323 - CRISPR and the future of gene editing: scientific advances, genetic therapies, & more

A short DNA sequence adjacent to the target that Cas proteins require for activation of recognition and cleavage, preventing the CRISPR system from cutting the bacteria's own DNA.

An earlier gene editing technology that uses protein domains (zinc fingers) to recognize specific three-letter DNA sequences, but was challenging to engineer for precise targeting.

A genetically modified mouse used in biomedical research, whose creation has been dramatically accelerated by CRISPR technology from years to months.

A genetic eye disease causing blindness, treated by Luxturna which provides a missing gene to restore some light sensitivity.

Guest on the podcast, a prominent scientist in the field of gene editing.

An older gene editing system derived from bacteria that uses proteins with programmable repeat domains to recognize single DNA letters, offering more ease than ZFNs but still cumbersome.

A researcher who, along with Maria Jasin, studied how DNA breaks are repaired in cells, crucial for understanding gene editing processes.

A delivery system used for mRNA vaccines (like COVID-19) and for delivering Cas9 and guide RNA into cells, especially liver cells, due to lipid recycling in the liver.

A biotech company that was developing a CRISPR strategy for LCA10.

A genetic isoform possibly linked to Alzheimer's disease, discussed in the context of ethical germline editing and its potential for improvement of human health.

A naturally occurring virus engineered to remove pathogenic elements and used as a vector to insert genes, such as the optogenetic algal gene, into brain cells.

Enzymes, such as nucleases, that work with CRISPR RNA to recognize and cut foreign DNA, particularly viral sequences, in bacteria.

An RNA-analog to Cas9, Cas13 is a CRISPR-associated protein that protects bacteria against RNA viruses by cleaving their RNA genome and has a 'suicidal' function that effectively shuts down infected cells.

Technologies developed using Cas13, such as for COVID-19 detection, designed to be simple and rapid, usable at a point of care or potentially at home.

A Spanish researcher who first observed that the "non-repeating" sequences within CRISPR corresponded to viral DNA, leading to understanding CRISPR's defense function.

A widely used CRISPR-associated protein that acts as an RNA-guided DNA targeting and cleaving enzyme, forming the basis for many gene editing applications.

An ancestral form of Cas9, a very small protein (450 amino acids) that carries out DNA targeting and cleavage, but works with a much larger guide RNA.

Genes related to lipoprotein(a), which is associated with cardiovascular risk, discussed in the context of germline editing and alternative drug treatments.

A genetic blood disorder caused by a single point mutation leading to misshapen red blood cells, addressed by CRISPR-based therapies in clinical trials.

A versatile gene editing technology that can make targeted insertions, deletions, and all 12 possible point mutations without requiring double-strand DNA breaks or donor DNA templates.

A laboratory-based test for detecting nucleic acid sequences, more complex and slower than Cas13-based diagnostics for viral detection.

Another genetic eye disease causing degenerative vision, for which a CRISPR-based strategy is being developed to inactivate a mutant gene.

Feng Zhang's PhD advisor at Stanford University, with whom he developed optogenetics.

A researcher who, along with Jim Haber, studied how DNA breaks are repaired in cells, informing gene editing strategies.

A method to screen out embryos with genetic mutations during IVF, offering an alternative to germline editing for preventing genetic diseases.

A DNA repair process utilized in gene editing that copies sequences from a template DNA into a DNA break site, allowing for designed changes.

The first gene therapy developed and approved in the US for an eye disease (LCA2), delivering a missing gene to eye cells.

The hospital where Feng Zhang volunteered in a gene therapy lab as a high school sophomore, gaining early experimental science experience.

A gene from green algae that senses light and converts it into electrical current, used in optogenetics to control brain activity.

A type of gene editing that modulates gene expression without altering the underlying DNA sequence, often leveraging modified Cas9 systems.

A reference to a public figure who is HIV positive, used to illustrate the context of HIV infection and immunity.