Genome Editing and Gene Therapy
Big idea: This Grade 12 capstone connects guide RNA, programmable nuclease, off-target effect, ex vivo gene therapy, somatic versus germline editing to evaluate complex biological systems through evidence, uncertainty, responsible inference and real-world application.
Learning goals
- guide RNA
- programmable nuclease
- off-target effect
- ex vivo gene therapy
- somatic versus germline editing
Core concepts
guide RNA
An engineered RNA that directs a programmable nuclease to a complementary target sequence.
programmable nuclease
An enzyme designed to cut or modify DNA at a selected genomic position.
off-target effect
An unintended edit at a genomic site sufficiently similar to the intended target.
ex vivo gene therapy
Removal of patient cells, genetic modification under controlled conditions, testing and return of the cells.
somatic versus germline editing
Somatic edits affect the treated individual, whereas germline edits may be inherited by future generations.