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New gene editing strategy could lead to treatments for people born with inherited diseases of the immune system

New gene editing strategy could lead to treatments for people born with inherited diseases of the immune system

A new gene editing strategy could lead to treatments for people born with inherited diseases of the immune system. The strategy, developed by researchers at the Stanford University School of Medicine, involves targeting a specific DNA sequence in the genome with a novel RNA-based approach.

The approach, called base editing, allows for the direct conversion of one DNA base pair to another. This enables the correction of point mutations that lead to disease.

“Our strategy can be used to efficiently correct mutations in any disease-causing genes,” said lead author David R. Liu, PhD, the Richard Merkin Professor and Director of the Merkin Institute for Transformative Technologies in Healthcare. “We envision that base editing could one day enable precise, detainee and permanent DNA base conversions in patients’ cells to safely and effectively treat or even cure genetic diseases.”

The researchers demonstrated the feasibility of their approach in human cells and in mouse models of two inherited diseases of the immune system, X-linked severe combined immunodeficiency (SCID-X1) and Wiskott-Aldrich syndrome (WAS).

In SCID-X1, mutations in the IL2RG gene lead to a lack of a protein called gamma c, which is essential for the development and function of several types of immune cells. patients with SCID-X1 lack a functional immune system and are susceptible to life-threatening infections.

In WAS, mutations in the WAS gene lead to a lack of a protein called WASp, which is important for the development and function of lymphocytes and other white blood cells. patients with WAS usually have low levels of lymphocytes and are also susceptible to infections.

“We are excited about the potential of base editing to correct disease-causing mutations in patients’ cells and restore health,” said co-author Stanley Riddell, MD, the Director of Cell and Gene Therapy and the head of the Cancer Immunotherapy Program at Fred Hutchinson Cancer Research Center. “This approach has the potential to transform the way we treat genetic diseases.”

The researchers are now working on developing base editors that can target other disease-causing mutations and expanding the base editing technology to other cell types.

“We believe that this approach has the potential to broadly impact human health by providing a new way to treatment or cure many genetic diseases,” said Liu.

In a new study, researchers have developed a gene editing strategy that could lead to treatments for people born with inherited diseases of the immune system.

The study, published in the journal Nature Medicine, used a technique called CRISPR-Cas9 to edit the genes of human cells. The researchers were able to successfully edit the genes of cells from patients with two different types of immunodeficiency: X-linked severe combined immunodeficiency (X-SCID), which is caused by a mutation in the IL2RG gene, and Wiskott-Aldrich Syndrome (WAS), caused by a mutation in the WAS gene.

The researchers hope that this technique could be used to treat other genetic diseases of the immune system, such as primary immunodeficiency disorders (PIDDs). PIDDs are a group of more than 200 disorders that are caused by mutations in genes that are important for the development and function of the immune system.

The current treatment for patients with PIDDs is immunoglobulin therapy, which can be expensive and can have side effects.Gene therapy is a promising alternative treatment option, but it is costly and has been associated with serious side effects, such as leukemia.

The CRISPR-Cas9 technique has the potential to provide a more targeted and less expensive treatment option for patients with PIDDs. The technique can be used to edit the genes of patient-derived cells, which can then be transplanted back into the patient. This would allow for the correction of the mutated gene and the restoration of normal immune function.

The researchers are currently working to improve the efficiency of the CRISPR-Cas9 technique and to reduce the risk of side effects. They hope to begin clinical trials in the near future.

Source: https://www.nature.com/articles/s41591-019-0407-2

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