91自拍

A new gene-editing therapy for sickle cell disease is using CRISPR to correct the mutation behind the disease, developers have announced.

Patients are being enrolled for a US-based trial, which will seek to correct the mutation within patients鈥� own blood stem cells, using a non-viral method to deliver the CRISPR-Cas9 editing technology into cells.

Developers say it will be a significant improvement in gene therapy for the disease, increasing the number of corrected cells by 50 times.

Rather than using viral vectors to deliver the gene editing technology into cells, the researchers seek to use electroporation 鈥� a widely-used laboratory technique using small electrical impulses to open pores in the cell membrane.

The first phase of the study, at the University of California, San Francisco (UCSF) Benioff Children鈥檚 Hospital, Oakland, will involve six adult patients. If the treatment is found to be safe and effective, the researchers hope to enrol three adolescent patients.

Researcher Professor Donald Kohn, of the University of California, Los Angeles, said: 鈥淭he use of CRISPR gene editing to fix the sickle cell disease-causing mutation in each patient鈥檚 own blood-forming stem cells required the development of new methods to produce more than 100 million cells per patient that are gene-corrected, healthy and pure.

鈥淭his represents a significant increase in scale from prior research that was limited to producing one to two million genetically corrected cells.鈥�

The principal investigator Professor Mark Walters, of UCSF, said: 鈥淭his therapy is intended to eliminate sickle cell disease by applying CRISPR technology that is safer than a standard stem cell transplant from a healthy bone marrow donor.

鈥淚t is a potential game changer for young sickle cell patients because the therapy eliminates the need for a suitable donor and removes the mutation for a life free of sickle cell disease.鈥�

Source: University of California San Francisco Medical Center

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