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