Genomeediting

Genome editing is a set of techniques that enable precise modification of an organism’s DNA. It includes programmable nucleases such as zinc finger nucleases (ZFNs) and TAL effector nucleases (TALENs), and the CRISPR-Cas systems, which use a guide RNA to direct a Cas nuclease to a target sequence. Newer approaches, such as base editing and prime editing, allow changes to the genetic code with greater precision or without creating double-strand breaks. Epigenome editing aims to alter gene activity without changing the underlying DNA sequence.

Applications of genome editing span basic research, medicine, and agriculture. In research, it accelerates functional genomics and the creation of disease models. In medicine, somatic editing targets patient cells to treat conditions such as blood disorders, cancer, and genetic diseases; several clinical trials are underway. Germline editing, which would affect eggs, sperm, or embryos and thereby be heritable, remains controversial and is restricted or prohibited in many countries due to safety, ethical, and social concerns.

Delivery of editing components to cells is a critical technical challenge, with methods including viral vectors, lipid nanoparticles, and physical approaches. Off-target effects and mosaicism pose safety considerations, and robust regulatory oversight is essential as the technology progresses.

Historically, the field gained prominence with the development of CRISPR-Cas9, and researchers like Jennifer Doudna and Emmanuelle Charpentier contributed to its foundational work; they were awarded the Nobel Prize in Chemistry in 2020. Regulatory frameworks continue to evolve, reflecting ongoing advances and societal dialogue about responsible use.