Researchers at ETH Zurich have developed advanced CRISPR gene editing technology to modify an entire gene network in one shot. This is a significant step forward in correcting genetic disorders, as many genetic abnormalities and mutations that lead to palpable symptoms are controlled by several genes in various locations on the genome. The advance resulted from the use of a CRISPR enzyme called Cas12a, as opposed to Cas9, which is currently used for all gene editing done with the CRISP technology.
The FDA panel’s unanimous recommendation to approve an autologous leukemia treatment represents a paradigm shift in medicine in which gene therapies and stem cells will play leading roles.
The treatment involves altering the genes of T cells, which are highly specialized stem cells obtained from bone marrow, to target a specific protein on the surface of defective immune cells that cause leukemia. It works by harvesting the cells from the patient, engineering them to target the protein CD-19 on the surface of B Cells, and intravenously administering the cells back into the patient, where they multiply and essentially eradicate the B Cells. Showing promise, over 80% of the patients in the trial have gone into remission.
A recent article in the NY Times served to highlight the progress the medical community is making in combating genetic disorders.
The development of Crispr-Cas9 gene editing technology is profoundly altering the way the medical community is approaching the treatment of genetic disorders. By enhancing accuracy and simplifying the process for the removal and insertion of specific genes in the DNA sequence, Crispr-Cas9 has brought gene therapy back to the forefront of research in the treatment of genetic disorders.To no surprise, one of the key components of this treatment approach are stem cells.
Scientists from the National Institute of Allergy and Infectious Diseases have developed a gene therapy utilizing stem cells, which restores immunity to patients with SCID-X1, also referred to as “Bubble Boy” disease. This genetic disorder prevents immune cells from developing properly, leaving patients prone to potentially fatal infections.