Bubble Boy Disease: A New Cure Using CRISPR Technology?

We just read exciting news for the SCID community from Stanford researchers. The original study was published in Nature Communications, but a summary appeared in Medical Xpress. The researchers have used the CRISPR-Cas9 gene editing system to replace the mutated gene that causes “bubble boy disease” (X-linked severe combined immunodeficiency, or SCID-X1) in mice.  SCID-X1 affects 1 in 50,000 males and is caused from a mutation in the 1L2R gamma gene, which prevent the immune system from developing.  Researchers took immune cell samples from people with SCID-X1. genetically altered those stem cells using CRISPR-Cas9 technology, and placed the genetically-altered cells into mice.  The mice were able to generate new immune cells– a promising result. The hope is that this CRISPR-Cas9 gene therapy can studied in humans in upcoming years.

Read the source article at Medical Xpress.

What are your thoughts on these research results? Share your stories, thoughts, and hopes with the Patient Worthy community!



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