CRISPR – Pros and Cons, Promise, Possibilities, and Concerns
Dr. David Segal, a researcher at the University of California at Davis, gave a talk on CRISPR-Cas at the 2018 FAST Science Summit. He discussed the history and potential applications of CRISPR gene editing technology, including its use in treating genetic diseases like Angelman syndrome. He highlighted the progress made in gene therapy and the potential of CRISPR to correct genetic mutations. However, he also emphasized the need for caution and ethical considerations when using CRISPR, especially in germline editing. He mentioned ongoing research and clinical trials using CRISPR for various applications, including HIV treatment and liver gene therapy. Dr. Segal also mentioned the recent controversial claim of gene-edited babies and the importance of conducting research in a socially and ethically responsible manner. He concluded by discussing potential future applications of CRISPR, such as RNA cutting and epigenetic modifications, and the need for further research to ensure safety and efficacy.
Dr. David Segal, a member of the FAST-FIRE team at the University of California at Davis, delivered a presentation on gene editing and its potential in treating neurologic disorders at the 2018 FAST Science Summit. In his talk, Dr. Segal discussed the revolutionary gene editing tool CRISPR-Cas and its implications for medical research and therapy. He also highlighted the promise and possibilities of CRISPR, while acknowledging the ethical concerns surrounding its use.
The Story of Gene Therapy
Dr. Segal began his talk by sharing his personal journey in the field of biology and his early fascination with gene therapy. He explained how gene therapy, which involves introducing healthy genes into a virus and using the virus to deliver the genes into a person’s cells, was initially met with challenges and skepticism. However, through perseverance and advancements in technology, gene therapy has now become a reality and is being used to treat various genetic diseases.
The Emergence of CRISPR-Cas
Dr. Segal then introduced CRISPR-Cas, a gene editing tool that has gained significant attention in recent years. He asked the audience how many of them were familiar with CRISPR, highlighting its growing popularity and importance in the scientific community. He explained that CRISPR-Cas is not a new concept, as scientists have been manipulating DNA in cells for a long time using different methods. However, CRISPR-Cas has revolutionized gene editing by making it easier and more accessible to researchers.
How CRISPR-Cas Works
Dr. Segal provided an overview of how CRISPR-Cas works. He explained that CRISPR is a targetable nuclease, similar to other gene editing tools like zinc finger nucleases and TALENs. These nucleases can make targeted double-strand breaks in DNA, leading to various downstream effects. By making these breaks, researchers can induce mutations in specific genes or introduce precise changes to the genome.
CRISPR-Cas in Therapy
Dr. Segal discussed the potential applications of CRISPR-Cas in therapy. He mentioned ongoing clinical trials using CRISPR-Cas to treat HIV by making cells resistant to the virus. He also highlighted the use of CRISPR-Cas to insert therapeutic genes into specific cells, such as liver cells, to treat lysosomal storage diseases and hemophilia. However, he emphasized the challenges of in vivo somatic cell editing, where the nuclease is delivered directly into the patient’s body, and the need for further research to ensure safety and efficacy.
Ethical Concerns and Recent Controversy
Dr. Segal addressed the ethical concerns surrounding gene editing, particularly germline gene editing, which involves editing embryos and potentially altering the genetic makeup of future generations. He mentioned the recent controversy surrounding a researcher who claimed to have edited human embryos and grown them into gene-edited babies. Dr. Segal emphasized the importance of conducting research in a socially and ethically responsible manner and the need for careful consideration of the consequences and safety of gene editing technologies.
CRISPR-Cas and Angelman Syndrome
Dr. Segal briefly discussed the potential use of CRISPR-Cas in treating Angelman syndrome, a neurodevelopmental disorder caused by mutations in the UBE3A gene. He mentioned ongoing research by Dr. Mark Zilka, who is using CRISPR-Cas to target specific regions of DNA and reactivate UBE3A expression. Dr. Segal also mentioned the possibility of using CRISPR to modify the epigenetic information that controls gene expression, potentially allowing UBE3A to be expressed in the brain without making targeted double-strand breaks. However, he emphasized the need for further research and safety considerations before these approaches can be used as therapies.
Dr. Segal concluded his talk by highlighting the tremendous potential of CRISPR-Cas in treating genetic diseases, including Angelman syndrome. He acknowledged the ongoing research and development in the field and the efforts to ensure the safety and efficacy of gene editing technologies. He encouraged further exploration and responsible use of CRISPR-Cas, while also acknowledging the need for caution and ethical considerations.
- Title: CRISPR – Pros and Cons, Promise, Possibilities, and Concerns
- Author(s): David Segal
- Author(s)’ affiliation: University of California, Davis
- Publication date: 2019-01-10
- Collection: 2018 FAST Science Summit