A single dose of Crispr-Cas9 therapy has been shown to have very useful effect on patients with hereditary angioedema. The DNA cutting and pasting system has proven to have very potent use in addressing the genetic condition. Angioedema patients that received a single dose of the gene therapy Crispr-Cas9 had little or no symptoms of the disease after the treatment, the study reveals.
The Crispr-Cas9 gene editing involves the use of a guide RNA molecule to detect the target gene. Once the faulty gene is located, Cas9 enzyme is used to cut through the DNA strands, to pave for either replacement of the faulty copy with a healthy one, or inactivation of the non-functional copy.
The single-dose therapy seems very promising with great potentials for use in the treatment of other genetic conditions. The treatment may provide a permanent solution for the terrible symptoms that hereditary angioedema patients experience.
Hereditary angioedema is a rare genetic condition that results in episodic attacks of swelling is different parts of the body including the airways. Most times, it is misdiagnosed as allergy and could result in mortality. Two types of hereditary angioedema have been identified namely: types I and II. Type I is associated with low levels of C1 inhibitor and decreased protein function, while type II has normal C1 inhibitor levels but decreased protein function. Both types are caused by mutations in the SERPING1 gene.
The study published in The New England Journal of Medicine revealed that a single dose of NTLA-2002 resulted in long-lasting reduction in total plasma kallikrein levels without any adverse reactions. According to the study the single-dose therapy resulted in over 95% reduction of angioedema symptoms
ASVEK Science & Tech 