Rustam Gilfanov: Boundaries of genetic modification. Breakthroughs, achievements, failures, and limits

2022-07-29 09:00:00 - Los Angeles, California, United States - (PR Distribution™)

Every year, genetic modifications find new areas of application, one way or another. Technologies that were innovative a few years ago are eventually becoming standard tools of clinical practice. 

Ex vivo modification

One of the first relatively successful cases of applying gene therapy for patient treatment took place in 1990. During the treatment, the patient's lymphocytes were taken, modified ex vivo using a retroviral vector, and delivered back to her body.

Numerous ex vivo modifications have been introduced to clinical practice to help against various disorders, with CAR-T therapy being one of the most promising methods. 

CRISPR gene editing

A 2015 Nature article on CRISPR/Cas9 technology [11] called it a “game-changer” and compared its development to the introduction of PCR to laboratory practice. “Molecular scissors” have indeed provided vast clinical opportunities for editing genes with utmost precision, “cutting off” defective genes and replacing them with normally functioning ones. 

CRISPR is used with relative success for treating ?-thalassemia, sickle cell anemia, and Duchenne muscular dystrophy. However, it has not fully become a systemic clinical practice.

Cancer therapy is another promising area of application for "molecular scissors." During their research, Chinese scientists used CRISPR to edit the genes of patients with non-small-cell lung carcinoma and disrupt a gene encoding the PD1 protein. The researchers concluded the technology is safe for clinical practice; however, further studies expressed concerns over the long-term health effects of genome editing.

In general, doubts about CRISPR accuracy are the main obstacle to making it a routine clinical practice. Researchers still cannot be 100% sure that "scissors" will do their cutting as intended, raising questions over the potential outcomes of such editing. 

 About the Author

Rustam Gilfanov is an IT entrepreneur and a venture partner of the LongeVC fund.

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