this year…
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