Breakthroughs in medicine during 2015: Gene editing

2015 has seen many discoveries in the world of science and medicine that truly are amazing and not to mention life changing! New advancements in research are achieved and evolving every day, however, during my next few posts I will take you through some of the highlights of the year starting with transformative advances in Gene Editing!

Layla Richards, a one year old child from London, suffered from incurable aggressive leukaemia and was told by doctors that all treatment had failed and thus, on Layla’s first birthday, was advised to take the transition into palliative care. A bone marrow transplant and chemotherapy didn’t work, however, the determination of her family, professionals and a biotechnology company called Cellectis allowed Layla to proceed with an experimental therapy that has so far saved her life.

This treatment had only ever been trialled on mice before and thus, the results were thought unlikely to be successful. The treatment is available due to substantial advances in gene editing and the steps that took place are as follows:

  1. Microscopic scissors (Talens) were used to place the DNA inside the donor’s immune cells.
  2. These cells were designed to kill only leukaemia cells and make them imperceptible from the drugs given to the patient.
  3. These designer cells were injected into Layla, whilst she also received a bone marrow transplant to repair her immune system.

It is too soon following thetreatment to record whether this procedure is a cure, however doctors at the Great Ormond Street Hospital have noted that this is a tremendous breakthrough and one to surely be seen in the future to correct other conditions where cells are engineered and returned to patients in the treatment of cancer.

“We’re in a wonderful place compared to where we were

five months ago, but that doesn’t mean cure.”

-Dr Paul Veys, Great Ormond Street.

Professor Waseem Qasim, involved in Layla’s leukaemia procedure has discussed that treatments involving re-inforcing the immune system to target cancers and other inherited disorders are imminent, where the easiest method will be to cut cells out of the body, modify them and reinsert them back into the patients body. Qasim states that, therefore, sickle cell anaemia or beta thalassaemia, which are diseases of the blood, will be of more focus than heart or kidney defects.

Earlier this year, gene editing has also seen success in making pig organs suitable for human transplant and genetically modified mosquitoes that are resistant to malaria using a technique called CRISPR, which could save the lives of 3.2 billion people which are currently at risk from the disease. This technique has also been used at Sun Yat-sen University in Guangdong to change the genome of a human embryo that results in a blood disorder, in order to avoid any damage and create a perfectly healthy embryo. Of course there are ethical dilemmas that arise alongside these breakthrough, such as the worry of abusing the use of ‘designer babies’, however if gene editing is allowed to become more widely used as a treatment, the opportunities would be truly life-changing.

What are your opinions on gene editing? Please comment any thoughts you have.

Thank you!

View videos covering this story by clicking on the links below:

Photo of Layla Richards by