Recent CRISPR News: Therapy for MD, studies about Alzheimer’s, and an end to heart attacks?
CRISPR is one of the most exciting and fast-moving fields of study in science today. Here’s a look at some recent breakthroughs that use the technology.
Could CRISPR prevent heart attacks?
A new effort from Verve Therapeutics is seeking to edit human DNA in an effort to put an end to heart attacks. As told by Bloomberg, Verve’s efforts pick up where today’s slate of pills and injections leave off.
Starting off with people who have already suffered from a heart attack due to familial hypercholesterolemia, which is a hereditary condition that affects 31 million people globally, the therapy focuses on genetic mutations that cause low levels of cholesterol. CRISPR was used to target one of two genes, with a lipid nanoparticle encasing the editing tool to protect it on its way to the liver, where it turns off the bad gene.
The treatment, Bloomberg said, was tested in monkeys, where it cut bad cholesterol by nearly 60 percent in just two weeks. Clinical trials could begin later this year, though it will likely be years before it obtains regulatory approval for widespread human use.
CRISPR-Cas9 in cancer therapy: Where we were and where we’re going
CRISPR-Cas9 has immense potential for use in therapeutic analysis in humans, notes a recent study by a team of Iranian scholars, and particularly in the diagnosis and treatment of cancer.
CRISPR applications, the team noted, extend to screening, animal modeling, diagnostics, elimination of carcinogenic viruses, DNA-based knockouts, and on potentiating CAR-T cells. The study details recent applications of CRISPR-Cas9 in liver cancer, colorectal cancer, breast cancer, cervical cancer, and others.
“Many studies have revealed that activating protein-1 (AP-1), a transcription factor, is related to cancer onset and progress, the authors wrote. “Hence, targeting AP-1 transcription factors in prostate cancer by genome editing could be a therapeutic approach.”
Could CRISPR combat muscular dystrophy?
Writing in Cell, a team of German researchers reported gains against muscular dystrophy using CRISPR-Cas9.
Gene correction of transplantable cells using tools based on CRISPR-Cas9 “is a realistic scenario for autologous cell replacement therapies to restore organ function in many genetic disorders,” they wrote. But so far, muscle stem cells have been left out in part because there has not been a way to isolate and propagate them.
In this study, the German team used mRNA-based delivery of SpCas9 linked to an adenine base editor to achieve greater than 90 percent efficiency in genome editing in human muscle stem cells from a variety of donors and without any enrichment.
“Moreover, mRNA-based delivery of a base editor led to the highly efficient repair of a muscular dystrophy-causing SGCA mutation in a single selection-free step,” they wrote. “Our work establishes mRNA-mediated delivery of CRISPR/Cas9-based tools as a promising and universal approach for taking gene edited muscle stem cells into clinical application to treat muscle disease.”
FDA grants Rare Pediatric Designation to a beta thalassemia CRISPR therapy
Earlier this year, Editas Medicine announced that the U.S. Food and Drug Administration had granted Rare Pediatric Disease designation to its CRISPR-edited cell therapy under development for transfusion-dependent beta thalassemia (TDT) and sickle cell disease.
Under the designation program, the FDA awards priority review to those developing products that meet certain criteria and are means to treat rare pediatric ailments. The agency received 284 requests in 2020, a 330 percent increase from 2019, although it did not disclose how many were approved.
For Editas, the award was for EDIT-301, which was developed using CRISPR-Cas12a in a process that would mimic a hereditary persistence of fetal hemoglobin expression observed in some patients, which is associated with protection from severe forms of sickle cell disease or transfusion-dependent beta thalassemia.
Treating Alzheimer’s with CRISPR?
As reported in May, researchers in Sweden successfully used CRISPR-Cas9 to selectively disrupt a mutant allele that causes familial Alzheimer’s disease.
In a proof-of-concept study, the researchers showed that targeting the mutant allele using CRISPR-Cas9 could reverse Alzheimer’s phenotypes.
“We realize that our approach is still at a very preliminary stage,” the lead author said. “But this type of research could bring us one step closer to developing efficient therapies for AD and other neurodegenerative diseases.”
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