Novel Gene Repair Technique Could Fix Sickle Cell Mutation

Posted on October 17, 2016 by Disability Help Group

Sickle cell anemia is a blood disorder that produces misshapen red blood cells, raising the risk of blood blockages and early death. This disorder, present from birth, is the result of a gene mutation. Sickle cell primarily affects children of African descent.

For the first time, doctors at the Benioff Children’s Hospital Oakland Research Institute are using gene editing to fix the sickle cell mutation. The researchers used the CRISPR-Cas9 gene editing process to correct the mutation in the stem cells of mice. The mice accepted the engineered cells and helped the body produce healthy blood cells for at least four months.

The length of time the genetic repair held is especially relevant, as any treatment of this disease must produce lasting results. After the animal trials, doctors are confident that the benefits of the potential therapy would well outweigh the risks and efforts.

The team will now move its study to larger-scale animal trials with the goal to develop a gene repair method that will permanently correct the gene mutation and cure the disease. These new findings, to be published in Science Translational Medicine, also raise the hope of better treatments or cures for other blood disorders.

Sickle Cell Anemia Can Qualify Your Child for Social Security Disability Benefits

Because sickle cell anemia is present from birth, your child might qualify for Social Security disability benefits from birth. Once your child’s doctor diagnoses them with sickle cell, talk to us for information on obtaining disability benefits. We can help your child with disability benefits all the way through adulthood.

Call the Disability Help Group to schedule a consultation with one of our disability advocates today: 800-800-2009.