"Science" magazine details: It takes time to treat with CRISPR

Whenever new technologies emerge, there will always be a lot of people excited, but in reality, it will take a while.

Researchers still have a long way to go before using CRISPR to repair genes in patients.

Recently, scientists gathered in Washington, DC to participate in an annual conference focused on gene therapy. Gene therapy is a long-standing struggle and has recently regained respect for a series of promising results in small clinical trials. Nowadays, many people believe that a powerful new gene editing technology called CRISPR will join the increasingly powerful gene therapy army.

But is CRISPR really ready? Science magazine recently explored the prospects and risks of this new technology.

Increasing attention

Traditional gene therapy works through a relatively "brute force" gene transfer method. A harmless virus or some other form of vector that carries a good copy of a gene into a cell. This copy can make up for the defective gene that is causing the disease. However, CRISPR can directly repair defective genes by cutting out bad DNA and replacing it with the correct sequence. In principle, this should work better than adding a new gene because it excludes the risk that foreign genes "land" in the wrong place in the cell's genome and open the cancer gene. The gene repaired by CRISPR will be under the control of the natural promoter of the gene, so the protein produced by the cell will not be too much or too little.

The researchers released some successful cases of using CRISPR to treat animals with hereditary liver disease and muscular dystrophy. At the same time, more such preclinical reports appeared at the annual meeting of the American Society of Gene and Cell Therapy (ASGCT). Discussions around CRISPR are increasing. This year's ASGCT conference had 93 abstracts on CRISPR (768 articles), compared to 33 last year. More importantly, investors are flocking to the field of CRISPR. Three startups, Editas Pharmaceuticals, Intellia Therapeutics, and CRISPR Therapy, have attracted hundreds of millions of dollars in investment.

However, there is still a long way to go before it can be safely and effectively used to repair human genes. This is especially true for most diseases such as muscular dystrophy and cystic fibrosis. These diseases require the correction of genes in living humans, because if cells are first removed, repaired, and then returned, few cells can survive. The need to treat cells in the body means that genetic editing, like gene transfer, faces many of the same challenges in transporting cells. For example, researchers must design effective ways to get a working CRISPR into a specific organization.

CRISPR also poses a security risk. The most frequently mentioned problem is that the Cas9 enzyme used by CRISPR to break DNA at a specific location will also be cut out unexpectedly, which may lead to cancer.

Further from the clinic

With these caveats, do people still need CRISPR?

In countries where gene therapy has been approved for a rare metabolic disorder, regulators are preparing to approve a second gene therapy for an immune disease called severely integrated immunodeficiency (SCID). In the United States, a company is expected to be approved this year to launch a gene transfer therapy for a blind eye in children called Leber congenital black sputum (LCA).

At the ASCGT meeting, researchers working with Bluebird Bio showed a temporary data on a later trial. The results suggest that gene addition can prevent the deterioration of a destructive childhood neurological disease, adrenal leukodystrophy. The final result will lay the groundwork for regulatory approval. Bluebird also reported the use of gene transfer to treat two blood diseases, sickle cell disease and beta-thalassemia, to make these therapies further clinical.

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