Clinical Trial
October 14, 2020 10:14 amA test of a new medical treatment or procedure in human volunteers with the purpose of evaluating a medical, surgical... View Article
Archives
Clinical Trial Application (CTA)
October 14, 2020 10:14 amAlso known as a CTA. Documentation required by regulatory authorities in many countries outside the U.S. seeking authorization to move... View Article
CRISPR/Cas9
June 29, 2020 9:35 amAdapted from a naturally occurring bacterial immune system, CRISPR is an acronym for Clustered Regularly Interspaced Short Palindromic Repeats. One... View Article
DNA
June 29, 2020 9:35 amAcronym for deoxyribonucleic acid, the hereditary material in humans and almost all other organisms. DNA can be found in the... View Article
Durability
October 14, 2020 11:11 amUsed to describe gene edits or protein expression sustained over a long period of time. Durability is an important indicator... View Article
Endpoint
October 14, 2020 11:20 amAn outcome of a clinical trial measuring a direct clinical benefit, such as safety, survival, decreased pain or the absence... View Article
Ex Vivo Therapy
June 29, 2020 9:36 amAlso referred to as a cell therapy. In an ex vivo therapy, cells are removed from the body for modification.... View Article
Factor 9 Gene
October 14, 2020 11:13 amAlso known as F9 gene. F9 is the gene that produces the FIX protein and is the target for Intellia... View Article
Factor IX Protein
October 14, 2020 11:14 amAlso known as FIX protein. FIX is a blood-clotting protein that is missing or defective in hemophilia B patients. Intellia... View Article
Gene Sequence
October 14, 2020 11:15 amAlso known as gene or DNA sequencing. Researchers use sequencing to determine the order of the four chemical bases that... View Article