CRISPR-Cas9 Gene Editing in Treating Genetic Disorders

The article explores various aspects on role of CRISPR-Cas9 a gene editing technology in treating genetic disorders. CRISPR-Cas9 makes changes at the DNA level, opens the door for curing diseases such as sickle cell disease and cystic fibrosis.

Crissa9 has proved its effectiveness in the correction of diseases associated with genetic mutations on the DNA level. Some of its application is a monogenic disorders treatment, improving cancer treatments and in the creation of gene therapies. Some of the issues in the use of the treatment include the routes of administration, side effects and ethical factors. Recent advancements are the constant improvement of the methods to achieve the best outcome with the help of CRISPR and usage in clinical practice.

Some preclinical and clinical studies have shown how the use of CRISPR-Cas9 results in the modification of the related genetic error. In sickle cell disease latest methods such as CRISPR has been employed to enhance Hbf, and thus reduce the severity of the disease. For instance in Cystic fibrosis CRISPR has seen it fit to target the CFTR gene and bring it back to functionality. It has also been used in experimental cancer therapy and has produced some encouraging outcomes of eliminating cancer cells.

Author: Noah Sinclair

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