With a new CRISPR gene-editing methodology, scientists from the University of Illinois at Urbana-Champaign inactivated one of the genes responsible for an inherited form of amyotrophic lateral sclerosis – a debilitating and fatal neurological disease for which there is no cure. The novel treatment slowed disease progression, improved muscle function and extended lifespan in mice with an aggressive form of ALS.
“ALS unfortunately has few treatment options. This is an important first step in showing that this new form of gene editing could be used to potentially treat the disease,” said bioengineering professor Thomas Gaj, who co-led the study with bioengineering professor Pablo Perez-Pinera.
The method relied on an emerging gene-editing technology known as CRISPR base editors.
Traditional CRISPR gene-editing technologies cut both strands of a DNA molecule, which can introduce a variety of errors in the DNA sequence, limiting its efficiency and potentially leading to a number of unintended mutations in the genome. The Illinois group instead used base editing “to change one letter of the DNA sequence to another without cutting through both DNA strands,” Perez-Pinera said.
“Base editors are too large to be delivered into cells with one of the most promising and successful gene therapy vectors, known as adeno-associated virus,” Gaj said. However, in 2019, Perez-Pinera’s group developed a method of splitting the base editor proteins into halves that can be delivered by two separate AAV particles. Once inside the cell, the halves reassemble into the full-length base editor protein.
By combining the power of AAV gene delivery and split-base editors, Gaj and Perez-Pinera targeted and permanently disabled a mutant SOD1 gene, which is responsible for roughly 20% of inherited forms of ALS. They published their results in the journal Molecular Therapy.
“Many ALS studies are focused on preventing or delaying the onset of the disease. However, in the real world, most patients are not diagnosed until symptoms are advanced,” said graduate student Colin Lim. “Slowing progression, rather than preventing it, may have a greater impact on patients.” Lim is the co-first author of the study along with graduate students Michael Gapinske and Alexandra Brooks.
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