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Genetic Editing Human Clinical Trials to Begin by Mickael Marsali

CRISPR (i.e. clustered regularly interspaced short palindromic repeat), a segment of gene editing treatment that is thought to be one of the cutting edge developments in genetic engineering, is finally approaching clinical trials to be used on humans. Many are hopeful that cell therapies using CRISPR will be able to aid in the treatment and prevention of cancer and the vulnerabilities surrounding it. So what does this exactly mean? This means that in the near future, CRISPR is going to be tested to see its safety for humans and then eventually tested to see its efficacy against Cancerous diseases. CRISPR is considered one of the most reliable and effective genome editing techniques in the medical world today.

Procedure

Researchers at the University of Pennsylvania have been called upon to manufacture the CRISPR cells. Then they are going to be shipped and eventually tested in the United States, specifically in California and Texas. Funding will be provided for these clinical trials by the Parker Foundation for Immunotherapy. Throughout the testing, there will be three types of CRISPR edits that are going to be tested. In regards to the testing itself, researchers will use specific T-cells from 18 individuals with various forms of Melanoma. The T-cells will be subjected to three CRISPR edits.

Edit One

Proteins designed to detect cancer cells will be added and the T-cells will be instructed to target the malicious cancer cells.

Edit Two

A certain type of T-cell that may interrupt the process will be removed during this edit.

Edit Three

A gene that identifies T-cells as immune cells will be removed so that the cancer cells cannot disable them.

After the three edits are performed on the T-cells, all of the newly edited cells be transported back into the patient.

These clinical trials have been much anticipated over the past year since the original announcement about CRISPR. The technique is heavily favored as the future of immunotherapy due to its relative ease in nature when testing. CRISPR will also be tested on various other diseases as soon as 2017. There is much success that surrounds CRISPR, but researchers are looking to ensure that no unintended changes are made during the editing process, as this can be one of the largest obstacles that needs to be “ironed out” moving forward.