Students Explore Gene Editing and Careers in STEM


Gene editing is an exciting field with enormous potential to help researchers improve our understanding of gene function and develop ways to potentially treat and cure a range of diseases.  This month, students gathered for a webinar where NYSCF Principal Scientist in Functional Genomics Josephine Wesely, PhD, shared how NYSCF uses gene editing in the lab to unlock the secrets of disease. This event was made possible through lead support from the Stavros Niarchos Foundation (SNF).

“So, what is DNA? DNA consists of billions of small units that we call nucleotides (or bases), and it lives in the nucleus of each of our cells,” Dr. Wesely explained. “It is the genetic code that determines how we look, how healthy we are, our eye color, and so much more.”

“If you look at your neighbor, the differences between your genomes is millions of [DNA] bases. These differences can interact with each other and, in some cases, can lead to disease,” she continued. “For us scientists, we are very interested in investigating how the genome plays a role in disease, and gene editing allows us to understand which genes are important for specific diseases.”

How do scientists use gene editing to understand disease? Dr. Wesely gave an example from her work leading NYSCF’s functional genomics team.

Imagine there is one gene that has two variants. We would take stem cells, and [gene edit them to] create – for example – liver cells that contain one of these two variants,” she said. “We can then see that one variant leads to a protein that helps with fatty acid metabolism [a function of the liver], but maybe the other variant leads to a protein that doesn’t do this job correctly.”

Dr. Wesely also explained the different gene editing strategies that scientists can use to investigate what genetic variants do or correct the ones that lead to dysfunction.

“So, we could then try to repair that variant [with gene editing] by changing it to the functional one, or we could try ‘knocking it out’ – essentially deleting it – to see what happens to the protein produced at the end.”

Importantly, there are many ethical questions surrounding gene editing, and students were encouraged to share their opinions on how the technology should be used: whether for research, cell replacement therapies, improving agriculture, preventing disease, or other applications. 

Dr. Wesely also answered questions about gene editing and how students might go about following a career path similar to hers.

“What made you interested in becoming a scientist?” asked one student.

“I was diagnosed with type one diabetes when I was around 10 years old,” she recalled. “That triggered my curiosity to figure out more about my own disease, and I was always interested in optimizing things – specifically, how my disease was treated. So if you are a curious person who is interested in biology, then this may be the field for you.”

“What do you find most hopeful about the field of genome editing?” asked another student.

“There are actually therapies now – for example, for sickle cell anemia – that apply CRISPR [gene editing] to give patients a better life,” Dr. Wesely noted. “And so as a type one diabetic, I’m hopeful for a cell transplant for myself in the future. It’s really encouraging to see how the field is moving really fast in the right direction.”

Hear more on Dr. Wesely’s journey with type 1 diabetes and how she is working on gene editing research at NYSCF towards a cure.

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Diseases & Conditions:

Genomics & Gene Editing